Christine Kilgore

Sleep-disordered breathing is common in patients with neuromuscular disease and is increasingly addressed with noninvasive ventilation, but its patterns go beyond obstructive sleep apnea (OSA) and include hypoventilation, hypoxemia, central sleep apnea, pseudocentrals, periodic breathing, and Cheyne-Stokes respiration, Gaurav Singh, MD, MPH said at the virtual annual meeting of the Associated Professional Sleep Societies.

The prevalence of sleep-related disordered breathing surpasses 40% in patients diagnosed with neuromuscular disease, but “sleep disordered breathing [in these patients] does not equal obstructive sleep apnea,” said Dr. Singh, staff physician at the Veteran Affairs Palo Alto (Calif.) Health Care System in the section of pulmonary, critical care and sleep medicine, and an affiliated clinical assistant professor at Stanford (Calif.) University.

“The most common sleep-related breathing disorder in neuromuscular disease is probably hypopnea and hypoventilation with the sawtooth pattern of dips in oxygen saturation that occur during REM sleep,” he said. As neuromuscular diseases progress, hypoventilation may occur during non-REM sleep as well.

Evaluation is usually performed with polysomnography and pulmonary function testing, he said, but supplementary testing including serum bicarbonate levels, arterial blood gases, and echocardiography to assess for left ventricular ejection fraction and cardiomyopathy may be useful as well.

While a sleep study is not required per Centers for Medicare & Medicaid coverage criteria for the use of respiratory assist devices in patients with neuromuscular disease, polysomnography is valuable for identifying early nocturnal respiratory impairment before the appearance of symptoms and daytime abnormalities in gas exchange, and is better than home testing for distinguishing different types of events (including pseudocentrals). It also is helpful for determining the appropriate pressures needed for ventilatory support and for assessing the need for a backup rate, Dr. Singh said.

Commonly used types of noninvasive ventilation include bilevel positive airway pressure on the spontaneous/timed or pressure control modes, with or without volume-assured pressure support, he said.

Expiratory positive airway pressure (EPAP) is usually set low initially to help decrease the work of breathing and improve triggering, then titrated up to ensure that upper airway obstructive events are treated. Pressure support (the difference between the inspiratory positive airway pressure and EPAP) is set to achieve target tidal volume and to rest the respiratory muscles. And inspiratory time is set “on the longer end” to achieve maximal target volume and ensure appropriate gas exchange, Dr. Singh said.

Data from randomized controlled trials evaluating the effectiveness of NIV are limited, he said. A study published 15 years ago showed a survival benefit and improvement in quality of life measures in patients with amyotrophic lateral sclerosis (ALS) with normal or moderately impaired bulbar function but not in those with severe bulbar weakness.

Regarding the timing of initiating NIV, a retrospective study published several years ago looked at almost 200 ALS patients and evaluated differences in survival amongst those started earlier with NIV (forced vital capacity ≥80%) and those started later (FVC <80%). At 36 months from diagnosis, mortality was 35% for the early group and 53% for the later group. “Improved survival was driven by benefit in patients with non–bulbar-onset ALS, compared with bulbar-onset disease,” Dr. Singh said.

“This study and several other similar studies seem to indicate that the earlier NIV [noninvasive ventilation] is started in patients with neuromuscular disease, the better in terms of improving survival and other relevant measures such as quality of life,” he said.

Asked about Dr. Singh’s presentation, Michelle Cao, DO, clinical associate professor at Stanford University, said that NIV is an “invaluable tool in the treatment of conditions leading to chronic respiratory failure,” such as neuromuscular disease, and that it’s important to incorporate NIV training for future pulmonary, critical care and sleep physicians. Dr. Cao directs the adult NIV program for the neuromuscular medical program at Stanford Health Care.

Saiprakash B. Venkateshiah, MD, of Emory University, Atlanta, also said in introducing Dr. Singh at the meeting that earlier diagnosis and appropriate NIV therapy “may improve quality of life and possibly even lower survival in certain disorders.”

In addition, he noted that sleep disturbances “may be the earliest sign of muscle weakness in [patients with neuromuscular disease], sometimes being detected before their underlying neuromuscular disease is diagnosed.”

Dr. Singh, Dr. Cao, and Dr. Venkateshiah each reported that they had no potential conflicts of interest.

Sleep-disordered breathing is common in patients with neuromuscular disease and is increasingly addressed with noninvasive ventilation, but its patterns go beyond obstructive sleep apnea (OSA) and include hypoventilation, hypoxemia, central sleep apnea, pseudocentrals, periodic breathing, and Cheyne-Stokes respiration, Gaurav Singh, MD, MPH said at the virtual annual meeting of the Associated Professional Sleep Societies.

The prevalence of sleep-related disordered breathing surpasses 40% in patients diagnosed with neuromuscular disease, but “sleep disordered breathing [in these patients] does not equal obstructive sleep apnea,” said Dr. Singh, staff physician at the Veteran Affairs Palo Alto (Calif.) Health Care System in the section of pulmonary, critical care and sleep medicine, and an affiliated clinical assistant professor at Stanford (Calif.) University.

“The most common sleep-related breathing disorder in neuromuscular disease is probably hypopnea and hypoventilation with the sawtooth pattern of dips in oxygen saturation that occur during REM sleep,” he said. As neuromuscular diseases progress, hypoventilation may occur during non-REM sleep as well.

Evaluation is usually performed with polysomnography and pulmonary function testing, he said, but supplementary testing including serum bicarbonate levels, arterial blood gases, and echocardiography to assess for left ventricular ejection fraction and cardiomyopathy may be useful as well.

While a sleep study is not required per Centers for Medicare & Medicaid coverage criteria for the use of respiratory assist devices in patients with neuromuscular disease, polysomnography is valuable for identifying early nocturnal respiratory impairment before the appearance of symptoms and daytime abnormalities in gas exchange, and is better than home testing for distinguishing different types of events (including pseudocentrals). It also is helpful for determining the appropriate pressures needed for ventilatory support and for assessing the need for a backup rate, Dr. Singh said.

Commonly used types of noninvasive ventilation include bilevel positive airway pressure on the spontaneous/timed or pressure control modes, with or without volume-assured pressure support, he said.

Expiratory positive airway pressure (EPAP) is usually set low initially to help decrease the work of breathing and improve triggering, then titrated up to ensure that upper airway obstructive events are treated. Pressure support (the difference between the inspiratory positive airway pressure and EPAP) is set to achieve target tidal volume and to rest the respiratory muscles. And inspiratory time is set “on the longer end” to achieve maximal target volume and ensure appropriate gas exchange, Dr. Singh said.

Data from randomized controlled trials evaluating the effectiveness of NIV are limited, he said. A study published 15 years ago showed a survival benefit and improvement in quality of life measures in patients with amyotrophic lateral sclerosis (ALS) with normal or moderately impaired bulbar function but not in those with severe bulbar weakness.

Regarding the timing of initiating NIV, a retrospective study published several years ago looked at almost 200 ALS patients and evaluated differences in survival amongst those started earlier with NIV (forced vital capacity ≥80%) and those started later (FVC <80%). At 36 months from diagnosis, mortality was 35% for the early group and 53% for the later group. “Improved survival was driven by benefit in patients with non–bulbar-onset ALS, compared with bulbar-onset disease,” Dr. Singh said.

“This study and several other similar studies seem to indicate that the earlier NIV [noninvasive ventilation] is started in patients with neuromuscular disease, the better in terms of improving survival and other relevant measures such as quality of life,” he said.

Asked about Dr. Singh’s presentation, Michelle Cao, DO, clinical associate professor at Stanford University, said that NIV is an “invaluable tool in the treatment of conditions leading to chronic respiratory failure,” such as neuromuscular disease, and that it’s important to incorporate NIV training for future pulmonary, critical care and sleep physicians. Dr. Cao directs the adult NIV program for the neuromuscular medical program at Stanford Health Care.

Saiprakash B. Venkateshiah, MD, of Emory University, Atlanta, also said in introducing Dr. Singh at the meeting that earlier diagnosis and appropriate NIV therapy “may improve quality of life and possibly even lower survival in certain disorders.”

In addition, he noted that sleep disturbances “may be the earliest sign of muscle weakness in [patients with neuromuscular disease], sometimes being detected before their underlying neuromuscular disease is diagnosed.”

Dr. Singh, Dr. Cao, and Dr. Venkateshiah each reported that they had no potential conflicts of interest.

Sleep-disordered breathing is common in patients with neuromuscular disease and is increasingly addressed with noninvasive ventilation, but its patterns go beyond obstructive sleep apnea (OSA) and include hypoventilation, hypoxemia, central sleep apnea, pseudocentrals, periodic breathing, and Cheyne-Stokes respiration, Gaurav Singh, MD, MPH said at the virtual annual meeting of the Associated Professional Sleep Societies.

The prevalence of sleep-related disordered breathing surpasses 40% in patients diagnosed with neuromuscular disease, but “sleep disordered breathing [in these patients] does not equal obstructive sleep apnea,” said Dr. Singh, staff physician at the Veteran Affairs Palo Alto (Calif.) Health Care System in the section of pulmonary, critical care and sleep medicine, and an affiliated clinical assistant professor at Stanford (Calif.) University.

“The most common sleep-related breathing disorder in neuromuscular disease is probably hypopnea and hypoventilation with the sawtooth pattern of dips in oxygen saturation that occur during REM sleep,” he said. As neuromuscular diseases progress, hypoventilation may occur during non-REM sleep as well.

Evaluation is usually performed with polysomnography and pulmonary function testing, he said, but supplementary testing including serum bicarbonate levels, arterial blood gases, and echocardiography to assess for left ventricular ejection fraction and cardiomyopathy may be useful as well.

While a sleep study is not required per Centers for Medicare & Medicaid coverage criteria for the use of respiratory assist devices in patients with neuromuscular disease, polysomnography is valuable for identifying early nocturnal respiratory impairment before the appearance of symptoms and daytime abnormalities in gas exchange, and is better than home testing for distinguishing different types of events (including pseudocentrals). It also is helpful for determining the appropriate pressures needed for ventilatory support and for assessing the need for a backup rate, Dr. Singh said.

Commonly used types of noninvasive ventilation include bilevel positive airway pressure on the spontaneous/timed or pressure control modes, with or without volume-assured pressure support, he said.

Expiratory positive airway pressure (EPAP) is usually set low initially to help decrease the work of breathing and improve triggering, then titrated up to ensure that upper airway obstructive events are treated. Pressure support (the difference between the inspiratory positive airway pressure and EPAP) is set to achieve target tidal volume and to rest the respiratory muscles. And inspiratory time is set “on the longer end” to achieve maximal target volume and ensure appropriate gas exchange, Dr. Singh said.

Data from randomized controlled trials evaluating the effectiveness of NIV are limited, he said. A study published 15 years ago showed a survival benefit and improvement in quality of life measures in patients with amyotrophic lateral sclerosis (ALS) with normal or moderately impaired bulbar function but not in those with severe bulbar weakness.

Regarding the timing of initiating NIV, a retrospective study published several years ago looked at almost 200 ALS patients and evaluated differences in survival amongst those started earlier with NIV (forced vital capacity ≥80%) and those started later (FVC <80%). At 36 months from diagnosis, mortality was 35% for the early group and 53% for the later group. “Improved survival was driven by benefit in patients with non–bulbar-onset ALS, compared with bulbar-onset disease,” Dr. Singh said.

“This study and several other similar studies seem to indicate that the earlier NIV [noninvasive ventilation] is started in patients with neuromuscular disease, the better in terms of improving survival and other relevant measures such as quality of life,” he said.

Asked about Dr. Singh’s presentation, Michelle Cao, DO, clinical associate professor at Stanford University, said that NIV is an “invaluable tool in the treatment of conditions leading to chronic respiratory failure,” such as neuromuscular disease, and that it’s important to incorporate NIV training for future pulmonary, critical care and sleep physicians. Dr. Cao directs the adult NIV program for the neuromuscular medical program at Stanford Health Care.

Saiprakash B. Venkateshiah, MD, of Emory University, Atlanta, also said in introducing Dr. Singh at the meeting that earlier diagnosis and appropriate NIV therapy “may improve quality of life and possibly even lower survival in certain disorders.”

In addition, he noted that sleep disturbances “may be the earliest sign of muscle weakness in [patients with neuromuscular disease], sometimes being detected before their underlying neuromuscular disease is diagnosed.”

Dr. Singh, Dr. Cao, and Dr. Venkateshiah each reported that they had no potential conflicts of interest.

Pregnant women who have even mild sleep apnea should be treated for their sleep-disordered breathing given what is known about associated risks for hypertensive disorders of pregnancy and gestational diabetes, Carolyn M. D’Ambrosio, MS, MD, FCCP, said at the virtual annual meeting of the Associated Professional Sleep Societies.

“This is the current standard of care,” Dr. D’Ambrosio said. “Although guidelines on this issue are not hard and fast, I’d say that knowing what we know about the risk of adverse [maternal] outcomes, we should all try to treat these problems as soon as they’re identified” and then repeat polysomnography or home sleep testing 3-6 months post partum to “be sure the sleep-disordered breathing has resolved.”

Estimates of obstructive sleep apnea (OSA) prevalence range from approximately 9% in the first trimester to 20% in the third trimester. Yet recognizing the significance of OSA in pregnant women and identifying women for testing remains a major challenge. “Most women won’t [report sleep problems] because it’s pretty much common folklore that you don’t sleep well when you’re pregnant,” said Dr. D’Ambrosio, associate professor of medicine at Harvard Medical School, Boston, and current past-chair of the Women’s Lung Health Network for CHEST.

Many obstetricians and obstetrics providers, meanwhile, do not adequately screen. Typical screening tools like the Epworth Sleepiness Scale have low sensitivity and specificity during pregnancy, which means that inquiries about sleepiness, snoring, and disruptions in sleep are important, as is attention to potential risks for OSA posed by obesity, chronic hypertension, and neck circumference.

Only about a quarter of women in the United States snore during pregnancy, she noted. Snoring prevalence does increase as pregnancy progresses, reaching up to almost 50% in during the third trimester in some studies.

A four-variable screening tool reported almost 10 years ago for pregnant women is reliable for gauging risk, Dr. D’Ambrosio said. The model considers self-reported frequent snoring (more than three times/week), chronic hypertension, advanced maternal age, and a pregestational body mass index of at least 30 kg/m². “If these [factors] are present, the patient is at significant risk for OSA and should be strongly considered for testing,” she said.

Home sleep apnea testing (HSAT) is validated for pregnant women but “it can underestimate,” she said. “If you get a negative result and [have clinical suspicion], then don’t stop there.”

And considering that the prevalence of OSA – at all levels of severity – increases as pregnancy progresses, it’s important to continue talking about sleep with patients who have frequent snoring, for instance, but negative sleep test results early in pregnancy. “They could develop [OSA] as time goes on,” she said.
 

Associated risk factors

Independent associations between sleep-disordered breathing and adverse maternal outcomes were demonstrated in a prospective cohort study published several years ago of 3,705 women who underwent HSAT in early and mid-pregnancy. The adjusted odds ratios for preeclampsia when sleep-disordered breathing (an apnea-hypopnea index of ≥5) was present early in pregnancy and in mid-pregnancy were 1.94 and 1.95, respectively.

For hypertensive disorders of pregnancy more broadly, the ORs were 1.46 and 1.73, and for gestational diabetes, the ORs were 3.47 and 2.79.

“Faced with the question about why it’s important to diagnosis and treat OSA [during pregnancy] since the pregnancy will be over in a few months, I go to this study,” Dr. D’Ambrosio said. “Waiting until the end of pregnancy is not safe. There are increased risks of very serious conditions if sleep apnea is there and it’s not treated.”

Another study demonstrating a link between OSA and maternal outcomes looked over 1.5 million deliveries in the United States and found a significantly higher prevalence of gestational diabetes (OR, 2.08), gestational hypertension (OR, 1.77), preeclampsia (OR, 2.07), and eclampsia (OR, 2.70) in pregnant women with OSA than without, after adjusting for maternal obesity. Associations remained significant after adjusting for a more comprehensive list of covariates.

Multiple potential casual pathways are at play, Dr. D’Ambrosio said. Short sleep duration decreases leptin and increases ghrelin levels, for instance, and sleep fragmentation activates the HPA axis and increases cortisol. Intermittent hypoxemia affects sympathetic activity, and intrathoracic pressure swings cause increased oxidative stress and systemic inflammation.

The resulting endothelial dysfunction, glucose dysfunction, and dyslipidemia can drive the adverse maternal outcomes documented in these studies, she said, noting that the adverse outcomes can have long-term cardiovascular consequences.

Continuous positive airway pressure therapy is well tolerated in pregnancy, and given pregnancy’s continual weight change, auto-titrating CPAP may be the best option, she said.

There is “some limited data that treatment improves maternal outcomes, and we’re still working on trying to get better data and more solid recommendations,” Dr. D’Ambrosio said. There currently are no guidelines covering the diagnosis and management of OSA during pregnancy.

“We’ve come a long way ... but we still have more to do,” she said. “We have a long way to go to getting [OSA in pregnant women] well recognized, with screening techniques and diagnosis.”

Asked after the meeting about Dr. D’Ambrosio’s messages, Anita Rajagopal, MD, said that OSA screening during pregnancy needs to be improved through more collaboration “with our ob.gyn. and primary care colleagues.”

Too often, she said, “the signs and symptoms of OSA in pregnancy are written off as ‘just harmless snoring’ while in fact the patient has treatable sleep disordered breathing with potential adverse effects.” Dr. Rajagopal is department medical director for sleep medicine at Community Physician Network and medical director of the Community Health Network Sleep-Wake Disorders Center, both in Indianapolis.

