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FDA Expands Dostarlimab-gxly Approval for Endometrial Cancer
Prior FDA approval of the combination was granted for adults with primary advanced or recurrent endometrial cancer that was mismatch repair deficient (dMMR) or microsatellite instability–high (MSI-H).
The expanded approval, granted following a priority review, was based on efficacy and safety demonstrated in the randomized, controlled, multicenter RUBY trial, which included 494 patients who were randomized to receive the dostarlimab-gxly regimen or placebo plus carboplatin and paclitaxel, followed by placebo.
Researchers observed a significant improvement in median overall survival with treatment vs placebo in the overall population — 44.6 vs 28.2 months, respectively (hazard ratio [HR], 0.69). Median progression-free survival was also significantly better in the treatment vs placebo group — 11.8 vs 7.9 months, respectively (HR, 0.64).
“Today’s expanded approval will offer even more patients the opportunity for improved outcomes,” Matthew Powell, MD, of Washington University School of Medicine, and principal investigator on the RUBY trial, said in a press release. “This is the only immuno-oncology treatment regimen that has shown a statistically significant overall survival benefit for the full patient population, which is a meaningful step forward in treating this challenging cancer.”
Adverse reactions occurring in at least 20% of patients receiving dostarlimab-gxly include anemia, increased creatinine levels, peripheral neuropathy, decreased white blood cell counts, fatigue, nausea, alopecia, low platelet counts, increased glucose levels, lymphopenia, neutropenia, liver function test abnormalities, arthralgia, rash, constipation, diarrhea, decreased albumin levels, abdominal pain, dyspnea, decreased appetite, increased amylase levels, urinary tract infection, and vomiting. Immune-mediated adverse reactions with dostarlimab-gxly were similar to those previously reported.
The recommended dostarlimab-gxly dose, according to the full prescribing information, is 500 mg every 3 weeks for six cycles administered before carboplatin and paclitaxel if given on the same day, followed by 1000 mg monotherapy every 6 weeks until disease progression or unacceptable toxicity, or up to 3 years.
A version of this article first appeared on Medscape.com.
Prior FDA approval of the combination was granted for adults with primary advanced or recurrent endometrial cancer that was mismatch repair deficient (dMMR) or microsatellite instability–high (MSI-H).
The expanded approval, granted following a priority review, was based on efficacy and safety demonstrated in the randomized, controlled, multicenter RUBY trial, which included 494 patients who were randomized to receive the dostarlimab-gxly regimen or placebo plus carboplatin and paclitaxel, followed by placebo.
Researchers observed a significant improvement in median overall survival with treatment vs placebo in the overall population — 44.6 vs 28.2 months, respectively (hazard ratio [HR], 0.69). Median progression-free survival was also significantly better in the treatment vs placebo group — 11.8 vs 7.9 months, respectively (HR, 0.64).
“Today’s expanded approval will offer even more patients the opportunity for improved outcomes,” Matthew Powell, MD, of Washington University School of Medicine, and principal investigator on the RUBY trial, said in a press release. “This is the only immuno-oncology treatment regimen that has shown a statistically significant overall survival benefit for the full patient population, which is a meaningful step forward in treating this challenging cancer.”
Adverse reactions occurring in at least 20% of patients receiving dostarlimab-gxly include anemia, increased creatinine levels, peripheral neuropathy, decreased white blood cell counts, fatigue, nausea, alopecia, low platelet counts, increased glucose levels, lymphopenia, neutropenia, liver function test abnormalities, arthralgia, rash, constipation, diarrhea, decreased albumin levels, abdominal pain, dyspnea, decreased appetite, increased amylase levels, urinary tract infection, and vomiting. Immune-mediated adverse reactions with dostarlimab-gxly were similar to those previously reported.
The recommended dostarlimab-gxly dose, according to the full prescribing information, is 500 mg every 3 weeks for six cycles administered before carboplatin and paclitaxel if given on the same day, followed by 1000 mg monotherapy every 6 weeks until disease progression or unacceptable toxicity, or up to 3 years.
A version of this article first appeared on Medscape.com.
Prior FDA approval of the combination was granted for adults with primary advanced or recurrent endometrial cancer that was mismatch repair deficient (dMMR) or microsatellite instability–high (MSI-H).
The expanded approval, granted following a priority review, was based on efficacy and safety demonstrated in the randomized, controlled, multicenter RUBY trial, which included 494 patients who were randomized to receive the dostarlimab-gxly regimen or placebo plus carboplatin and paclitaxel, followed by placebo.