Dr. D’Ambrosio reported that she has no potential conflicts of interest related to the material she presented, and Dr. Rajagopal stated she has no potential conflicts of interest.

Pregnant women who have even mild sleep apnea should be treated for their sleep-disordered breathing given what is known about associated risks for hypertensive disorders of pregnancy and gestational diabetes, Carolyn M. D’Ambrosio, MS, MD, FCCP, said at the virtual annual meeting of the Associated Professional Sleep Societies.

“This is the current standard of care,” Dr. D’Ambrosio said. “Although guidelines on this issue are not hard and fast, I’d say that knowing what we know about the risk of adverse [maternal] outcomes, we should all try to treat these problems as soon as they’re identified” and then repeat polysomnography or home sleep testing 3-6 months post partum to “be sure the sleep-disordered breathing has resolved.”

Estimates of obstructive sleep apnea (OSA) prevalence range from approximately 9% in the first trimester to 20% in the third trimester. Yet recognizing the significance of OSA in pregnant women and identifying women for testing remains a major challenge. “Most women won’t [report sleep problems] because it’s pretty much common folklore that you don’t sleep well when you’re pregnant,” said Dr. D’Ambrosio, associate professor of medicine at Harvard Medical School, Boston, and current past-chair of the Women’s Lung Health Network for CHEST.

Many obstetricians and obstetrics providers, meanwhile, do not adequately screen. Typical screening tools like the Epworth Sleepiness Scale have low sensitivity and specificity during pregnancy, which means that inquiries about sleepiness, snoring, and disruptions in sleep are important, as is attention to potential risks for OSA posed by obesity, chronic hypertension, and neck circumference.

Only about a quarter of women in the United States snore during pregnancy, she noted. Snoring prevalence does increase as pregnancy progresses, reaching up to almost 50% in during the third trimester in some studies.

A four-variable screening tool reported almost 10 years ago for pregnant women is reliable for gauging risk, Dr. D’Ambrosio said. The model considers self-reported frequent snoring (more than three times/week), chronic hypertension, advanced maternal age, and a pregestational body mass index of at least 30 kg/m². “If these [factors] are present, the patient is at significant risk for OSA and should be strongly considered for testing,” she said.

Home sleep apnea testing (HSAT) is validated for pregnant women but “it can underestimate,” she said. “If you get a negative result and [have clinical suspicion], then don’t stop there.”

And considering that the prevalence of OSA – at all levels of severity – increases as pregnancy progresses, it’s important to continue talking about sleep with patients who have frequent snoring, for instance, but negative sleep test results early in pregnancy. “They could develop [OSA] as time goes on,” she said.
 

Associated risk factors

Independent associations between sleep-disordered breathing and adverse maternal outcomes were demonstrated in a prospective cohort study published several years ago of 3,705 women who underwent HSAT in early and mid-pregnancy. The adjusted odds ratios for preeclampsia when sleep-disordered breathing (an apnea-hypopnea index of ≥5) was present early in pregnancy and in mid-pregnancy were 1.94 and 1.95, respectively.

For hypertensive disorders of pregnancy more broadly, the ORs were 1.46 and 1.73, and for gestational diabetes, the ORs were 3.47 and 2.79.

“Faced with the question about why it’s important to diagnosis and treat OSA [during pregnancy] since the pregnancy will be over in a few months, I go to this study,” Dr. D’Ambrosio said. “Waiting until the end of pregnancy is not safe. There are increased risks of very serious conditions if sleep apnea is there and it’s not treated.”

Another study demonstrating a link between OSA and maternal outcomes looked over 1.5 million deliveries in the United States and found a significantly higher prevalence of gestational diabetes (OR, 2.08), gestational hypertension (OR, 1.77), preeclampsia (OR, 2.07), and eclampsia (OR, 2.70) in pregnant women with OSA than without, after adjusting for maternal obesity. Associations remained significant after adjusting for a more comprehensive list of covariates.

Multiple potential casual pathways are at play, Dr. D’Ambrosio said. Short sleep duration decreases leptin and increases ghrelin levels, for instance, and sleep fragmentation activates the HPA axis and increases cortisol. Intermittent hypoxemia affects sympathetic activity, and intrathoracic pressure swings cause increased oxidative stress and systemic inflammation.

The resulting endothelial dysfunction, glucose dysfunction, and dyslipidemia can drive the adverse maternal outcomes documented in these studies, she said, noting that the adverse outcomes can have long-term cardiovascular consequences.

Continuous positive airway pressure therapy is well tolerated in pregnancy, and given pregnancy’s continual weight change, auto-titrating CPAP may be the best option, she said.

There is “some limited data that treatment improves maternal outcomes, and we’re still working on trying to get better data and more solid recommendations,” Dr. D’Ambrosio said. There currently are no guidelines covering the diagnosis and management of OSA during pregnancy.

“We’ve come a long way ... but we still have more to do,” she said. “We have a long way to go to getting [OSA in pregnant women] well recognized, with screening techniques and diagnosis.”

Asked after the meeting about Dr. D’Ambrosio’s messages, Anita Rajagopal, MD, said that OSA screening during pregnancy needs to be improved through more collaboration “with our ob.gyn. and primary care colleagues.”

Too often, she said, “the signs and symptoms of OSA in pregnancy are written off as ‘just harmless snoring’ while in fact the patient has treatable sleep disordered breathing with potential adverse effects.” Dr. Rajagopal is department medical director for sleep medicine at Community Physician Network and medical director of the Community Health Network Sleep-Wake Disorders Center, both in Indianapolis.

Dr. D’Ambrosio reported that she has no potential conflicts of interest related to the material she presented, and Dr. Rajagopal stated she has no potential conflicts of interest.

Pregnant women who have even mild sleep apnea should be treated for their sleep-disordered breathing given what is known about associated risks for hypertensive disorders of pregnancy and gestational diabetes, Carolyn M. D’Ambrosio, MS, MD, FCCP, said at the virtual annual meeting of the Associated Professional Sleep Societies.

“This is the current standard of care,” Dr. D’Ambrosio said. “Although guidelines on this issue are not hard and fast, I’d say that knowing what we know about the risk of adverse [maternal] outcomes, we should all try to treat these problems as soon as they’re identified” and then repeat polysomnography or home sleep testing 3-6 months post partum to “be sure the sleep-disordered breathing has resolved.”

Estimates of obstructive sleep apnea (OSA) prevalence range from approximately 9% in the first trimester to 20% in the third trimester. Yet recognizing the significance of OSA in pregnant women and identifying women for testing remains a major challenge. “Most women won’t [report sleep problems] because it’s pretty much common folklore that you don’t sleep well when you’re pregnant,” said Dr. D’Ambrosio, associate professor of medicine at Harvard Medical School, Boston, and current past-chair of the Women’s Lung Health Network for CHEST.

Many obstetricians and obstetrics providers, meanwhile, do not adequately screen. Typical screening tools like the Epworth Sleepiness Scale have low sensitivity and specificity during pregnancy, which means that inquiries about sleepiness, snoring, and disruptions in sleep are important, as is attention to potential risks for OSA posed by obesity, chronic hypertension, and neck circumference.

Only about a quarter of women in the United States snore during pregnancy, she noted. Snoring prevalence does increase as pregnancy progresses, reaching up to almost 50% in during the third trimester in some studies.

A four-variable screening tool reported almost 10 years ago for pregnant women is reliable for gauging risk, Dr. D’Ambrosio said. The model considers self-reported frequent snoring (more than three times/week), chronic hypertension, advanced maternal age, and a pregestational body mass index of at least 30 kg/m². “If these [factors] are present, the patient is at significant risk for OSA and should be strongly considered for testing,” she said.

Home sleep apnea testing (HSAT) is validated for pregnant women but “it can underestimate,” she said. “If you get a negative result and [have clinical suspicion], then don’t stop there.”

And considering that the prevalence of OSA – at all levels of severity – increases as pregnancy progresses, it’s important to continue talking about sleep with patients who have frequent snoring, for instance, but negative sleep test results early in pregnancy. “They could develop [OSA] as time goes on,” she said.
 

Associated risk factors

Independent associations between sleep-disordered breathing and adverse maternal outcomes were demonstrated in a prospective cohort study published several years ago of 3,705 women who underwent HSAT in early and mid-pregnancy. The adjusted odds ratios for preeclampsia when sleep-disordered breathing (an apnea-hypopnea index of ≥5) was present early in pregnancy and in mid-pregnancy were 1.94 and 1.95, respectively.

For hypertensive disorders of pregnancy more broadly, the ORs were 1.46 and 1.73, and for gestational diabetes, the ORs were 3.47 and 2.79.

“Faced with the question about why it’s important to diagnosis and treat OSA [during pregnancy] since the pregnancy will be over in a few months, I go to this study,” Dr. D’Ambrosio said. “Waiting until the end of pregnancy is not safe. There are increased risks of very serious conditions if sleep apnea is there and it’s not treated.”

Another study demonstrating a link between OSA and maternal outcomes looked over 1.5 million deliveries in the United States and found a significantly higher prevalence of gestational diabetes (OR, 2.08), gestational hypertension (OR, 1.77), preeclampsia (OR, 2.07), and eclampsia (OR, 2.70) in pregnant women with OSA than without, after adjusting for maternal obesity. Associations remained significant after adjusting for a more comprehensive list of covariates.

Multiple potential casual pathways are at play, Dr. D’Ambrosio said. Short sleep duration decreases leptin and increases ghrelin levels, for instance, and sleep fragmentation activates the HPA axis and increases cortisol. Intermittent hypoxemia affects sympathetic activity, and intrathoracic pressure swings cause increased oxidative stress and systemic inflammation.

The resulting endothelial dysfunction, glucose dysfunction, and dyslipidemia can drive the adverse maternal outcomes documented in these studies, she said, noting that the adverse outcomes can have long-term cardiovascular consequences.

Continuous positive airway pressure therapy is well tolerated in pregnancy, and given pregnancy’s continual weight change, auto-titrating CPAP may be the best option, she said.

There is “some limited data that treatment improves maternal outcomes, and we’re still working on trying to get better data and more solid recommendations,” Dr. D’Ambrosio said. There currently are no guidelines covering the diagnosis and management of OSA during pregnancy.

“We’ve come a long way ... but we still have more to do,” she said. “We have a long way to go to getting [OSA in pregnant women] well recognized, with screening techniques and diagnosis.”

Asked after the meeting about Dr. D’Ambrosio’s messages, Anita Rajagopal, MD, said that OSA screening during pregnancy needs to be improved through more collaboration “with our ob.gyn. and primary care colleagues.”

Too often, she said, “the signs and symptoms of OSA in pregnancy are written off as ‘just harmless snoring’ while in fact the patient has treatable sleep disordered breathing with potential adverse effects.” Dr. Rajagopal is department medical director for sleep medicine at Community Physician Network and medical director of the Community Health Network Sleep-Wake Disorders Center, both in Indianapolis.

Dr. D’Ambrosio reported that she has no potential conflicts of interest related to the material she presented, and Dr. Rajagopal stated she has no potential conflicts of interest.

Obesity hypoventilation syndrome (OHS) is bound to be increasing because of the rising obesity epidemic but is underrecognized and “frequently underdiagnosed,” Saiprakash B. Venkateshiah, MD, said at the virtual annual meeting of the Associated Professional Sleep Societies.

The condition, which can cause significant morbidity and mortality, is defined by the combination of obesity and awake alveolar hypoventilation (PaCO₂ ≥45 mm Hg), with the exclusion of alternate causes of hypoventilation. Sleep-disordered breathing (SDB) is almost universally present, with approximately 90% of individuals with OHS also having obstructive sleep apnea (OSA), most often severe, and approximately 10% having sleep-related hypoventilation, or a “pure hypoventilation subtype, if you will,” said Dr. Venkateshiah, assistant professor of medicine at Emory University, Atlanta.

The prevalence of OHS in the general population is unknown, but its prevalence in patients who present for the evaluation of SDB has ranged from 8%-20% across multiple studies, he said. Up to 40% of patients with OHS present for the first time with acute hypercapnic respiratory failure, which has an in-hospital mortality of 18%.

Postmenopausal women appear to have a higher prevalence, compared with premenopausal women and men, he noted, and women appear to be more likely than men to present with the clinical phenotype of OHS without associated OSA.

The arterial blood gas measurement needed to document alveolar hypoventilation and definitively diagnosis OHA is a “simple and economical test,” he said, “but it is logistically very difficult to obtain [these measurements] routinely in all patients in the clinic ... and is one of the reasons why OSH is underdiagnosed.”
 

Guideline advice

A practice guideline published in 2019 by the American Thoracic Society suggests that, for obese patients with SDB and a low to moderate probability of having OSH, a serum bicarbonate level be measured first. “In patients with serum bicarbonate less than 27 mmol/L, clinicians might forgo measuring PaCO₂, as the diagnosis in them is very unlikely,” Dr. Venkateshiah said, referring to the guideline. “In patients with a serum bicarbonate greater than 27, you might need to measure PaCO₂ to confirm or rule out the diagnosis of OHS.”

(Patients strongly suspected of having OHS, with more than a low to moderate probability – those in whom arterial blood gases should be measured – are “usually severely obese with typical signs and symptoms such as dyspnea, nocturia, lower-extremity edema, excessive daytime sleepiness, fatigue, loud disruptive snoring, witnessed apneas, as well as mild hypoxemia during wake and/or significant hypoxemia during sleep,” the ATS guideline says.)

The guideline panel considered the use of oxygen saturation measured with pulse oximetry during wakefulness to screen for OHS and decided to advise against it because of the paucity of evidence-based literature, Dr. Venkateshiah noted. (In making its five conditional recommendations, the guideline panel cited an overall very low quality of evidence.)

Symptoms of OHS overlap with those of OSA (for example, daytime hypersomnolence, witnessed apneas, loud snoring, and morning headaches), so “symptoms alone cannot be used to discriminate between the two disorders,” he advised. Signs of OHS commonly seen in clinical exams, however, are low resting daytime oxygen saturations and lower-extremity edema. A sleep study, he added, is needed to document and characterize SDB in patients with OHS.

Positive airway pressure therapy is the first-line treatment for OHS, and long-term outcomes of patients with OHS on PAP treatment are significantly better, compared with untreated individuals. There is no strong evidence to recommend one form of PAP therapy over another for patients with OHS and concomitant severe OSA, he said, but “the bottom line” from both short- and long-term randomized clinical trials comparing CPAP with noninvasive ventilation “is that CPAP is equivalent to noninvasive ventilation as far as outcomes are concerned.”

The ATS guideline panel recommends continuous positive airway pressure therapy for patients with OHS and severe OSA. And for OHS with nonsevere OSA, bilevel PAP is traditionally used – including pure hypoventilators, Dr. Venkateshiah said.

Weight-loss interventions are paramount, since “the primary driver of OHS is obesity,” he said at the meeting. There are only a few studies that have looked at bariatric surgery in patients with OHS, he said, “but they did note significant improvements in gas exchange, sleep apnea, lung volumes and pulmonary hypertension.”

The ATS guideline suggests weight-loss interventions that produce sustained weight loss of 25%-30% of the actual body weight. Such interventions are “most likely required to achieve resolution of hypoventilation,” Dr. Venkateshiah said.
 

OHS vs. COPD

In a separate presentation on OHS, Michelle Cao, DO, clinical associate professor at Stanford (Calif.) University, emphasized the importance of distinguishing the patient with OHS from the patient with hypercapnic chronic obstructive pulmonary disease (COPD). Spirometry and the flow volume curve can help rule out hypercapnic COPD and other conditions that cause daytime hypoventilation.

A study published in 2016 of 600 hospitalized patients determined to have unequivocal OHS found that 43% had been misdiagnosed as having COPD and none had been previously diagnosed with OHS, Dr. Cao noted. Patients in the study had a mean age of 58 and a mean body mass index of 48.2 kg/m²; 64% were women.

Dr. Venkateshiah and Dr. Cao had no relevant disclosures.

Obesity hypoventilation syndrome (OHS) is bound to be increasing because of the rising obesity epidemic but is underrecognized and “frequently underdiagnosed,” Saiprakash B. Venkateshiah, MD, said at the virtual annual meeting of the Associated Professional Sleep Societies.

The condition, which can cause significant morbidity and mortality, is defined by the combination of obesity and awake alveolar hypoventilation (PaCO₂ ≥45 mm Hg), with the exclusion of alternate causes of hypoventilation. Sleep-disordered breathing (SDB) is almost universally present, with approximately 90% of individuals with OHS also having obstructive sleep apnea (OSA), most often severe, and approximately 10% having sleep-related hypoventilation, or a “pure hypoventilation subtype, if you will,” said Dr. Venkateshiah, assistant professor of medicine at Emory University, Atlanta.

The prevalence of OHS in the general population is unknown, but its prevalence in patients who present for the evaluation of SDB has ranged from 8%-20% across multiple studies, he said. Up to 40% of patients with OHS present for the first time with acute hypercapnic respiratory failure, which has an in-hospital mortality of 18%.

Postmenopausal women appear to have a higher prevalence, compared with premenopausal women and men, he noted, and women appear to be more likely than men to present with the clinical phenotype of OHS without associated OSA.

The arterial blood gas measurement needed to document alveolar hypoventilation and definitively diagnosis OHA is a “simple and economical test,” he said, “but it is logistically very difficult to obtain [these measurements] routinely in all patients in the clinic ... and is one of the reasons why OSH is underdiagnosed.”
 

Guideline advice

A practice guideline published in 2019 by the American Thoracic Society suggests that, for obese patients with SDB and a low to moderate probability of having OSH, a serum bicarbonate level be measured first. “In patients with serum bicarbonate less than 27 mmol/L, clinicians might forgo measuring PaCO₂, as the diagnosis in them is very unlikely,” Dr. Venkateshiah said, referring to the guideline. “In patients with a serum bicarbonate greater than 27, you might need to measure PaCO₂ to confirm or rule out the diagnosis of OHS.”

(Patients strongly suspected of having OHS, with more than a low to moderate probability – those in whom arterial blood gases should be measured – are “usually severely obese with typical signs and symptoms such as dyspnea, nocturia, lower-extremity edema, excessive daytime sleepiness, fatigue, loud disruptive snoring, witnessed apneas, as well as mild hypoxemia during wake and/or significant hypoxemia during sleep,” the ATS guideline says.)

The guideline panel considered the use of oxygen saturation measured with pulse oximetry during wakefulness to screen for OHS and decided to advise against it because of the paucity of evidence-based literature, Dr. Venkateshiah noted. (In making its five conditional recommendations, the guideline panel cited an overall very low quality of evidence.)

Symptoms of OHS overlap with those of OSA (for example, daytime hypersomnolence, witnessed apneas, loud snoring, and morning headaches), so “symptoms alone cannot be used to discriminate between the two disorders,” he advised. Signs of OHS commonly seen in clinical exams, however, are low resting daytime oxygen saturations and lower-extremity edema. A sleep study, he added, is needed to document and characterize SDB in patients with OHS.

Positive airway pressure therapy is the first-line treatment for OHS, and long-term outcomes of patients with OHS on PAP treatment are significantly better, compared with untreated individuals. There is no strong evidence to recommend one form of PAP therapy over another for patients with OHS and concomitant severe OSA, he said, but “the bottom line” from both short- and long-term randomized clinical trials comparing CPAP with noninvasive ventilation “is that CPAP is equivalent to noninvasive ventilation as far as outcomes are concerned.”

The ATS guideline panel recommends continuous positive airway pressure therapy for patients with OHS and severe OSA. And for OHS with nonsevere OSA, bilevel PAP is traditionally used – including pure hypoventilators, Dr. Venkateshiah said.

Weight-loss interventions are paramount, since “the primary driver of OHS is obesity,” he said at the meeting. There are only a few studies that have looked at bariatric surgery in patients with OHS, he said, “but they did note significant improvements in gas exchange, sleep apnea, lung volumes and pulmonary hypertension.”

The ATS guideline suggests weight-loss interventions that produce sustained weight loss of 25%-30% of the actual body weight. Such interventions are “most likely required to achieve resolution of hypoventilation,” Dr. Venkateshiah said.
 

OHS vs. COPD

In a separate presentation on OHS, Michelle Cao, DO, clinical associate professor at Stanford (Calif.) University, emphasized the importance of distinguishing the patient with OHS from the patient with hypercapnic chronic obstructive pulmonary disease (COPD). Spirometry and the flow volume curve can help rule out hypercapnic COPD and other conditions that cause daytime hypoventilation.

A study published in 2016 of 600 hospitalized patients determined to have unequivocal OHS found that 43% had been misdiagnosed as having COPD and none had been previously diagnosed with OHS, Dr. Cao noted. Patients in the study had a mean age of 58 and a mean body mass index of 48.2 kg/m²; 64% were women.

Dr. Venkateshiah and Dr. Cao had no relevant disclosures.

Obesity hypoventilation syndrome (OHS) is bound to be increasing because of the rising obesity epidemic but is underrecognized and “frequently underdiagnosed,” Saiprakash B. Venkateshiah, MD, said at the virtual annual meeting of the Associated Professional Sleep Societies.

The condition, which can cause significant morbidity and mortality, is defined by the combination of obesity and awake alveolar hypoventilation (PaCO₂ ≥45 mm Hg), with the exclusion of alternate causes of hypoventilation. Sleep-disordered breathing (SDB) is almost universally present, with approximately 90% of individuals with OHS also having obstructive sleep apnea (OSA), most often severe, and approximately 10% having sleep-related hypoventilation, or a “pure hypoventilation subtype, if you will,” said Dr. Venkateshiah, assistant professor of medicine at Emory University, Atlanta.

The prevalence of OHS in the general population is unknown, but its prevalence in patients who present for the evaluation of SDB has ranged from 8%-20% across multiple studies, he said. Up to 40% of patients with OHS present for the first time with acute hypercapnic respiratory failure, which has an in-hospital mortality of 18%.

Postmenopausal women appear to have a higher prevalence, compared with premenopausal women and men, he noted, and women appear to be more likely than men to present with the clinical phenotype of OHS without associated OSA.

The arterial blood gas measurement needed to document alveolar hypoventilation and definitively diagnosis OHA is a “simple and economical test,” he said, “but it is logistically very difficult to obtain [these measurements] routinely in all patients in the clinic ... and is one of the reasons why OSH is underdiagnosed.”
 

Guideline advice

A practice guideline published in 2019 by the American Thoracic Society suggests that, for obese patients with SDB and a low to moderate probability of having OSH, a serum bicarbonate level be measured first. “In patients with serum bicarbonate less than 27 mmol/L, clinicians might forgo measuring PaCO₂, as the diagnosis in them is very unlikely,” Dr. Venkateshiah said, referring to the guideline. “In patients with a serum bicarbonate greater than 27, you might need to measure PaCO₂ to confirm or rule out the diagnosis of OHS.”

(Patients strongly suspected of having OHS, with more than a low to moderate probability – those in whom arterial blood gases should be measured – are “usually severely obese with typical signs and symptoms such as dyspnea, nocturia, lower-extremity edema, excessive daytime sleepiness, fatigue, loud disruptive snoring, witnessed apneas, as well as mild hypoxemia during wake and/or significant hypoxemia during sleep,” the ATS guideline says.)

The guideline panel considered the use of oxygen saturation measured with pulse oximetry during wakefulness to screen for OHS and decided to advise against it because of the paucity of evidence-based literature, Dr. Venkateshiah noted. (In making its five conditional recommendations, the guideline panel cited an overall very low quality of evidence.)

Symptoms of OHS overlap with those of OSA (for example, daytime hypersomnolence, witnessed apneas, loud snoring, and morning headaches), so “symptoms alone cannot be used to discriminate between the two disorders,” he advised. Signs of OHS commonly seen in clinical exams, however, are low resting daytime oxygen saturations and lower-extremity edema. A sleep study, he added, is needed to document and characterize SDB in patients with OHS.

Positive airway pressure therapy is the first-line treatment for OHS, and long-term outcomes of patients with OHS on PAP treatment are significantly better, compared with untreated individuals. There is no strong evidence to recommend one form of PAP therapy over another for patients with OHS and concomitant severe OSA, he said, but “the bottom line” from both short- and long-term randomized clinical trials comparing CPAP with noninvasive ventilation “is that CPAP is equivalent to noninvasive ventilation as far as outcomes are concerned.”

The ATS guideline panel recommends continuous positive airway pressure therapy for patients with OHS and severe OSA. And for OHS with nonsevere OSA, bilevel PAP is traditionally used – including pure hypoventilators, Dr. Venkateshiah said.

Weight-loss interventions are paramount, since “the primary driver of OHS is obesity,” he said at the meeting. There are only a few studies that have looked at bariatric surgery in patients with OHS, he said, “but they did note significant improvements in gas exchange, sleep apnea, lung volumes and pulmonary hypertension.”

The ATS guideline suggests weight-loss interventions that produce sustained weight loss of 25%-30% of the actual body weight. Such interventions are “most likely required to achieve resolution of hypoventilation,” Dr. Venkateshiah said.
 

OHS vs. COPD

In a separate presentation on OHS, Michelle Cao, DO, clinical associate professor at Stanford (Calif.) University, emphasized the importance of distinguishing the patient with OHS from the patient with hypercapnic chronic obstructive pulmonary disease (COPD). Spirometry and the flow volume curve can help rule out hypercapnic COPD and other conditions that cause daytime hypoventilation.

A study published in 2016 of 600 hospitalized patients determined to have unequivocal OHS found that 43% had been misdiagnosed as having COPD and none had been previously diagnosed with OHS, Dr. Cao noted. Patients in the study had a mean age of 58 and a mean body mass index of 48.2 kg/m²; 64% were women.

Dr. Venkateshiah and Dr. Cao had no relevant disclosures.

Inpatient care utilization for hidradenitis suppurativa (HS) was higher for Black and Hispanic patients, compared with White patients, in an analysis of the 2012-2017 National Inpatient Sample.

The differences occurred despite Black and Hispanic patients being younger at the time of admission than White patients, and may reflect increased disease severity and management challenges in these patients with skin of color, Nishadh Sutaria, BS, a medical student at Tufts University, Boston, said at the annual Skin of Color Society symposium. “They may also reflect social inequities in access to dermatologists, with racial and ethnic minorities using inpatient services in lieu of outpatient care.”

Mr. Sutaria and coinvestigators, led by Shawn Kwatra, MD, of Johns Hopkins University, Baltimore, identified 8,040 HS admissions for White patients, 16,490 Black patients, and 2,405 for Hispanic patients during the 5-year period.

Black and Hispanic patients were significantly younger than White patients, with a mean age of 38.1 years and 35 years, respectively, compared with 42 years for White patients (P < .001 in each case). Compared with White patients, Black patients had more procedures (2.03 vs. 1.84, P = .006), a longer length of stay (5.82 days vs. 4.97 days, P = .001), and higher cost of care ($46,119 vs. $39,862, P = .010). Compared with White patients, Hispanic patients had higher cost of care ($52,334 vs. $39,862, P = .004).

“In these models, Black patients stayed almost a full day longer and accrued a charge of $8,000 more than White patients, and Hispanic patients stayed about a half-day longer and accrued a charge of almost $15,000 more than White patients,” Mr. Sutaria said.

In a multilinear regression analysis adjusting for age, sex, and insurance type, Black race correlated with more procedures, higher length of stay, and higher cost of care, and Hispanic ethnicity with more procedures and higher cost of care.

Prior research has shown that Black patients may be disproportionately affected by HS. A 2017 analysis of electronic health record data for tens of millions of patients nationally, for instance, showed an incidence of HS that was over 2.5 times greater in Blacks than Whites. And a recent analysis of electronic data in Wisconsin for patients with an HS diagnosis and 3 or more encounters for the disease showed that Blacks are more likely to have HS that is Hurley Stage 3, the most severe type.

Increased severity “has not been explicitly shown in Hispanic patients,” Dr. Kwatra said in an interview, “[but] there is a strong relationship between obesity/metabolic syndrome with HS. Because Hispanic patients have higher rates of obesity and metabolic syndrome, it’s [thought] that they may have more severe HS.”

HS patients with skin of color are underrepresented in clinical trials, he said. “Severe HS can be difficult to treat because there are few effective treatments,” he said, noting that adalimumab is the only Food and Drug Administration–approved therapy.

The National Inpatient Sample is a publicly available, all-payer inpatient care database developed for the Agency for Healthcare Research and Quality’s Healthcare Cost and Utilization Project.

Mr. Sutaria is a dermatology research fellow working under the guidance of Dr. Kwatra.

Inpatient care utilization for hidradenitis suppurativa (HS) was higher for Black and Hispanic patients, compared with White patients, in an analysis of the 2012-2017 National Inpatient Sample.

The differences occurred despite Black and Hispanic patients being younger at the time of admission than White patients, and may reflect increased disease severity and management challenges in these patients with skin of color, Nishadh Sutaria, BS, a medical student at Tufts University, Boston, said at the annual Skin of Color Society symposium. “They may also reflect social inequities in access to dermatologists, with racial and ethnic minorities using inpatient services in lieu of outpatient care.”

Mr. Sutaria and coinvestigators, led by Shawn Kwatra, MD, of Johns Hopkins University, Baltimore, identified 8,040 HS admissions for White patients, 16,490 Black patients, and 2,405 for Hispanic patients during the 5-year period.

Black and Hispanic patients were significantly younger than White patients, with a mean age of 38.1 years and 35 years, respectively, compared with 42 years for White patients (P < .001 in each case). Compared with White patients, Black patients had more procedures (2.03 vs. 1.84, P = .006), a longer length of stay (5.82 days vs. 4.97 days, P = .001), and higher cost of care ($46,119 vs. $39,862, P = .010). Compared with White patients, Hispanic patients had higher cost of care ($52,334 vs. $39,862, P = .004).

“In these models, Black patients stayed almost a full day longer and accrued a charge of $8,000 more than White patients, and Hispanic patients stayed about a half-day longer and accrued a charge of almost $15,000 more than White patients,” Mr. Sutaria said.

In a multilinear regression analysis adjusting for age, sex, and insurance type, Black race correlated with more procedures, higher length of stay, and higher cost of care, and Hispanic ethnicity with more procedures and higher cost of care.

Prior research has shown that Black patients may be disproportionately affected by HS. A 2017 analysis of electronic health record data for tens of millions of patients nationally, for instance, showed an incidence of HS that was over 2.5 times greater in Blacks than Whites. And a recent analysis of electronic data in Wisconsin for patients with an HS diagnosis and 3 or more encounters for the disease showed that Blacks are more likely to have HS that is Hurley Stage 3, the most severe type.

Increased severity “has not been explicitly shown in Hispanic patients,” Dr. Kwatra said in an interview, “[but] there is a strong relationship between obesity/metabolic syndrome with HS. Because Hispanic patients have higher rates of obesity and metabolic syndrome, it’s [thought] that they may have more severe HS.”

HS patients with skin of color are underrepresented in clinical trials, he said. “Severe HS can be difficult to treat because there are few effective treatments,” he said, noting that adalimumab is the only Food and Drug Administration–approved therapy.

The National Inpatient Sample is a publicly available, all-payer inpatient care database developed for the Agency for Healthcare Research and Quality’s Healthcare Cost and Utilization Project.

Mr. Sutaria is a dermatology research fellow working under the guidance of Dr. Kwatra.

Inpatient care utilization for hidradenitis suppurativa (HS) was higher for Black and Hispanic patients, compared with White patients, in an analysis of the 2012-2017 National Inpatient Sample.

The differences occurred despite Black and Hispanic patients being younger at the time of admission than White patients, and may reflect increased disease severity and management challenges in these patients with skin of color, Nishadh Sutaria, BS, a medical student at Tufts University, Boston, said at the annual Skin of Color Society symposium. “They may also reflect social inequities in access to dermatologists, with racial and ethnic minorities using inpatient services in lieu of outpatient care.”

Mr. Sutaria and coinvestigators, led by Shawn Kwatra, MD, of Johns Hopkins University, Baltimore, identified 8,040 HS admissions for White patients, 16,490 Black patients, and 2,405 for Hispanic patients during the 5-year period.

Black and Hispanic patients were significantly younger than White patients, with a mean age of 38.1 years and 35 years, respectively, compared with 42 years for White patients (P < .001 in each case). Compared with White patients, Black patients had more procedures (2.03 vs. 1.84, P = .006), a longer length of stay (5.82 days vs. 4.97 days, P = .001), and higher cost of care ($46,119 vs. $39,862, P = .010). Compared with White patients, Hispanic patients had higher cost of care ($52,334 vs. $39,862, P = .004).

“In these models, Black patients stayed almost a full day longer and accrued a charge of $8,000 more than White patients, and Hispanic patients stayed about a half-day longer and accrued a charge of almost $15,000 more than White patients,” Mr. Sutaria said.

In a multilinear regression analysis adjusting for age, sex, and insurance type, Black race correlated with more procedures, higher length of stay, and higher cost of care, and Hispanic ethnicity with more procedures and higher cost of care.

Prior research has shown that Black patients may be disproportionately affected by HS. A 2017 analysis of electronic health record data for tens of millions of patients nationally, for instance, showed an incidence of HS that was over 2.5 times greater in Blacks than Whites. And a recent analysis of electronic data in Wisconsin for patients with an HS diagnosis and 3 or more encounters for the disease showed that Blacks are more likely to have HS that is Hurley Stage 3, the most severe type.

Increased severity “has not been explicitly shown in Hispanic patients,” Dr. Kwatra said in an interview, “[but] there is a strong relationship between obesity/metabolic syndrome with HS. Because Hispanic patients have higher rates of obesity and metabolic syndrome, it’s [thought] that they may have more severe HS.”

HS patients with skin of color are underrepresented in clinical trials, he said. “Severe HS can be difficult to treat because there are few effective treatments,” he said, noting that adalimumab is the only Food and Drug Administration–approved therapy.

The National Inpatient Sample is a publicly available, all-payer inpatient care database developed for the Agency for Healthcare Research and Quality’s Healthcare Cost and Utilization Project.

Mr. Sutaria is a dermatology research fellow working under the guidance of Dr. Kwatra.

A new physician workforce study documents that almost all clinically active pediatric emergency physicians in the United States – 99% of them – work in urban areas, and that those who do practice in rural areas are significantly older and closer to retirement age.

The portrait of approximately 2,400 self-identified pediatric emergency medicine (EM) physicians may be unsurprising given the overall propensity of physicians – including board-certified general emergency physicians – to practice in urban areas. Even so, it underscores a decades-long concern that many children do not have access to optimal pediatric emergency care.

And the findings highlight the need, the authors say, to keep pressing to improve emergency care for a population of children with “a mortality rate that is already higher than that of its suburban and urban peers (JAMA Network Open 2021;4[5]:e2110084).”

Emergent care of pediatric patients is well within the scope of practice for physicians with training and board certification in general EM, but children and adolescents have different clinical needs and “there are high-stakes scenarios [in children] that we [as emergency physicians] don’t get exposed to as often because we’re not in a children’s hospital or we just don’t have that additional level of training,” said Christopher L. Bennett, MD, MA, of the department of emergency medicine at Stanford University and lead author of the study.

Researchers have documented that some emergency physicians have some discomfort in caring for very ill pediatric patients, he and his coauthors wrote.

Children account for more than 20% of annual ED visits, and most children who seek emergency care in the United States – upwards of 80% – present to general emergency departments. Yet the vast majority of these EDs care for fewer than 14-15 children a day.

With such low pediatric volume, “there will never be pediatric emergency medicine physicians in the rural hospitals in [our] health care system,” said Kathleen M. Brown, MD, medical director for quality and safety of the Emergency Medicine and Trauma Center at Children’s National Medical Center in Washington.

Redistribution “is not a practical solution, and we’ve known that for a long time,” said Dr. Brown, past chairperson of the American College of Emergency Physicians’ pediatric emergency medicine committee. “That’s why national efforts have focused on better preparing the general emergency department and making sure the hospital workforce is ready to take care of children ... to manage and stabilize [them] and recognize when they need more definitive care.”

Continuing efforts to increase “pediatric readiness” in general EDs is one of the recommendations issued by the American Academy of Pediatrics, ACEP, and Emergency Nurses Association in its most recent joint policy statement on emergency care for children, published in May (Pediatrics 2021;147[5]:e2021050787). A 2018 joint policy statement detailed the resources – medications, equipment, policies, and education – necessary for EDs to provide effective pediatric care (Pediatrics 2018;142[5]:e20182459).

There is some evidence that pediatric readiness has improved and that EDs with higher readiness scores may have better pediatric outcomes and lower mortality, said Dr. Brown, a coauthor of both policy statements. (One study cited in the 2018 policy statement, for example, found that children with extremity immobilization and a pain score of 5 or greater had faster management of their pain and decreased exposure to radiation when they were treated in a better-prepared facility than in a facility with less readiness.)

Yet many hospitals still do not have designated pediatric emergency care coordinators (PECCs) – roles that are widely believed to be central to pediatric readiness. PECCs (physicians and nurses) were recommended in 2006 by the then-Institute of Medicine and have been advocated by the AAP, ACEP, and other organizations.

According to 2013 data from the National Pediatric Readiness Project (NPRP), launched that year by the AAP, ACEP, ENA, and the federal Emergency Medical Services for Children program of the Health Resources and Services Administration, at least 15% of EDs lacked at least 1 piece of recommended equipment, and 81% reported barriers to pediatric emergency care guidelines. The NPRP is currently conducting an updated assessment, Dr. Brown said.

Some experts have proposed a different kind of solution – one in which American Board of Pediatrics–certified pediatric EM physicians would care for selective adult patients with common disease patterns who present to rural EDs, in addition to children. They might provide direct patient care across several hospitals in a region, while also addressing quality improvement and assisting EPs and other providers in the region on pediatric care issues.

The proposal, published in May 2020, comes from the 13-member special subcommittee of the ACEP committee on PEM that was tasked with exploring strategies to improve access to emergency pediatric expertise and disaster preparedness in all settings. The proposal was endorsed by the ACEP board of directors, said Jim Homme, MD, a coauthor of the paper (JACEP Open 2020;1:1520-6.)

“We’re saying, look at the ped-trained pediatric emergency provider more broadly. They can actually successfully care for a broader patient population and make it financially feasible ... [for that physician] to be a part of the system,” said Dr. Homme, program director of the emergency medicine residency at the Mayo Clinic College of Medicine and Science in Rochester, Minn.

“The benefit would be not only having the expertise to see children, but to train up other individuals in the institution, and be advocates for the care of children,” he said.

“We’re not saying we want a pediatrics-trained EM physician in every site so that every child would be seen by one – that’s not the goal,” Dr. Homme said. “The goal is to distribute them more broadly than they currently are, and in doing so, make available the other benefits besides direct patient care.”

Most of the physicians in the United States who identify as pediatric EM physicians have completed either a pediatrics or EM residency, followed by a pediatric EM fellowship. It is much more common to have primary training in pediatrics than in EM, said Dr. Homme and Dr. Bennett. A small number of physicians, like Dr. Homme, are dually trained in pediatrics and EM through the completion of two residencies. Dr. Bennett’s workforce study used the American Medical Association Physician Masterfile database and identified 2,403 clinically active pediatric EPs – 5% of all clinically active emergency physicians. Those practicing in rural areas had a median age of 59, compared with a median age of 46 in urban areas. More than half of the pediatric EPs – 68% – reported having pediatric EM board certification.

Three states – Montana, South Dakota, and Wyoming – had no pediatric EMs at all, Dr. Bennett noted.

Readiness in rural Oregon, New England

Torree McGowan, MD, an emergency physician with the St. Charles Health System in Oregon, works in small critical access hospitals in the rural towns of Madras and Prineville, each several hours by ground to the nearest pediatric hospital. She said she feels well equipped to care for children through her training (a rotation in a pediatric ICU and several months working in pediatric EDs) and through her ongoing work with pediatric patients. Children and adolescents comprise about 20%-30% of her volume.

She sees more pediatric illness – children with respiratory syncytial virus who need respiratory support, for instance, and children with severe asthma or diabetic ketoacidosis – than pediatric trauma. When she faces questions, uncertainties, or wants confirmation of a decision, she consults by phone with pediatric subspecialists.

“I don’t take care of kids on vasopressor drips on a regular basis [for instance],” said Dr. McGowan, who sits on ACEP’s disaster preparedness committee and is an Air Force veteran. “But I know the basics and can phone a colleague to be sure I’m doing it correctly. The ability to outreach is there.”

Telemedicine is valuable, she said, but there may also be value in working alongside a pediatric EM physician. One of her EP colleagues is fellowship-trained in ultrasonography and “leads us in training and quality control,” Dr. McGowan said. “And if she’s on shift with you she’ll teach you all about ultrasound. There’s probably utility in having a pediatric EP who does that as well. But incentivizing that and taking them away from the pediatric hospital is a paradigm shift.”

Either way, she said, “being able to bring that expertise out of urban centers, whether it’s to a hospital group like ours or whether it’s by telemedicine, is really, really helpful.”

Her group does not have official PECCs, but the joint policy statements by AAP/ACEP/ENA on pediatric readiness and the “whole pediatric readiness effort’ have been valuable in “driving conversations” with administrators about needs such as purchasing pediatric-sized video laryngoscope blades and other equipment needed for pediatric emergencies, however infrequently they may occur, Dr. McGowan said.

In New England, researchers leading a grassroots regional intervention to establish a PECC in every ED in the region have reported an increased prevalence of “pediatric champions” from less than 30% 5 years ago to greater than 90% in 2019, investigators have reported (Pediatr Emerg Care. 2021. doi: 10.1097/PEC.0000000000002456).

The initiative involved individual outreach to leaders of each ED – largely through phone and e-mail appeals – and collaboration among the State Emergency Medical Services for Children agencies and ACEP and ENA state chapters. The researchers are currently investigating the direct impact of PECCs on patient outcomes.

More on regionalization of ped-trained EPs

Dr. Bennett sees telemedicine as a primary part of improving pediatric emergency care. “I think that’s where things are going to go in pediatric emergency medicine,” he said, especially in the wake of COVID-19: “I don’t see how it’s not going to become much more common.”

Dr. Homme maintains that a broader integration of ABP-certified pediatric EM physicians into underserved regions would advance ED preparedness in a way that telemedicine, or even the appointment of PECCs, does not, said Dr. Homme.

Institutions would need to acknowledge that many of the current restrictions on pediatric EM physicians’ scope of practice are based on arbitrary age cut-offs, and their leaders would need to expand hospital-defined privileges to better align with training and capabilities, he said. Local credentialing provisions and other policies would also need to be adjusted.

Pediatric EM physicians spend at least 4 months of their graduate EM training in an adult ED, and there is significant overlap in the core competencies and the procedures considered essential for practice between pediatric EM fellowship programs and EM programs, Dr. Homme and his coauthors wrote in their proposal. “The pandemic really reinforced this concept,” Dr. Homme said. “As the number of patients in pediatric EDs plummeted, many of the ped-trained providers had to pivot and help care for adults. ... It worked great.”

The broader integration of pediatrics-trained pediatric EM physicians fits well, he believes, with current workforce dynamics. “There aren’t enough individuals coming out of an EM background and doing that subspecialty training to have any hope that they’d be able to cover these underserved areas,” he said. “And the academic pediatric workforce is getting kind of saturated. So having additional employment opportunities would be great.”

Dr. Homme pursued an EM residency after pediatrics training (rather than a pediatric EM fellowship) because he did not want to be limited geographically and because, while he wanted to focus on children, he also “wanted to be available to a larger population.”

He believes that some pediatrics-trained pediatric EM physicians would choose rural practice options, and hopes that the proposal will gain traction. Some EPs will be opposed, he said, and some pediatrics-trained EPs will not interested, “but if we can find people open to the idea on both sides, I think we can really move the needle in the direction we’re trying to, which is to disseminate an area of expertise into areas that just don’t have it.”

A new physician workforce study documents that almost all clinically active pediatric emergency physicians in the United States – 99% of them – work in urban areas, and that those who do practice in rural areas are significantly older and closer to retirement age.

The portrait of approximately 2,400 self-identified pediatric emergency medicine (EM) physicians may be unsurprising given the overall propensity of physicians – including board-certified general emergency physicians – to practice in urban areas. Even so, it underscores a decades-long concern that many children do not have access to optimal pediatric emergency care.

And the findings highlight the need, the authors say, to keep pressing to improve emergency care for a population of children with “a mortality rate that is already higher than that of its suburban and urban peers (JAMA Network Open 2021;4[5]:e2110084).”

Emergent care of pediatric patients is well within the scope of practice for physicians with training and board certification in general EM, but children and adolescents have different clinical needs and “there are high-stakes scenarios [in children] that we [as emergency physicians] don’t get exposed to as often because we’re not in a children’s hospital or we just don’t have that additional level of training,” said Christopher L. Bennett, MD, MA, of the department of emergency medicine at Stanford University and lead author of the study.

Researchers have documented that some emergency physicians have some discomfort in caring for very ill pediatric patients, he and his coauthors wrote.

Children account for more than 20% of annual ED visits, and most children who seek emergency care in the United States – upwards of 80% – present to general emergency departments. Yet the vast majority of these EDs care for fewer than 14-15 children a day.

With such low pediatric volume, “there will never be pediatric emergency medicine physicians in the rural hospitals in [our] health care system,” said Kathleen M. Brown, MD, medical director for quality and safety of the Emergency Medicine and Trauma Center at Children’s National Medical Center in Washington.

Redistribution “is not a practical solution, and we’ve known that for a long time,” said Dr. Brown, past chairperson of the American College of Emergency Physicians’ pediatric emergency medicine committee. “That’s why national efforts have focused on better preparing the general emergency department and making sure the hospital workforce is ready to take care of children ... to manage and stabilize [them] and recognize when they need more definitive care.”

Continuing efforts to increase “pediatric readiness” in general EDs is one of the recommendations issued by the American Academy of Pediatrics, ACEP, and Emergency Nurses Association in its most recent joint policy statement on emergency care for children, published in May (Pediatrics 2021;147[5]:e2021050787). A 2018 joint policy statement detailed the resources – medications, equipment, policies, and education – necessary for EDs to provide effective pediatric care (Pediatrics 2018;142[5]:e20182459).

There is some evidence that pediatric readiness has improved and that EDs with higher readiness scores may have better pediatric outcomes and lower mortality, said Dr. Brown, a coauthor of both policy statements. (One study cited in the 2018 policy statement, for example, found that children with extremity immobilization and a pain score of 5 or greater had faster management of their pain and decreased exposure to radiation when they were treated in a better-prepared facility than in a facility with less readiness.)

Yet many hospitals still do not have designated pediatric emergency care coordinators (PECCs) – roles that are widely believed to be central to pediatric readiness. PECCs (physicians and nurses) were recommended in 2006 by the then-Institute of Medicine and have been advocated by the AAP, ACEP, and other organizations.

According to 2013 data from the National Pediatric Readiness Project (NPRP), launched that year by the AAP, ACEP, ENA, and the federal Emergency Medical Services for Children program of the Health Resources and Services Administration, at least 15% of EDs lacked at least 1 piece of recommended equipment, and 81% reported barriers to pediatric emergency care guidelines. The NPRP is currently conducting an updated assessment, Dr. Brown said.

Some experts have proposed a different kind of solution – one in which American Board of Pediatrics–certified pediatric EM physicians would care for selective adult patients with common disease patterns who present to rural EDs, in addition to children. They might provide direct patient care across several hospitals in a region, while also addressing quality improvement and assisting EPs and other providers in the region on pediatric care issues.

The proposal, published in May 2020, comes from the 13-member special subcommittee of the ACEP committee on PEM that was tasked with exploring strategies to improve access to emergency pediatric expertise and disaster preparedness in all settings. The proposal was endorsed by the ACEP board of directors, said Jim Homme, MD, a coauthor of the paper (JACEP Open 2020;1:1520-6.)

“We’re saying, look at the ped-trained pediatric emergency provider more broadly. They can actually successfully care for a broader patient population and make it financially feasible ... [for that physician] to be a part of the system,” said Dr. Homme, program director of the emergency medicine residency at the Mayo Clinic College of Medicine and Science in Rochester, Minn.

“The benefit would be not only having the expertise to see children, but to train up other individuals in the institution, and be advocates for the care of children,” he said.

“We’re not saying we want a pediatrics-trained EM physician in every site so that every child would be seen by one – that’s not the goal,” Dr. Homme said. “The goal is to distribute them more broadly than they currently are, and in doing so, make available the other benefits besides direct patient care.”

Most of the physicians in the United States who identify as pediatric EM physicians have completed either a pediatrics or EM residency, followed by a pediatric EM fellowship. It is much more common to have primary training in pediatrics than in EM, said Dr. Homme and Dr. Bennett. A small number of physicians, like Dr. Homme, are dually trained in pediatrics and EM through the completion of two residencies. Dr. Bennett’s workforce study used the American Medical Association Physician Masterfile database and identified 2,403 clinically active pediatric EPs – 5% of all clinically active emergency physicians. Those practicing in rural areas had a median age of 59, compared with a median age of 46 in urban areas. More than half of the pediatric EPs – 68% – reported having pediatric EM board certification.

Three states – Montana, South Dakota, and Wyoming – had no pediatric EMs at all, Dr. Bennett noted.

Readiness in rural Oregon, New England

Torree McGowan, MD, an emergency physician with the St. Charles Health System in Oregon, works in small critical access hospitals in the rural towns of Madras and Prineville, each several hours by ground to the nearest pediatric hospital. She said she feels well equipped to care for children through her training (a rotation in a pediatric ICU and several months working in pediatric EDs) and through her ongoing work with pediatric patients. Children and adolescents comprise about 20%-30% of her volume.

She sees more pediatric illness – children with respiratory syncytial virus who need respiratory support, for instance, and children with severe asthma or diabetic ketoacidosis – than pediatric trauma. When she faces questions, uncertainties, or wants confirmation of a decision, she consults by phone with pediatric subspecialists.

“I don’t take care of kids on vasopressor drips on a regular basis [for instance],” said Dr. McGowan, who sits on ACEP’s disaster preparedness committee and is an Air Force veteran. “But I know the basics and can phone a colleague to be sure I’m doing it correctly. The ability to outreach is there.”

Telemedicine is valuable, she said, but there may also be value in working alongside a pediatric EM physician. One of her EP colleagues is fellowship-trained in ultrasonography and “leads us in training and quality control,” Dr. McGowan said. “And if she’s on shift with you she’ll teach you all about ultrasound. There’s probably utility in having a pediatric EP who does that as well. But incentivizing that and taking them away from the pediatric hospital is a paradigm shift.”

Either way, she said, “being able to bring that expertise out of urban centers, whether it’s to a hospital group like ours or whether it’s by telemedicine, is really, really helpful.”

Her group does not have official PECCs, but the joint policy statements by AAP/ACEP/ENA on pediatric readiness and the “whole pediatric readiness effort’ have been valuable in “driving conversations” with administrators about needs such as purchasing pediatric-sized video laryngoscope blades and other equipment needed for pediatric emergencies, however infrequently they may occur, Dr. McGowan said.

In New England, researchers leading a grassroots regional intervention to establish a PECC in every ED in the region have reported an increased prevalence of “pediatric champions” from less than 30% 5 years ago to greater than 90% in 2019, investigators have reported (Pediatr Emerg Care. 2021. doi: 10.1097/PEC.0000000000002456).

The initiative involved individual outreach to leaders of each ED – largely through phone and e-mail appeals – and collaboration among the State Emergency Medical Services for Children agencies and ACEP and ENA state chapters. The researchers are currently investigating the direct impact of PECCs on patient outcomes.

More on regionalization of ped-trained EPs

Dr. Bennett sees telemedicine as a primary part of improving pediatric emergency care. “I think that’s where things are going to go in pediatric emergency medicine,” he said, especially in the wake of COVID-19: “I don’t see how it’s not going to become much more common.”

Dr. Homme maintains that a broader integration of ABP-certified pediatric EM physicians into underserved regions would advance ED preparedness in a way that telemedicine, or even the appointment of PECCs, does not, said Dr. Homme.

Institutions would need to acknowledge that many of the current restrictions on pediatric EM physicians’ scope of practice are based on arbitrary age cut-offs, and their leaders would need to expand hospital-defined privileges to better align with training and capabilities, he said. Local credentialing provisions and other policies would also need to be adjusted.

Pediatric EM physicians spend at least 4 months of their graduate EM training in an adult ED, and there is significant overlap in the core competencies and the procedures considered essential for practice between pediatric EM fellowship programs and EM programs, Dr. Homme and his coauthors wrote in their proposal. “The pandemic really reinforced this concept,” Dr. Homme said. “As the number of patients in pediatric EDs plummeted, many of the ped-trained providers had to pivot and help care for adults. ... It worked great.”

The broader integration of pediatrics-trained pediatric EM physicians fits well, he believes, with current workforce dynamics. “There aren’t enough individuals coming out of an EM background and doing that subspecialty training to have any hope that they’d be able to cover these underserved areas,” he said. “And the academic pediatric workforce is getting kind of saturated. So having additional employment opportunities would be great.”

Dr. Homme pursued an EM residency after pediatrics training (rather than a pediatric EM fellowship) because he did not want to be limited geographically and because, while he wanted to focus on children, he also “wanted to be available to a larger population.”

He believes that some pediatrics-trained pediatric EM physicians would choose rural practice options, and hopes that the proposal will gain traction. Some EPs will be opposed, he said, and some pediatrics-trained EPs will not interested, “but if we can find people open to the idea on both sides, I think we can really move the needle in the direction we’re trying to, which is to disseminate an area of expertise into areas that just don’t have it.”

A new physician workforce study documents that almost all clinically active pediatric emergency physicians in the United States – 99% of them – work in urban areas, and that those who do practice in rural areas are significantly older and closer to retirement age.

The portrait of approximately 2,400 self-identified pediatric emergency medicine (EM) physicians may be unsurprising given the overall propensity of physicians – including board-certified general emergency physicians – to practice in urban areas. Even so, it underscores a decades-long concern that many children do not have access to optimal pediatric emergency care.

And the findings highlight the need, the authors say, to keep pressing to improve emergency care for a population of children with “a mortality rate that is already higher than that of its suburban and urban peers (JAMA Network Open 2021;4[5]:e2110084).”

Emergent care of pediatric patients is well within the scope of practice for physicians with training and board certification in general EM, but children and adolescents have different clinical needs and “there are high-stakes scenarios [in children] that we [as emergency physicians] don’t get exposed to as often because we’re not in a children’s hospital or we just don’t have that additional level of training,” said Christopher L. Bennett, MD, MA, of the department of emergency medicine at Stanford University and lead author of the study.

Researchers have documented that some emergency physicians have some discomfort in caring for very ill pediatric patients, he and his coauthors wrote.

Children account for more than 20% of annual ED visits, and most children who seek emergency care in the United States – upwards of 80% – present to general emergency departments. Yet the vast majority of these EDs care for fewer than 14-15 children a day.

With such low pediatric volume, “there will never be pediatric emergency medicine physicians in the rural hospitals in [our] health care system,” said Kathleen M. Brown, MD, medical director for quality and safety of the Emergency Medicine and Trauma Center at Children’s National Medical Center in Washington.

Redistribution “is not a practical solution, and we’ve known that for a long time,” said Dr. Brown, past chairperson of the American College of Emergency Physicians’ pediatric emergency medicine committee. “That’s why national efforts have focused on better preparing the general emergency department and making sure the hospital workforce is ready to take care of children ... to manage and stabilize [them] and recognize when they need more definitive care.”

Continuing efforts to increase “pediatric readiness” in general EDs is one of the recommendations issued by the American Academy of Pediatrics, ACEP, and Emergency Nurses Association in its most recent joint policy statement on emergency care for children, published in May (Pediatrics 2021;147[5]:e2021050787). A 2018 joint policy statement detailed the resources – medications, equipment, policies, and education – necessary for EDs to provide effective pediatric care (Pediatrics 2018;142[5]:e20182459).

There is some evidence that pediatric readiness has improved and that EDs with higher readiness scores may have better pediatric outcomes and lower mortality, said Dr. Brown, a coauthor of both policy statements. (One study cited in the 2018 policy statement, for example, found that children with extremity immobilization and a pain score of 5 or greater had faster management of their pain and decreased exposure to radiation when they were treated in a better-prepared facility than in a facility with less readiness.)

Yet many hospitals still do not have designated pediatric emergency care coordinators (PECCs) – roles that are widely believed to be central to pediatric readiness. PECCs (physicians and nurses) were recommended in 2006 by the then-Institute of Medicine and have been advocated by the AAP, ACEP, and other organizations.

According to 2013 data from the National Pediatric Readiness Project (NPRP), launched that year by the AAP, ACEP, ENA, and the federal Emergency Medical Services for Children program of the Health Resources and Services Administration, at least 15% of EDs lacked at least 1 piece of recommended equipment, and 81% reported barriers to pediatric emergency care guidelines. The NPRP is currently conducting an updated assessment, Dr. Brown said.

Some experts have proposed a different kind of solution – one in which American Board of Pediatrics–certified pediatric EM physicians would care for selective adult patients with common disease patterns who present to rural EDs, in addition to children. They might provide direct patient care across several hospitals in a region, while also addressing quality improvement and assisting EPs and other providers in the region on pediatric care issues.

The proposal, published in May 2020, comes from the 13-member special subcommittee of the ACEP committee on PEM that was tasked with exploring strategies to improve access to emergency pediatric expertise and disaster preparedness in all settings. The proposal was endorsed by the ACEP board of directors, said Jim Homme, MD, a coauthor of the paper (JACEP Open 2020;1:1520-6.)

“We’re saying, look at the ped-trained pediatric emergency provider more broadly. They can actually successfully care for a broader patient population and make it financially feasible ... [for that physician] to be a part of the system,” said Dr. Homme, program director of the emergency medicine residency at the Mayo Clinic College of Medicine and Science in Rochester, Minn.

“The benefit would be not only having the expertise to see children, but to train up other individuals in the institution, and be advocates for the care of children,” he said.

“We’re not saying we want a pediatrics-trained EM physician in every site so that every child would be seen by one – that’s not the goal,” Dr. Homme said. “The goal is to distribute them more broadly than they currently are, and in doing so, make available the other benefits besides direct patient care.”

Most of the physicians in the United States who identify as pediatric EM physicians have completed either a pediatrics or EM residency, followed by a pediatric EM fellowship. It is much more common to have primary training in pediatrics than in EM, said Dr. Homme and Dr. Bennett. A small number of physicians, like Dr. Homme, are dually trained in pediatrics and EM through the completion of two residencies. Dr. Bennett’s workforce study used the American Medical Association Physician Masterfile database and identified 2,403 clinically active pediatric EPs – 5% of all clinically active emergency physicians. Those practicing in rural areas had a median age of 59, compared with a median age of 46 in urban areas. More than half of the pediatric EPs – 68% – reported having pediatric EM board certification.

Three states – Montana, South Dakota, and Wyoming – had no pediatric EMs at all, Dr. Bennett noted.

Readiness in rural Oregon, New England

Torree McGowan, MD, an emergency physician with the St. Charles Health System in Oregon, works in small critical access hospitals in the rural towns of Madras and Prineville, each several hours by ground to the nearest pediatric hospital. She said she feels well equipped to care for children through her training (a rotation in a pediatric ICU and several months working in pediatric EDs) and through her ongoing work with pediatric patients. Children and adolescents comprise about 20%-30% of her volume.

She sees more pediatric illness – children with respiratory syncytial virus who need respiratory support, for instance, and children with severe asthma or diabetic ketoacidosis – than pediatric trauma. When she faces questions, uncertainties, or wants confirmation of a decision, she consults by phone with pediatric subspecialists.

“I don’t take care of kids on vasopressor drips on a regular basis [for instance],” said Dr. McGowan, who sits on ACEP’s disaster preparedness committee and is an Air Force veteran. “But I know the basics and can phone a colleague to be sure I’m doing it correctly. The ability to outreach is there.”

Telemedicine is valuable, she said, but there may also be value in working alongside a pediatric EM physician. One of her EP colleagues is fellowship-trained in ultrasonography and “leads us in training and quality control,” Dr. McGowan said. “And if she’s on shift with you she’ll teach you all about ultrasound. There’s probably utility in having a pediatric EP who does that as well. But incentivizing that and taking them away from the pediatric hospital is a paradigm shift.”

Either way, she said, “being able to bring that expertise out of urban centers, whether it’s to a hospital group like ours or whether it’s by telemedicine, is really, really helpful.”

Her group does not have official PECCs, but the joint policy statements by AAP/ACEP/ENA on pediatric readiness and the “whole pediatric readiness effort’ have been valuable in “driving conversations” with administrators about needs such as purchasing pediatric-sized video laryngoscope blades and other equipment needed for pediatric emergencies, however infrequently they may occur, Dr. McGowan said.

In New England, researchers leading a grassroots regional intervention to establish a PECC in every ED in the region have reported an increased prevalence of “pediatric champions” from less than 30% 5 years ago to greater than 90% in 2019, investigators have reported (Pediatr Emerg Care. 2021. doi: 10.1097/PEC.0000000000002456).

The initiative involved individual outreach to leaders of each ED – largely through phone and e-mail appeals – and collaboration among the State Emergency Medical Services for Children agencies and ACEP and ENA state chapters. The researchers are currently investigating the direct impact of PECCs on patient outcomes.

More on regionalization of ped-trained EPs

Dr. Bennett sees telemedicine as a primary part of improving pediatric emergency care. “I think that’s where things are going to go in pediatric emergency medicine,” he said, especially in the wake of COVID-19: “I don’t see how it’s not going to become much more common.”

Dr. Homme maintains that a broader integration of ABP-certified pediatric EM physicians into underserved regions would advance ED preparedness in a way that telemedicine, or even the appointment of PECCs, does not, said Dr. Homme.

Institutions would need to acknowledge that many of the current restrictions on pediatric EM physicians’ scope of practice are based on arbitrary age cut-offs, and their leaders would need to expand hospital-defined privileges to better align with training and capabilities, he said. Local credentialing provisions and other policies would also need to be adjusted.

Pediatric EM physicians spend at least 4 months of their graduate EM training in an adult ED, and there is significant overlap in the core competencies and the procedures considered essential for practice between pediatric EM fellowship programs and EM programs, Dr. Homme and his coauthors wrote in their proposal. “The pandemic really reinforced this concept,” Dr. Homme said. “As the number of patients in pediatric EDs plummeted, many of the ped-trained providers had to pivot and help care for adults. ... It worked great.”

The broader integration of pediatrics-trained pediatric EM physicians fits well, he believes, with current workforce dynamics. “There aren’t enough individuals coming out of an EM background and doing that subspecialty training to have any hope that they’d be able to cover these underserved areas,” he said. “And the academic pediatric workforce is getting kind of saturated. So having additional employment opportunities would be great.”

Dr. Homme pursued an EM residency after pediatrics training (rather than a pediatric EM fellowship) because he did not want to be limited geographically and because, while he wanted to focus on children, he also “wanted to be available to a larger population.”

He believes that some pediatrics-trained pediatric EM physicians would choose rural practice options, and hopes that the proposal will gain traction. Some EPs will be opposed, he said, and some pediatrics-trained EPs will not interested, “but if we can find people open to the idea on both sides, I think we can really move the needle in the direction we’re trying to, which is to disseminate an area of expertise into areas that just don’t have it.”

G. Gayle Stephens, MD, who is roundly regarded as one of the founders of family medicine, gave his talk “Family Medicine as Counterculture” at the Society of Teachers of Family Medicine annual conference in 1979, 10 years after the specialty’s establishment.

The speech was then published, republished 10 years later, and, like many of Dr. Stephen’s other essays and articles, remains very much alive in the minds of practicing family physicians, in the teachings of FP academicians, and in the Google searches of budding FPs.

The late Dr. Stephens saw family medicine as a counterculture within medicine, rooted in social change. In his speech he examined these roots – in reform initiatives in the 1960s, and in certain philosophies and “minority” movements such as agrarianism and the preservation of rural life, utopianism, humanism, consumerism, and feminism.

He also looked forward, challenging the specialty to remain true to itself and its roots – to its belief in “uninhibited access” to medical care for everyone, for instance, and to continual whole-person and family-oriented care – and cautioned against moving to resemble the “rest of the medical bureaucracy.”

“Clearly we have been on the side of change in American life. We have identified ourselves with certain minorities and minority positions ... [and] been counter to many of the dominant forces in society,” Dr. Stephens said in his talk. Family practice “succeeded in the decade just past because we were identified with reforms that are more pervasive and powerful than ourselves.”

The family practice movement has “more in common with [the] counterculture than it does with the dominant scientific medical establishment,” he said.
 

A teacher and founder of medical education programs

Larry A. Green, MD, who was pursuing his own residency training as Dr. Stephens was leading a department of family practice, said Dr. Stephens “insisted that family medicine adhere to the notion that medicine is a moral vocation.”

“It was from this philosophical position that he became a synthesizer and observer and interpreter of what was going on in the development of family medicine,” said Dr. Green, Distinguished Professor and Epperson Zorn Chair for Innovation in Family Medicine and Primary Care at the University of Colorado at Denver, Aurora.

Dr. Stephens, who died at home in 2014 at the age of 85, was “probably the most important person in exposing what I now consider to be a fact – that family medicine was the product of social changes ... of social movements related to women’s rights, civil rights, and social responsibility,” Dr. Green said. “He could recall lessons from the past and forecast the challenges of the future. And there was no one more effective in clarifying the importance of personal [doctor-patient] relationships in family medicine.”

After years of general practice in rural Wichita, Kan., his wife Eula Jean’s hometown, Dr. Stephens founded and led one of the first family medicine residencies at Wesley Hospital in Wichita in 1967. His core principles, as described on today’s Wesley Family Medicine Residency website, included that a family physician consider the whole person, be honest, have a full scope of training including behavioral and mental health, and be “reflective about him/herself ... [learning about] his/her assets, liabilities, foibles, and idiosyncrasies.” Dr. Stephens, who had grown up in rural Ashburn, Mo., later became the founding dean of the School of Primary Medical Care at the University of Alabama in Huntsville and then chaired the department of family practice at the University of Alabama at Birmingham.
 

A thought leader for family medicine

He held numerous state and national leadership positions, and initiated what became the Keystone Conference Series – an invitational gathering of leaders in family medicine that examined and discuss the specialty’s ongoing development. In 2006, he was elected to the Institute of Medicine of the National Academies of Science.

Dr. Stephens authored a textbook, The Intellectual Basis of Family Medicine (Tucson, Ariz.: Winter Publishing Company, 1982), and authored essays, which Dr. Green said will stand the test of time.

“Some of us refer to him as the poet laureate of family medicine,” Dr. Green noted.

In a 1974 article on clinical wisdom, Dr. Stephens wrote that “it is not enough to determine what condition the patient has, but also what patient has the condition.” In another of these essays, which was published in 1979, Dr. Stephens wrote that “physicians need to keep in touch with their own tradition and with public welfare if they are to be considered moral by the society that sponsors them, and from which they take their strength and privilege.”

These excerpts are featured in an article by John P. Geyman, MD, published in 2011 in Family Medicine, called “G. Gayle Stephens Festschrift”.
 

A ‘progressive force’

Linda Prine, MD, professor of family and community medicine at the Icahn School of Medicine at Mount Sinai, New York, knows of Dr. Stephens from her teachers. “The people I looked up to when I was a younger physician were quoting his Counterculture article,” she said.

“It’s not that I studied him. But whenever I heard someone speak about the values of family medicine, his name would come up [and] the values of universal health care and community care and putting the patients’ interests first ahead of the insurance companies and being a doctor for the whole family,” Dr. Prine said. Dr. Stephens was a “progressive force that our specialty has not always lived up to.”

Dr. Stephens voiced serious concerns about the impact of managed care in the 1980s and of “gatekeeping,” a practice intended to control access to specialists and reduce costs.

“He was many times not welcomed by family medicine [for his warnings] against the temptations that managed care presented,” said Dr. Green, the founding director of the Robert Graham Center, Washington. “He saw the conflict of interest of being a gatekeeper, how that would erode trust in a personal relationship with your personal doctor.”

“Gayle thought it was a disaster waiting to happen, and it was,” he said, referring to the eventual rejection by the public of barriers to direct access to specialists.

Through the 1990s and more recently, Dr. Stephens expressed frustration with the “medical-industrial complex” and the decline of family medicine after its surge in the 1970s and 1980s, Dr. Green said. “But in my opinion, near the end of his life, he was encouraged by young leaders who he saw grasped the important ideas from the ages.”

Dr. Stephens’ interest in medical education extended to nurses and nurse practitioners (the latter of whom had begun their discipline in the mid-1960s), and to optometrists, for whom he taught a recurring course in “physical diagnosis.”
 

A listener and proponent of listening

Linda Tompkins, RN, FNP, of Newton, Kan., trained with Dr. Stephens at part of a year-long nurse education program in the early 1970s at Wichita (Kan.) State University, where he was leading the department of family practice (prior to moving to Alabama). “You couldn’t ask too many questions,” she said. “And he never talked down to us, he wasn’t condescending. There were not a lot of doctors like that.”

Dr. Stephens spoke and wrote often about the importance of listening –about how it was vital to the “durable clinical relationship.” It was also vital to his writing and to his impact on the teachers of family medicine, said Dan Ostergaard, MD, who served as a residency director and in various staff leadership positions at the American Academy of Family Physicians, including in its division of education.

“He created a lot of aha moments for me, about where we came from and what we really need to be [as a specialty] and where we need to go,” said Dr. Ostergaard. “To be such a great thinker and a great writer, you have to be a great listener.”

“I can just visualize him,” he said, “leaning back in his chair while we were talking about residency criteria [or other issues], with a half-smile on his face and his reading glasses down his note, smoking his pipe and just looking at all of us, listening.”

Dr. Stephens’ papers are housed in the Center for the History of Family Medicine, a project of the AAFP Foundation.

G. Gayle Stephens, MD, who is roundly regarded as one of the founders of family medicine, gave his talk “Family Medicine as Counterculture” at the Society of Teachers of Family Medicine annual conference in 1979, 10 years after the specialty’s establishment.

The speech was then published, republished 10 years later, and, like many of Dr. Stephen’s other essays and articles, remains very much alive in the minds of practicing family physicians, in the teachings of FP academicians, and in the Google searches of budding FPs.

The late Dr. Stephens saw family medicine as a counterculture within medicine, rooted in social change. In his speech he examined these roots – in reform initiatives in the 1960s, and in certain philosophies and “minority” movements such as agrarianism and the preservation of rural life, utopianism, humanism, consumerism, and feminism.

He also looked forward, challenging the specialty to remain true to itself and its roots – to its belief in “uninhibited access” to medical care for everyone, for instance, and to continual whole-person and family-oriented care – and cautioned against moving to resemble the “rest of the medical bureaucracy.”

“Clearly we have been on the side of change in American life. We have identified ourselves with certain minorities and minority positions ... [and] been counter to many of the dominant forces in society,” Dr. Stephens said in his talk. Family practice “succeeded in the decade just past because we were identified with reforms that are more pervasive and powerful than ourselves.”

The family practice movement has “more in common with [the] counterculture than it does with the dominant scientific medical establishment,” he said.
 

A teacher and founder of medical education programs

Larry A. Green, MD, who was pursuing his own residency training as Dr. Stephens was leading a department of family practice, said Dr. Stephens “insisted that family medicine adhere to the notion that medicine is a moral vocation.”

“It was from this philosophical position that he became a synthesizer and observer and interpreter of what was going on in the development of family medicine,” said Dr. Green, Distinguished Professor and Epperson Zorn Chair for Innovation in Family Medicine and Primary Care at the University of Colorado at Denver, Aurora.

Dr. Stephens, who died at home in 2014 at the age of 85, was “probably the most important person in exposing what I now consider to be a fact – that family medicine was the product of social changes ... of social movements related to women’s rights, civil rights, and social responsibility,” Dr. Green said. “He could recall lessons from the past and forecast the challenges of the future. And there was no one more effective in clarifying the importance of personal [doctor-patient] relationships in family medicine.”

After years of general practice in rural Wichita, Kan., his wife Eula Jean’s hometown, Dr. Stephens founded and led one of the first family medicine residencies at Wesley Hospital in Wichita in 1967. His core principles, as described on today’s Wesley Family Medicine Residency website, included that a family physician consider the whole person, be honest, have a full scope of training including behavioral and mental health, and be “reflective about him/herself ... [learning about] his/her assets, liabilities, foibles, and idiosyncrasies.” Dr. Stephens, who had grown up in rural Ashburn, Mo., later became the founding dean of the School of Primary Medical Care at the University of Alabama in Huntsville and then chaired the department of family practice at the University of Alabama at Birmingham.
 

A thought leader for family medicine

He held numerous state and national leadership positions, and initiated what became the Keystone Conference Series – an invitational gathering of leaders in family medicine that examined and discuss the specialty’s ongoing development. In 2006, he was elected to the Institute of Medicine of the National Academies of Science.

Dr. Stephens authored a textbook, The Intellectual Basis of Family Medicine (Tucson, Ariz.: Winter Publishing Company, 1982), and authored essays, which Dr. Green said will stand the test of time.

“Some of us refer to him as the poet laureate of family medicine,” Dr. Green noted.

In a 1974 article on clinical wisdom, Dr. Stephens wrote that “it is not enough to determine what condition the patient has, but also what patient has the condition.” In another of these essays, which was published in 1979, Dr. Stephens wrote that “physicians need to keep in touch with their own tradition and with public welfare if they are to be considered moral by the society that sponsors them, and from which they take their strength and privilege.”

These excerpts are featured in an article by John P. Geyman, MD, published in 2011 in Family Medicine, called “G. Gayle Stephens Festschrift”.
 

A ‘progressive force’

Linda Prine, MD, professor of family and community medicine at the Icahn School of Medicine at Mount Sinai, New York, knows of Dr. Stephens from her teachers. “The people I looked up to when I was a younger physician were quoting his Counterculture article,” she said.

“It’s not that I studied him. But whenever I heard someone speak about the values of family medicine, his name would come up [and] the values of universal health care and community care and putting the patients’ interests first ahead of the insurance companies and being a doctor for the whole family,” Dr. Prine said. Dr. Stephens was a “progressive force that our specialty has not always lived up to.”

Dr. Stephens voiced serious concerns about the impact of managed care in the 1980s and of “gatekeeping,” a practice intended to control access to specialists and reduce costs.

“He was many times not welcomed by family medicine [for his warnings] against the temptations that managed care presented,” said Dr. Green, the founding director of the Robert Graham Center, Washington. “He saw the conflict of interest of being a gatekeeper, how that would erode trust in a personal relationship with your personal doctor.”

“Gayle thought it was a disaster waiting to happen, and it was,” he said, referring to the eventual rejection by the public of barriers to direct access to specialists.

Through the 1990s and more recently, Dr. Stephens expressed frustration with the “medical-industrial complex” and the decline of family medicine after its surge in the 1970s and 1980s, Dr. Green said. “But in my opinion, near the end of his life, he was encouraged by young leaders who he saw grasped the important ideas from the ages.”

Dr. Stephens’ interest in medical education extended to nurses and nurse practitioners (the latter of whom had begun their discipline in the mid-1960s), and to optometrists, for whom he taught a recurring course in “physical diagnosis.”
 

A listener and proponent of listening

Linda Tompkins, RN, FNP, of Newton, Kan., trained with Dr. Stephens at part of a year-long nurse education program in the early 1970s at Wichita (Kan.) State University, where he was leading the department of family practice (prior to moving to Alabama). “You couldn’t ask too many questions,” she said. “And he never talked down to us, he wasn’t condescending. There were not a lot of doctors like that.”

Dr. Stephens spoke and wrote often about the importance of listening –about how it was vital to the “durable clinical relationship.” It was also vital to his writing and to his impact on the teachers of family medicine, said Dan Ostergaard, MD, who served as a residency director and in various staff leadership positions at the American Academy of Family Physicians, including in its division of education.

“He created a lot of aha moments for me, about where we came from and what we really need to be [as a specialty] and where we need to go,” said Dr. Ostergaard. “To be such a great thinker and a great writer, you have to be a great listener.”

“I can just visualize him,” he said, “leaning back in his chair while we were talking about residency criteria [or other issues], with a half-smile on his face and his reading glasses down his note, smoking his pipe and just looking at all of us, listening.”

Dr. Stephens’ papers are housed in the Center for the History of Family Medicine, a project of the AAFP Foundation.

G. Gayle Stephens, MD, who is roundly regarded as one of the founders of family medicine, gave his talk “Family Medicine as Counterculture” at the Society of Teachers of Family Medicine annual conference in 1979, 10 years after the specialty’s establishment.

The speech was then published, republished 10 years later, and, like many of Dr. Stephen’s other essays and articles, remains very much alive in the minds of practicing family physicians, in the teachings of FP academicians, and in the Google searches of budding FPs.

The late Dr. Stephens saw family medicine as a counterculture within medicine, rooted in social change. In his speech he examined these roots – in reform initiatives in the 1960s, and in certain philosophies and “minority” movements such as agrarianism and the preservation of rural life, utopianism, humanism, consumerism, and feminism.

He also looked forward, challenging the specialty to remain true to itself and its roots – to its belief in “uninhibited access” to medical care for everyone, for instance, and to continual whole-person and family-oriented care – and cautioned against moving to resemble the “rest of the medical bureaucracy.”

“Clearly we have been on the side of change in American life. We have identified ourselves with certain minorities and minority positions ... [and] been counter to many of the dominant forces in society,” Dr. Stephens said in his talk. Family practice “succeeded in the decade just past because we were identified with reforms that are more pervasive and powerful than ourselves.”

The family practice movement has “more in common with [the] counterculture than it does with the dominant scientific medical establishment,” he said.
 

A teacher and founder of medical education programs

Larry A. Green, MD, who was pursuing his own residency training as Dr. Stephens was leading a department of family practice, said Dr. Stephens “insisted that family medicine adhere to the notion that medicine is a moral vocation.”

“It was from this philosophical position that he became a synthesizer and observer and interpreter of what was going on in the development of family medicine,” said Dr. Green, Distinguished Professor and Epperson Zorn Chair for Innovation in Family Medicine and Primary Care at the University of Colorado at Denver, Aurora.

Dr. Stephens, who died at home in 2014 at the age of 85, was “probably the most important person in exposing what I now consider to be a fact – that family medicine was the product of social changes ... of social movements related to women’s rights, civil rights, and social responsibility,” Dr. Green said. “He could recall lessons from the past and forecast the challenges of the future. And there was no one more effective in clarifying the importance of personal [doctor-patient] relationships in family medicine.”

After years of general practice in rural Wichita, Kan., his wife Eula Jean’s hometown, Dr. Stephens founded and led one of the first family medicine residencies at Wesley Hospital in Wichita in 1967. His core principles, as described on today’s Wesley Family Medicine Residency website, included that a family physician consider the whole person, be honest, have a full scope of training including behavioral and mental health, and be “reflective about him/herself ... [learning about] his/her assets, liabilities, foibles, and idiosyncrasies.” Dr. Stephens, who had grown up in rural Ashburn, Mo., later became the founding dean of the School of Primary Medical Care at the University of Alabama in Huntsville and then chaired the department of family practice at the University of Alabama at Birmingham.
 

A thought leader for family medicine

He held numerous state and national leadership positions, and initiated what became the Keystone Conference Series – an invitational gathering of leaders in family medicine that examined and discuss the specialty’s ongoing development. In 2006, he was elected to the Institute of Medicine of the National Academies of Science.

Dr. Stephens authored a textbook, The Intellectual Basis of Family Medicine (Tucson, Ariz.: Winter Publishing Company, 1982), and authored essays, which Dr. Green said will stand the test of time.

“Some of us refer to him as the poet laureate of family medicine,” Dr. Green noted.

In a 1974 article on clinical wisdom, Dr. Stephens wrote that “it is not enough to determine what condition the patient has, but also what patient has the condition.” In another of these essays, which was published in 1979, Dr. Stephens wrote that “physicians need to keep in touch with their own tradition and with public welfare if they are to be considered moral by the society that sponsors them, and from which they take their strength and privilege.”

These excerpts are featured in an article by John P. Geyman, MD, published in 2011 in Family Medicine, called “G. Gayle Stephens Festschrift”.
 

A ‘progressive force’

Linda Prine, MD, professor of family and community medicine at the Icahn School of Medicine at Mount Sinai, New York, knows of Dr. Stephens from her teachers. “The people I looked up to when I was a younger physician were quoting his Counterculture article,” she said.

“It’s not that I studied him. But whenever I heard someone speak about the values of family medicine, his name would come up [and] the values of universal health care and community care and putting the patients’ interests first ahead of the insurance companies and being a doctor for the whole family,” Dr. Prine said. Dr. Stephens was a “progressive force that our specialty has not always lived up to.”

Dr. Stephens voiced serious concerns about the impact of managed care in the 1980s and of “gatekeeping,” a practice intended to control access to specialists and reduce costs.

“He was many times not welcomed by family medicine [for his warnings] against the temptations that managed care presented,” said Dr. Green, the founding director of the Robert Graham Center, Washington. “He saw the conflict of interest of being a gatekeeper, how that would erode trust in a personal relationship with your personal doctor.”

“Gayle thought it was a disaster waiting to happen, and it was,” he said, referring to the eventual rejection by the public of barriers to direct access to specialists.

Through the 1990s and more recently, Dr. Stephens expressed frustration with the “medical-industrial complex” and the decline of family medicine after its surge in the 1970s and 1980s, Dr. Green said. “But in my opinion, near the end of his life, he was encouraged by young leaders who he saw grasped the important ideas from the ages.”

Dr. Stephens’ interest in medical education extended to nurses and nurse practitioners (the latter of whom had begun their discipline in the mid-1960s), and to optometrists, for whom he taught a recurring course in “physical diagnosis.”
 

A listener and proponent of listening

Linda Tompkins, RN, FNP, of Newton, Kan., trained with Dr. Stephens at part of a year-long nurse education program in the early 1970s at Wichita (Kan.) State University, where he was leading the department of family practice (prior to moving to Alabama). “You couldn’t ask too many questions,” she said. “And he never talked down to us, he wasn’t condescending. There were not a lot of doctors like that.”

Dr. Stephens spoke and wrote often about the importance of listening –about how it was vital to the “durable clinical relationship.” It was also vital to his writing and to his impact on the teachers of family medicine, said Dan Ostergaard, MD, who served as a residency director and in various staff leadership positions at the American Academy of Family Physicians, including in its division of education.

“He created a lot of aha moments for me, about where we came from and what we really need to be [as a specialty] and where we need to go,” said Dr. Ostergaard. “To be such a great thinker and a great writer, you have to be a great listener.”

“I can just visualize him,” he said, “leaning back in his chair while we were talking about residency criteria [or other issues], with a half-smile on his face and his reading glasses down his note, smoking his pipe and just looking at all of us, listening.”

Dr. Stephens’ papers are housed in the Center for the History of Family Medicine, a project of the AAFP Foundation.

Black patients diagnosed by dermatologists with cutaneous sarcoidosis were significantly more likely to have unrecognized systemic organ involvement than were non-Black patients, according to a retrospective chart review of patients seen at Massachusetts General Hospital and Brigham and Women’s Hospital, both in Boston.

Black patients were also significantly more likely to have two or more organs involved and have higher rates of cardiac involvement, the latter of which is associated with worse prognosis. “Our data suggest there may be substantial variations in organ involvement between racial groups of patients presenting with cutaneous sarcoidosis,” said medical student Kylee Kus, a medical student at Oakland University, Auburn Hills, Mich., who presented the findings with Bina Kassamali, a medical student at Harvard University, Boston, at the annual Skin of Color Society scientific symposium.

Sotonye Imadojemu, MD, MBE; Avery LeChance, MD, MPH; and Ruth Anne Vleugels, MD, MPH, MBA; of Brigham and Women’s Hospital, are cosenior authors of the abstract.

The researchers identified 111 patients who were diagnosed with cutaneous sarcoidosis over a 20-year period (January 2000–December 2019), 50 of whom presented without established extracutaneous disease. They examined the charts of these 50 patients for whether subsequent work-up revealed systemic disease.

Of the 50 patients, 9 were Black. Seven of these nine patients (77.8%), were found to have systemic involvement, compared with 14 of 41 (46.3%) non-Black patients – a 31.5% higher probability (P < .05). One-third of the nine Black patients were found to have disease in one organ, and 44.4% in two or more organs. In non-Black patients, these rates were 12.2% and 34.1%, respectively.

Cardiovascular involvement was not found in any of the non-Black patients who had extracutaneous disease, but was found in 29% of the Black patients with extracutaneous disease, a statistically significant difference.

Black patients are known to be at higher risk for sarcoidosis than non-Black patients, and because “there is an association between cardiac sarcoid involvement and poor prognosis largely due to manifestations such as heart block, arrhythmias, and heart failure ... the study helps demonstrate how this organ involvement can disproportionately affect the Black population,” Ms. Kassamali said in an interview after the meeting.

A separate, recently published analysis of data from the same patient population examined the work-ups that patients received after a dermatologist’s diagnosis of sarcoidosis and found that patients with no previous systemic work-up were subsequently assessed for cardiac involvement in only 58.3% of cases. Assessment for pulmonary and ocular disease was completed more than 90% of the time.

“Crucial testing for cardiac involvement fell short,” Dr. Imadojemu, of the department of dermatology, Brigham and Women’s Hospital, and coinvestigators wrote in the research letter.

“Because the cutaneous manifestations of sarcoidosis often present at disease onset, dermatologists may be the first physicians to diagnose a patient with sarcoidosis,” they wrote. “As such, dermatologists are often responsible for initiating the appropriate evaluation of patients with sarcoidosis.”

Pulmonary involvement occurs in nearly all cases of sarcoidosis, while ocular and cardiac disease develop in approximately 25% and 10% of patients, respectively. Cardiac sarcoidosis is usually asymptomatic and accounts for 13%-25% of sarcoidosis-related deaths in the United States, they wrote.

An electrocardiogram is the appropriate initial screening tool and “is warranted in all patients with sarcoidosis,” they advised.

Black patients diagnosed by dermatologists with cutaneous sarcoidosis were significantly more likely to have unrecognized systemic organ involvement than were non-Black patients, according to a retrospective chart review of patients seen at Massachusetts General Hospital and Brigham and Women’s Hospital, both in Boston.

Black patients were also significantly more likely to have two or more organs involved and have higher rates of cardiac involvement, the latter of which is associated with worse prognosis. “Our data suggest there may be substantial variations in organ involvement between racial groups of patients presenting with cutaneous sarcoidosis,” said medical student Kylee Kus, a medical student at Oakland University, Auburn Hills, Mich., who presented the findings with Bina Kassamali, a medical student at Harvard University, Boston, at the annual Skin of Color Society scientific symposium.

Sotonye Imadojemu, MD, MBE; Avery LeChance, MD, MPH; and Ruth Anne Vleugels, MD, MPH, MBA; of Brigham and Women’s Hospital, are cosenior authors of the abstract.

The researchers identified 111 patients who were diagnosed with cutaneous sarcoidosis over a 20-year period (January 2000–December 2019), 50 of whom presented without established extracutaneous disease. They examined the charts of these 50 patients for whether subsequent work-up revealed systemic disease.

Of the 50 patients, 9 were Black. Seven of these nine patients (77.8%), were found to have systemic involvement, compared with 14 of 41 (46.3%) non-Black patients – a 31.5% higher probability (P < .05). One-third of the nine Black patients were found to have disease in one organ, and 44.4% in two or more organs. In non-Black patients, these rates were 12.2% and 34.1%, respectively.

Cardiovascular involvement was not found in any of the non-Black patients who had extracutaneous disease, but was found in 29% of the Black patients with extracutaneous disease, a statistically significant difference.

Black patients are known to be at higher risk for sarcoidosis than non-Black patients, and because “there is an association between cardiac sarcoid involvement and poor prognosis largely due to manifestations such as heart block, arrhythmias, and heart failure ... the study helps demonstrate how this organ involvement can disproportionately affect the Black population,” Ms. Kassamali said in an interview after the meeting.

A separate, recently published analysis of data from the same patient population examined the work-ups that patients received after a dermatologist’s diagnosis of sarcoidosis and found that patients with no previous systemic work-up were subsequently assessed for cardiac involvement in only 58.3% of cases. Assessment for pulmonary and ocular disease was completed more than 90% of the time.

“Crucial testing for cardiac involvement fell short,” Dr. Imadojemu, of the department of dermatology, Brigham and Women’s Hospital, and coinvestigators wrote in the research letter.

“Because the cutaneous manifestations of sarcoidosis often present at disease onset, dermatologists may be the first physicians to diagnose a patient with sarcoidosis,” they wrote. “As such, dermatologists are often responsible for initiating the appropriate evaluation of patients with sarcoidosis.”

Pulmonary involvement occurs in nearly all cases of sarcoidosis, while ocular and cardiac disease develop in approximately 25% and 10% of patients, respectively. Cardiac sarcoidosis is usually asymptomatic and accounts for 13%-25% of sarcoidosis-related deaths in the United States, they wrote.

An electrocardiogram is the appropriate initial screening tool and “is warranted in all patients with sarcoidosis,” they advised.

Black patients diagnosed by dermatologists with cutaneous sarcoidosis were significantly more likely to have unrecognized systemic organ involvement than were non-Black patients, according to a retrospective chart review of patients seen at Massachusetts General Hospital and Brigham and Women’s Hospital, both in Boston.

Black patients were also significantly more likely to have two or more organs involved and have higher rates of cardiac involvement, the latter of which is associated with worse prognosis. “Our data suggest there may be substantial variations in organ involvement between racial groups of patients presenting with cutaneous sarcoidosis,” said medical student Kylee Kus, a medical student at Oakland University, Auburn Hills, Mich., who presented the findings with Bina Kassamali, a medical student at Harvard University, Boston, at the annual Skin of Color Society scientific symposium.

Sotonye Imadojemu, MD, MBE; Avery LeChance, MD, MPH; and Ruth Anne Vleugels, MD, MPH, MBA; of Brigham and Women’s Hospital, are cosenior authors of the abstract.

The researchers identified 111 patients who were diagnosed with cutaneous sarcoidosis over a 20-year period (January 2000–December 2019), 50 of whom presented without established extracutaneous disease. They examined the charts of these 50 patients for whether subsequent work-up revealed systemic disease.

Of the 50 patients, 9 were Black. Seven of these nine patients (77.8%), were found to have systemic involvement, compared with 14 of 41 (46.3%) non-Black patients – a 31.5% higher probability (P < .05). One-third of the nine Black patients were found to have disease in one organ, and 44.4% in two or more organs. In non-Black patients, these rates were 12.2% and 34.1%, respectively.

Cardiovascular involvement was not found in any of the non-Black patients who had extracutaneous disease, but was found in 29% of the Black patients with extracutaneous disease, a statistically significant difference.

Black patients are known to be at higher risk for sarcoidosis than non-Black patients, and because “there is an association between cardiac sarcoid involvement and poor prognosis largely due to manifestations such as heart block, arrhythmias, and heart failure ... the study helps demonstrate how this organ involvement can disproportionately affect the Black population,” Ms. Kassamali said in an interview after the meeting.

A separate, recently published analysis of data from the same patient population examined the work-ups that patients received after a dermatologist’s diagnosis of sarcoidosis and found that patients with no previous systemic work-up were subsequently assessed for cardiac involvement in only 58.3% of cases. Assessment for pulmonary and ocular disease was completed more than 90% of the time.

“Crucial testing for cardiac involvement fell short,” Dr. Imadojemu, of the department of dermatology, Brigham and Women’s Hospital, and coinvestigators wrote in the research letter.

“Because the cutaneous manifestations of sarcoidosis often present at disease onset, dermatologists may be the first physicians to diagnose a patient with sarcoidosis,” they wrote. “As such, dermatologists are often responsible for initiating the appropriate evaluation of patients with sarcoidosis.”

Pulmonary involvement occurs in nearly all cases of sarcoidosis, while ocular and cardiac disease develop in approximately 25% and 10% of patients, respectively. Cardiac sarcoidosis is usually asymptomatic and accounts for 13%-25% of sarcoidosis-related deaths in the United States, they wrote.

An electrocardiogram is the appropriate initial screening tool and “is warranted in all patients with sarcoidosis,” they advised.

Black and Hispanic patients had higher inpatient care utilization for mycosis fungoides (MF) – and were admitted at a younger age – compared with White patients, according to an analysis of the 2012-2017 National Inpatient Sample (NIS).

The findings are consistent with prior studies implicating earlier and more severe disease in Black and Hispanic patients, and reinforce the importance of accurate diagnosis and early treatment.

Dermatologists should maintain “a higher index of suspicion for MF in patients with skin of color, as early diagnosis may help mitigate the downstream costs of management,” Justin Choi, BA, a medical student at the University of Illinois at Chicago, said at the annual Skin of Color Society symposium.

Mr. Choi and coinvestigators, led by Shawn Kwatra, MD, of Johns Hopkins University, Baltimore, identified hospital admissions for MF in the NIS for 10,790 White patients, 4,020 Black patients, and 1,615 Hispanic patients over the 5-year period. The inpatient prevalence of MF – the most common variant of primary cutaneous T-cell lymphoma – was highest in these groups.

Black and Hispanic patients who were hospitalized for MF were significantly younger than White patients, with a mean age of 51.7 years and 48.5 years, respectively, compared with 59.9 years (P < .001 in each case). They also had longer lengths of stay: 8.34 days on average for Black patients and 8.88 for Hispanic patients, compared with 6.66 days for White patients (P < .001 and P = .001, respectively).

Hispanic patients accrued the highest costs of care (a mean of $107,242 vs. $64,049, P =.003) and underwent more procedures (a mean of 2.43 vs. 1.93, P = .004) than White patients. Black patients similarly had higher costs associated with their hospital stay (a mean of $75,053 vs. $64,049, P =.042).

In a multivariate linear regression adjusted for age, sex and insurance type, Black race remained significantly associated with a longer LOS than White race, and Hispanic ethnicity with a longer LOS, increased costs, and more procedures than White race.

The NIS is a publicly available, all-payer inpatient care database developed for the Agency for Healthcare Research and Quality’s Healthcare Cost and Utilization Project.

Mr. Choi is a dermatology research fellow working under the guidance of Dr. Kwatra.

Black and Hispanic patients had higher inpatient care utilization for mycosis fungoides (MF) – and were admitted at a younger age – compared with White patients, according to an analysis of the 2012-2017 National Inpatient Sample (NIS).

The findings are consistent with prior studies implicating earlier and more severe disease in Black and Hispanic patients, and reinforce the importance of accurate diagnosis and early treatment.

Dermatologists should maintain “a higher index of suspicion for MF in patients with skin of color, as early diagnosis may help mitigate the downstream costs of management,” Justin Choi, BA, a medical student at the University of Illinois at Chicago, said at the annual Skin of Color Society symposium.

Mr. Choi and coinvestigators, led by Shawn Kwatra, MD, of Johns Hopkins University, Baltimore, identified hospital admissions for MF in the NIS for 10,790 White patients, 4,020 Black patients, and 1,615 Hispanic patients over the 5-year period. The inpatient prevalence of MF – the most common variant of primary cutaneous T-cell lymphoma – was highest in these groups.

Black and Hispanic patients who were hospitalized for MF were significantly younger than White patients, with a mean age of 51.7 years and 48.5 years, respectively, compared with 59.9 years (P < .001 in each case). They also had longer lengths of stay: 8.34 days on average for Black patients and 8.88 for Hispanic patients, compared with 6.66 days for White patients (P < .001 and P = .001, respectively).

Hispanic patients accrued the highest costs of care (a mean of $107,242 vs. $64,049, P =.003) and underwent more procedures (a mean of 2.43 vs. 1.93, P = .004) than White patients. Black patients similarly had higher costs associated with their hospital stay (a mean of $75,053 vs. $64,049, P =.042).

In a multivariate linear regression adjusted for age, sex and insurance type, Black race remained significantly associated with a longer LOS than White race, and Hispanic ethnicity with a longer LOS, increased costs, and more procedures than White race.

The NIS is a publicly available, all-payer inpatient care database developed for the Agency for Healthcare Research and Quality’s Healthcare Cost and Utilization Project.

Mr. Choi is a dermatology research fellow working under the guidance of Dr. Kwatra.

Black and Hispanic patients had higher inpatient care utilization for mycosis fungoides (MF) – and were admitted at a younger age – compared with White patients, according to an analysis of the 2012-2017 National Inpatient Sample (NIS).

The findings are consistent with prior studies implicating earlier and more severe disease in Black and Hispanic patients, and reinforce the importance of accurate diagnosis and early treatment.

Dermatologists should maintain “a higher index of suspicion for MF in patients with skin of color, as early diagnosis may help mitigate the downstream costs of management,” Justin Choi, BA, a medical student at the University of Illinois at Chicago, said at the annual Skin of Color Society symposium.

Mr. Choi and coinvestigators, led by Shawn Kwatra, MD, of Johns Hopkins University, Baltimore, identified hospital admissions for MF in the NIS for 10,790 White patients, 4,020 Black patients, and 1,615 Hispanic patients over the 5-year period. The inpatient prevalence of MF – the most common variant of primary cutaneous T-cell lymphoma – was highest in these groups.

Black and Hispanic patients who were hospitalized for MF were significantly younger than White patients, with a mean age of 51.7 years and 48.5 years, respectively, compared with 59.9 years (P < .001 in each case). They also had longer lengths of stay: 8.34 days on average for Black patients and 8.88 for Hispanic patients, compared with 6.66 days for White patients (P < .001 and P = .001, respectively).

Hispanic patients accrued the highest costs of care (a mean of $107,242 vs. $64,049, P =.003) and underwent more procedures (a mean of 2.43 vs. 1.93, P = .004) than White patients. Black patients similarly had higher costs associated with their hospital stay (a mean of $75,053 vs. $64,049, P =.042).

In a multivariate linear regression adjusted for age, sex and insurance type, Black race remained significantly associated with a longer LOS than White race, and Hispanic ethnicity with a longer LOS, increased costs, and more procedures than White race.

The NIS is a publicly available, all-payer inpatient care database developed for the Agency for Healthcare Research and Quality’s Healthcare Cost and Utilization Project.

Mr. Choi is a dermatology research fellow working under the guidance of Dr. Kwatra.

Asian Americans with melanoma presented at a younger age but with higher rates of invasive disease and at later stages of disease, compared with non-Hispanic Whites, according to a secondary analysis of data from the National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) program.

The findings are consistent with previous studies indicating delayed detection of melanoma in Asians, compared with non-Hispanic Whites, and provide a window into Asian American communities specifically, Erica M. Lin, a medical student at Brown University, Providence, R.I., said at the annual Skin of Color Society Symposium. The majority of studies on melanoma in Asians have originated in Asia, noted Ms. Lin, whose coauthor was Eunyoung Cho, ScD, an associate professor in the department of dermatology and director of the clinical and translational research program at Brown University. Their analysis covered registries from 10 geographic areas representing 54% of the U.S. Asian American population over a 25-year period, from 1990 to 2014.

Asian Americans with melanoma were more likely to present at an invasive stage than non-Hispanic Whites (82.9% vs. 72.2%, P < .001), and they were significantly more likely to present when the disease had progressed to a distant stage (9.39% vs. 2.51%, P < .001), even though they were of younger ages at the time of those diagnoses, Ms. Lin reported at the meeting. (The numbers do not account for unknown or unstaged melanoma cases.)

Significantly fewer Asian Americans presented at the “in situ” stage, compared with non-Hispanic Whites (17.11% vs. 27.78%). The lower extremities were the most common site in Asian Americans, compared with the trunk in Non-Hispanic Whites.

The SEER registries covered the eight largest Asian American groups: Asian Indians/Pakistanis, Chinese, Filipinos, Japanese, Kampucheans (Cambodians), Koreans, Laotians, and Vietnamese. Melanoma was more common in females across the groups (53% of females vs. 47% of males), with the exception of Asian Indians/Pakistanis.

While melanoma increased significantly over time among non-Hispanic Whites – a mean 24% increase per 5-year period – there was “no significant change in melanoma rates in Asians,” Ms. Lin said.

The lack of increase in Asian American communities combined with the other findings is “potentially concerning” and suggests “that there may be cases that are not being identified,” she said in an interview after the meeting. In their abstract, she and Dr. Cho noted that their findings underscore the need for further prevention, screening, and surveillance measures.

The NCI’s SEER program is a coordinated system of cancer registries across the United States that collects data on every case of cancer reported in 19 geographic areas.

Asian Americans with melanoma presented at a younger age but with higher rates of invasive disease and at later stages of disease, compared with non-Hispanic Whites, according to a secondary analysis of data from the National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) program.

The findings are consistent with previous studies indicating delayed detection of melanoma in Asians, compared with non-Hispanic Whites, and provide a window into Asian American communities specifically, Erica M. Lin, a medical student at Brown University, Providence, R.I., said at the annual Skin of Color Society Symposium. The majority of studies on melanoma in Asians have originated in Asia, noted Ms. Lin, whose coauthor was Eunyoung Cho, ScD, an associate professor in the department of dermatology and director of the clinical and translational research program at Brown University. Their analysis covered registries from 10 geographic areas representing 54% of the U.S. Asian American population over a 25-year period, from 1990 to 2014.

Asian Americans with melanoma were more likely to present at an invasive stage than non-Hispanic Whites (82.9% vs. 72.2%, P < .001), and they were significantly more likely to present when the disease had progressed to a distant stage (9.39% vs. 2.51%, P < .001), even though they were of younger ages at the time of those diagnoses, Ms. Lin reported at the meeting. (The numbers do not account for unknown or unstaged melanoma cases.)

Significantly fewer Asian Americans presented at the “in situ” stage, compared with non-Hispanic Whites (17.11% vs. 27.78%). The lower extremities were the most common site in Asian Americans, compared with the trunk in Non-Hispanic Whites.

The SEER registries covered the eight largest Asian American groups: Asian Indians/Pakistanis, Chinese, Filipinos, Japanese, Kampucheans (Cambodians), Koreans, Laotians, and Vietnamese. Melanoma was more common in females across the groups (53% of females vs. 47% of males), with the exception of Asian Indians/Pakistanis.

While melanoma increased significantly over time among non-Hispanic Whites – a mean 24% increase per 5-year period – there was “no significant change in melanoma rates in Asians,” Ms. Lin said.

The lack of increase in Asian American communities combined with the other findings is “potentially concerning” and suggests “that there may be cases that are not being identified,” she said in an interview after the meeting. In their abstract, she and Dr. Cho noted that their findings underscore the need for further prevention, screening, and surveillance measures.

The NCI’s SEER program is a coordinated system of cancer registries across the United States that collects data on every case of cancer reported in 19 geographic areas.

Asian Americans with melanoma presented at a younger age but with higher rates of invasive disease and at later stages of disease, compared with non-Hispanic Whites, according to a secondary analysis of data from the National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) program.

The findings are consistent with previous studies indicating delayed detection of melanoma in Asians, compared with non-Hispanic Whites, and provide a window into Asian American communities specifically, Erica M. Lin, a medical student at Brown University, Providence, R.I., said at the annual Skin of Color Society Symposium. The majority of studies on melanoma in Asians have originated in Asia, noted Ms. Lin, whose coauthor was Eunyoung Cho, ScD, an associate professor in the department of dermatology and director of the clinical and translational research program at Brown University. Their analysis covered registries from 10 geographic areas representing 54% of the U.S. Asian American population over a 25-year period, from 1990 to 2014.

Asian Americans with melanoma were more likely to present at an invasive stage than non-Hispanic Whites (82.9% vs. 72.2%, P < .001), and they were significantly more likely to present when the disease had progressed to a distant stage (9.39% vs. 2.51%, P < .001), even though they were of younger ages at the time of those diagnoses, Ms. Lin reported at the meeting. (The numbers do not account for unknown or unstaged melanoma cases.)

Significantly fewer Asian Americans presented at the “in situ” stage, compared with non-Hispanic Whites (17.11% vs. 27.78%). The lower extremities were the most common site in Asian Americans, compared with the trunk in Non-Hispanic Whites.

The SEER registries covered the eight largest Asian American groups: Asian Indians/Pakistanis, Chinese, Filipinos, Japanese, Kampucheans (Cambodians), Koreans, Laotians, and Vietnamese. Melanoma was more common in females across the groups (53% of females vs. 47% of males), with the exception of Asian Indians/Pakistanis.

While melanoma increased significantly over time among non-Hispanic Whites – a mean 24% increase per 5-year period – there was “no significant change in melanoma rates in Asians,” Ms. Lin said.

The lack of increase in Asian American communities combined with the other findings is “potentially concerning” and suggests “that there may be cases that are not being identified,” she said in an interview after the meeting. In their abstract, she and Dr. Cho noted that their findings underscore the need for further prevention, screening, and surveillance measures.

The NCI’s SEER program is a coordinated system of cancer registries across the United States that collects data on every case of cancer reported in 19 geographic areas.

Patients with vitiligo have faced significant impacts psychosocially and in many cases, profound losses of identity – and they’ve had only minimal success with treatment, according to participants who spoke at and provided input at a public meeting on patient-focused drug development for the disease.

FG Trade/E+

The virtual meeting, held in March, was part of the Food and Drug Administration’s Patient-Focused Drug Development (PFDD) initiative, which began in 2012 and aims to provide a systematic way for patients’ experiences, needs and priorities to be “captured and meaningfully incorporated” into drug development and evaluation.

Seemal Desai, MD, of the department of dermatology at the University of Texas, Dallas, who attended the meeting as an observer, said in a later interview that while “all skin diseases have a psychosocial component … vitiligo is a really unique one, because it really relates to the patient’s own identity.

“What I heard loud and clear from the FDA [leaders who ran and attended the meeting] is recognition that patients are suffering. They needed to hear about the emotional devastation of the disease and how it is a medical condition,” Dr. Desai said.

The meeting was the “first-ever vitiligo meeting at the FDA” and was a “historic moment for the vitiligo community,” he added.

The pigmentation disorder affects 1% of the world’s population. Nearly 50% have an onset before age 20, and onset before age 12 is common, Brenda Carr, MD, medical officer with the FDA’s Division of Dermatology and Dentistry in the Center for Drug Evaluation and Research, said in an introductory overview.

The only FDA-approved treatment for vitiligo is monobenzone cream, but this is indicated for final depigmentation in extensive vitiligo and is no longer marketed. Treatment options include corticosteroids, calcineurin inhibitors, vitamin D analogues, phototherapy, surgical treatments (tissue grafts and cellular grafts), and camouflage (make-up, tattoos, self-tanning products), Dr. Carr said.

Patients participated in one of two panels – one about the health effects and daily impacts of vitiligo and the other about treatments – or submitted input electronically. All patients were invited to answer poll questions and open-ended queries, including questions about how they would assess new treatments.

Several panel members who are Black shared series of photos that showed the evolution of defined white patches into widespread, generalized depigmentation. One man with skin of color who lives in the Netherlands said he has had vitiligo since the age of 12, but that when he became older, over a 4-year period, he was “transformed from a man of Indonesian roots to a totally white man.”

Experiencing only minimal benefit from treatment and the short-term effectiveness of treatments were the top two answers to a poll question asking participants about the most burdensome impacts of the medical products and interventions they have used. Difficulty in accessing treatment, concern about serious risks of treatment, and uncertainty about long-term effects of treatment were other frequently chosen answers.

Patients described the onerous nature of phototherapy (treatments repeated several times a week over long periods) and other treatments, and several described feeling that some physicians did not take the condition seriously or fully know of treatment options.

In her closing remarks, Kendall Marcus, MD, director of the Division of Dermatology and Dentistry at the FDA, acknowledged the input. “Some of you have had difficulty having your disease taken seriously by physicians who view it as a cosmetic condition and are reluctant to treat because they believe your expectations will not be met, that it will be an exercise in frustration,” she said.

Regarding the impacts of treatments that have been utilized, “some of the treatments make it impossible to do other activities such as work or care for yourself in other ways,” Dr. Marcus said. “Certainly that’s not the kind of treatment … that anybody wants to have.”

Dr. Desai, who utilizes an array of oral and topical treatments and phototherapies in his practice, said he was surprised and disheartened to hear the level of concern about side effects of treatment. Most of those who expressed concerns alluded to phototherapy. “I think light treatments are very safe and effective,” he said in the interview. “I might equate [such concerns] to the older PUVA [psoralen plus UVA ultraviolet light] therapy, but not so much the newer therapies.”

The FDA participants probed patients for their perspective on a meaningful level of repigmentation and an acceptable level of risk for any new hypothetical treatment. Specifically, they asked whether patients would use a new topical cream approved for vitiligo if the cream needed to be applied once a day, would have up to 50% efficacy in some people, and would have common side effects of redness and irritation at the application site, mild acne, and burning, as well as several rarer but more serious side effects.

Only 36% answered yes; 24% said no, and 40% answered maybe. Some patients said during the meeting that they had accepted their condition and were not pursuing any treatment. Others said they were very interested in treatment but only if the level of repigmentation were significantly higher than 50%. Some described their fear that positive treatment effects would be short term only.

Meri Izrail Kohen, who lives in France and has lost half of her skin’s pigmentation, said that treatment efficacy is “not only about how much recovery of pigment it allows, but how long the recovery will last.” Some treatments will work for some patients, she said, “but even in these cases when we stop the treatment, it will come back somehow.”

Lee Thomas, a TV anchor in Detroit, and a reporter and author of the book “Turning White,” described how he tried “every treatment he could afford” but stopped trying 10 years ago. A treatment in Germany “gave me 80% of my pigment back, but it has gone again,” he said. “I would love to have my face back again. I was born a Black child, and I’d like to die a Black man.”

Patients also spoke of their skin burning easily outdoors; skin sensitivity, itchiness, and burning with the spread of disease; treatment expenses and not being able to afford treatment; and worsening of their vitiligo with the stress of the pandemic. Parents expressed having fear that their children would develop vitiligo and experience bullying, isolation, or other emotional or psychosocial impacts that they had experienced; one described having an almost-paralyzing anxiety when he saw patchy white spots on his 20-month-old daughter (it was not diagnosed as vitiligo).

Calls for further advancement with home phototherapy – which Dr. Desai said is a growing market but not yet adequately covered by insurance plans – were also made, as were pleas for research on the root causes of the disease.

Patients clearly indicated “that they need more efficacious treatments, and more comprehensive treatments,” said Dr. Desai, who chairs the advisory committee of the Global Vitiligo Foundation. “It’s disappointing to me that patients come in with a not fully optimistic viewpoint, with a lot of anxiety and angst that treatments are not going to work. … But the Agency needs to hear that. This means that there haven’t been good treatments and we need more.”

The FDA will accept public comments until May 10, 2021, at which time comments will be compiled into a summary report. FDA officials assured patients that the report would be visible and circulated not only within the FDA but among drug companies, researchers, and other product developers.

Patients with vitiligo have faced significant impacts psychosocially and in many cases, profound losses of identity – and they’ve had only minimal success with treatment, according to participants who spoke at and provided input at a public meeting on patient-focused drug development for the disease.

FG Trade/E+

The virtual meeting, held in March, was part of the Food and Drug Administration’s Patient-Focused Drug Development (PFDD) initiative, which began in 2012 and aims to provide a systematic way for patients’ experiences, needs and priorities to be “captured and meaningfully incorporated” into drug development and evaluation.

Seemal Desai, MD, of the department of dermatology at the University of Texas, Dallas, who attended the meeting as an observer, said in a later interview that while “all skin diseases have a psychosocial component … vitiligo is a really unique one, because it really relates to the patient’s own identity.

“What I heard loud and clear from the FDA [leaders who ran and attended the meeting] is recognition that patients are suffering. They needed to hear about the emotional devastation of the disease and how it is a medical condition,” Dr. Desai said.

The meeting was the “first-ever vitiligo meeting at the FDA” and was a “historic moment for the vitiligo community,” he added.

The pigmentation disorder affects 1% of the world’s population. Nearly 50% have an onset before age 20, and onset before age 12 is common, Brenda Carr, MD, medical officer with the FDA’s Division of Dermatology and Dentistry in the Center for Drug Evaluation and Research, said in an introductory overview.

The only FDA-approved treatment for vitiligo is monobenzone cream, but this is indicated for final depigmentation in extensive vitiligo and is no longer marketed. Treatment options include corticosteroids, calcineurin inhibitors, vitamin D analogues, phototherapy, surgical treatments (tissue grafts and cellular grafts), and camouflage (make-up, tattoos, self-tanning products), Dr. Carr said.

Patients participated in one of two panels – one about the health effects and daily impacts of vitiligo and the other about treatments – or submitted input electronically. All patients were invited to answer poll questions and open-ended queries, including questions about how they would assess new treatments.

Several panel members who are Black shared series of photos that showed the evolution of defined white patches into widespread, generalized depigmentation. One man with skin of color who lives in the Netherlands said he has had vitiligo since the age of 12, but that when he became older, over a 4-year period, he was “transformed from a man of Indonesian roots to a totally white man.”

Experiencing only minimal benefit from treatment and the short-term effectiveness of treatments were the top two answers to a poll question asking participants about the most burdensome impacts of the medical products and interventions they have used. Difficulty in accessing treatment, concern about serious risks of treatment, and uncertainty about long-term effects of treatment were other frequently chosen answers.

Patients described the onerous nature of phototherapy (treatments repeated several times a week over long periods) and other treatments, and several described feeling that some physicians did not take the condition seriously or fully know of treatment options.

In her closing remarks, Kendall Marcus, MD, director of the Division of Dermatology and Dentistry at the FDA, acknowledged the input. “Some of you have had difficulty having your disease taken seriously by physicians who view it as a cosmetic condition and are reluctant to treat because they believe your expectations will not be met, that it will be an exercise in frustration,” she said.

Regarding the impacts of treatments that have been utilized, “some of the treatments make it impossible to do other activities such as work or care for yourself in other ways,” Dr. Marcus said. “Certainly that’s not the kind of treatment … that anybody wants to have.”

Dr. Desai, who utilizes an array of oral and topical treatments and phototherapies in his practice, said he was surprised and disheartened to hear the level of concern about side effects of treatment. Most of those who expressed concerns alluded to phototherapy. “I think light treatments are very safe and effective,” he said in the interview. “I might equate [such concerns] to the older PUVA [psoralen plus UVA ultraviolet light] therapy, but not so much the newer therapies.”

The FDA participants probed patients for their perspective on a meaningful level of repigmentation and an acceptable level of risk for any new hypothetical treatment. Specifically, they asked whether patients would use a new topical cream approved for vitiligo if the cream needed to be applied once a day, would have up to 50% efficacy in some people, and would have common side effects of redness and irritation at the application site, mild acne, and burning, as well as several rarer but more serious side effects.

Only 36% answered yes; 24% said no, and 40% answered maybe. Some patients said during the meeting that they had accepted their condition and were not pursuing any treatment. Others said they were very interested in treatment but only if the level of repigmentation were significantly higher than 50%. Some described their fear that positive treatment effects would be short term only.

Meri Izrail Kohen, who lives in France and has lost half of her skin’s pigmentation, said that treatment efficacy is “not only about how much recovery of pigment it allows, but how long the recovery will last.” Some treatments will work for some patients, she said, “but even in these cases when we stop the treatment, it will come back somehow.”

Lee Thomas, a TV anchor in Detroit, and a reporter and author of the book “Turning White,” described how he tried “every treatment he could afford” but stopped trying 10 years ago. A treatment in Germany “gave me 80% of my pigment back, but it has gone again,” he said. “I would love to have my face back again. I was born a Black child, and I’d like to die a Black man.”

Patients also spoke of their skin burning easily outdoors; skin sensitivity, itchiness, and burning with the spread of disease; treatment expenses and not being able to afford treatment; and worsening of their vitiligo with the stress of the pandemic. Parents expressed having fear that their children would develop vitiligo and experience bullying, isolation, or other emotional or psychosocial impacts that they had experienced; one described having an almost-paralyzing anxiety when he saw patchy white spots on his 20-month-old daughter (it was not diagnosed as vitiligo).

Calls for further advancement with home phototherapy – which Dr. Desai said is a growing market but not yet adequately covered by insurance plans – were also made, as were pleas for research on the root causes of the disease.

Patients clearly indicated “that they need more efficacious treatments, and more comprehensive treatments,” said Dr. Desai, who chairs the advisory committee of the Global Vitiligo Foundation. “It’s disappointing to me that patients come in with a not fully optimistic viewpoint, with a lot of anxiety and angst that treatments are not going to work. … But the Agency needs to hear that. This means that there haven’t been good treatments and we need more.”

The FDA will accept public comments until May 10, 2021, at which time comments will be compiled into a summary report. FDA officials assured patients that the report would be visible and circulated not only within the FDA but among drug companies, researchers, and other product developers.

Patients with vitiligo have faced significant impacts psychosocially and in many cases, profound losses of identity – and they’ve had only minimal success with treatment, according to participants who spoke at and provided input at a public meeting on patient-focused drug development for the disease.

FG Trade/E+

The virtual meeting, held in March, was part of the Food and Drug Administration’s Patient-Focused Drug Development (PFDD) initiative, which began in 2012 and aims to provide a systematic way for patients’ experiences, needs and priorities to be “captured and meaningfully incorporated” into drug development and evaluation.

Seemal Desai, MD, of the department of dermatology at the University of Texas, Dallas, who attended the meeting as an observer, said in a later interview that while “all skin diseases have a psychosocial component … vitiligo is a really unique one, because it really relates to the patient’s own identity.

“What I heard loud and clear from the FDA [leaders who ran and attended the meeting] is recognition that patients are suffering. They needed to hear about the emotional devastation of the disease and how it is a medical condition,” Dr. Desai said.

The meeting was the “first-ever vitiligo meeting at the FDA” and was a “historic moment for the vitiligo community,” he added.

The pigmentation disorder affects 1% of the world’s population. Nearly 50% have an onset before age 20, and onset before age 12 is common, Brenda Carr, MD, medical officer with the FDA’s Division of Dermatology and Dentistry in the Center for Drug Evaluation and Research, said in an introductory overview.

The only FDA-approved treatment for vitiligo is monobenzone cream, but this is indicated for final depigmentation in extensive vitiligo and is no longer marketed. Treatment options include corticosteroids, calcineurin inhibitors, vitamin D analogues, phototherapy, surgical treatments (tissue grafts and cellular grafts), and camouflage (make-up, tattoos, self-tanning products), Dr. Carr said.

Patients participated in one of two panels – one about the health effects and daily impacts of vitiligo and the other about treatments – or submitted input electronically. All patients were invited to answer poll questions and open-ended queries, including questions about how they would assess new treatments.

Several panel members who are Black shared series of photos that showed the evolution of defined white patches into widespread, generalized depigmentation. One man with skin of color who lives in the Netherlands said he has had vitiligo since the age of 12, but that when he became older, over a 4-year period, he was “transformed from a man of Indonesian roots to a totally white man.”

Experiencing only minimal benefit from treatment and the short-term effectiveness of treatments were the top two answers to a poll question asking participants about the most burdensome impacts of the medical products and interventions they have used. Difficulty in accessing treatment, concern about serious risks of treatment, and uncertainty about long-term effects of treatment were other frequently chosen answers.

Patients described the onerous nature of phototherapy (treatments repeated several times a week over long periods) and other treatments, and several described feeling that some physicians did not take the condition seriously or fully know of treatment options.

In her closing remarks, Kendall Marcus, MD, director of the Division of Dermatology and Dentistry at the FDA, acknowledged the input. “Some of you have had difficulty having your disease taken seriously by physicians who view it as a cosmetic condition and are reluctant to treat because they believe your expectations will not be met, that it will be an exercise in frustration,” she said.

Regarding the impacts of treatments that have been utilized, “some of the treatments make it impossible to do other activities such as work or care for yourself in other ways,” Dr. Marcus said. “Certainly that’s not the kind of treatment … that anybody wants to have.”

Dr. Desai, who utilizes an array of oral and topical treatments and phototherapies in his practice, said he was surprised and disheartened to hear the level of concern about side effects of treatment. Most of those who expressed concerns alluded to phototherapy. “I think light treatments are very safe and effective,” he said in the interview. “I might equate [such concerns] to the older PUVA [psoralen plus UVA ultraviolet light] therapy, but not so much the newer therapies.”

The FDA participants probed patients for their perspective on a meaningful level of repigmentation and an acceptable level of risk for any new hypothetical treatment. Specifically, they asked whether patients would use a new topical cream approved for vitiligo if the cream needed to be applied once a day, would have up to 50% efficacy in some people, and would have common side effects of redness and irritation at the application site, mild acne, and burning, as well as several rarer but more serious side effects.

Only 36% answered yes; 24% said no, and 40% answered maybe. Some patients said during the meeting that they had accepted their condition and were not pursuing any treatment. Others said they were very interested in treatment but only if the level of repigmentation were significantly higher than 50%. Some described their fear that positive treatment effects would be short term only.

Meri Izrail Kohen, who lives in France and has lost half of her skin’s pigmentation, said that treatment efficacy is “not only about how much recovery of pigment it allows, but how long the recovery will last.” Some treatments will work for some patients, she said, “but even in these cases when we stop the treatment, it will come back somehow.”

Lee Thomas, a TV anchor in Detroit, and a reporter and author of the book “Turning White,” described how he tried “every treatment he could afford” but stopped trying 10 years ago. A treatment in Germany “gave me 80% of my pigment back, but it has gone again,” he said. “I would love to have my face back again. I was born a Black child, and I’d like to die a Black man.”

Patients also spoke of their skin burning easily outdoors; skin sensitivity, itchiness, and burning with the spread of disease; treatment expenses and not being able to afford treatment; and worsening of their vitiligo with the stress of the pandemic. Parents expressed having fear that their children would develop vitiligo and experience bullying, isolation, or other emotional or psychosocial impacts that they had experienced; one described having an almost-paralyzing anxiety when he saw patchy white spots on his 20-month-old daughter (it was not diagnosed as vitiligo).

Calls for further advancement with home phototherapy – which Dr. Desai said is a growing market but not yet adequately covered by insurance plans – were also made, as were pleas for research on the root causes of the disease.

Patients clearly indicated “that they need more efficacious treatments, and more comprehensive treatments,” said Dr. Desai, who chairs the advisory committee of the Global Vitiligo Foundation. “It’s disappointing to me that patients come in with a not fully optimistic viewpoint, with a lot of anxiety and angst that treatments are not going to work. … But the Agency needs to hear that. This means that there haven’t been good treatments and we need more.”

The FDA will accept public comments until May 10, 2021, at which time comments will be compiled into a summary report. FDA officials assured patients that the report would be visible and circulated not only within the FDA but among drug companies, researchers, and other product developers.