Researchers observed a significant improvement in median overall survival with treatment vs placebo in the overall population — 44.6 vs 28.2 months, respectively (hazard ratio [HR], 0.69). Median progression-free survival was also significantly better in the treatment vs placebo group — 11.8 vs 7.9 months, respectively (HR, 0.64).
“Today’s expanded approval will offer even more patients the opportunity for improved outcomes,” Matthew Powell, MD, of Washington University School of Medicine, and principal investigator on the RUBY trial, said in a press release. “This is the only immuno-oncology treatment regimen that has shown a statistically significant overall survival benefit for the full patient population, which is a meaningful step forward in treating this challenging cancer.”
Adverse reactions occurring in at least 20% of patients receiving dostarlimab-gxly include anemia, increased creatinine levels, peripheral neuropathy, decreased white blood cell counts, fatigue, nausea, alopecia, low platelet counts, increased glucose levels, lymphopenia, neutropenia, liver function test abnormalities, arthralgia, rash, constipation, diarrhea, decreased albumin levels, abdominal pain, dyspnea, decreased appetite, increased amylase levels, urinary tract infection, and vomiting. Immune-mediated adverse reactions with dostarlimab-gxly were similar to those previously reported.
The recommended dostarlimab-gxly dose, according to the full prescribing information, is 500 mg every 3 weeks for six cycles administered before carboplatin and paclitaxel if given on the same day, followed by 1000 mg monotherapy every 6 weeks until disease progression or unacceptable toxicity, or up to 3 years.
A version of this article first appeared on Medscape.com.
FDA Expands Darzalex Faspro Indication in Myeloma
Approval followed priority review and was based on efficacy and safety findings from the open-label PERSEUS trial involving 709 patients under age 70 years who were randomized to receive bortezomib, lenalidomide, and dexamethasone alone or in combination with daratumumab and hyaluronidase-fihj, according to the FDA.
Compared with bortezomib, lenalidomide, and dexamethasone alone, the addition of daratumumab and hyaluronidase-fihj resulted in a 60% reduction in the risk for disease progression or death (hazard ratio, 0.40). Median progression-free survival was not reached in either group.
Adverse reactions occurring in ≥ 20% of patients were peripheral neuropathy, fatigue, edema, pyrexia, upper respiratory infection, constipation, diarrhea, musculoskeletal pain, insomnia, and rash.
The recommended dosage for this indication is 1800 mg daratumumab and 30,000 units hyaluronidase, according to the full prescribing information.
Daratumumab and hyaluronidase-fihj, which was first approved in 2020, has a range of other indications in multiple myeloma.
A version of this article first appeared on Medscape.com.
Approval followed priority review and was based on efficacy and safety findings from the open-label PERSEUS trial involving 709 patients under age 70 years who were randomized to receive bortezomib, lenalidomide, and dexamethasone alone or in combination with daratumumab and hyaluronidase-fihj, according to the FDA.
Compared with bortezomib, lenalidomide, and dexamethasone alone, the addition of daratumumab and hyaluronidase-fihj resulted in a 60% reduction in the risk for disease progression or death (hazard ratio, 0.40). Median progression-free survival was not reached in either group.
Adverse reactions occurring in ≥ 20% of patients were peripheral neuropathy, fatigue, edema, pyrexia, upper respiratory infection, constipation, diarrhea, musculoskeletal pain, insomnia, and rash.
The recommended dosage for this indication is 1800 mg daratumumab and 30,000 units hyaluronidase, according to the full prescribing information.
Daratumumab and hyaluronidase-fihj, which was first approved in 2020, has a range of other indications in multiple myeloma.
A version of this article first appeared on Medscape.com.
Approval followed priority review and was based on efficacy and safety findings from the open-label PERSEUS trial involving 709 patients under age 70 years who were randomized to receive bortezomib, lenalidomide, and dexamethasone alone or in combination with daratumumab and hyaluronidase-fihj, according to the FDA.
Compared with bortezomib, lenalidomide, and dexamethasone alone, the addition of daratumumab and hyaluronidase-fihj resulted in a 60% reduction in the risk for disease progression or death (hazard ratio, 0.40). Median progression-free survival was not reached in either group.
Adverse reactions occurring in ≥ 20% of patients were peripheral neuropathy, fatigue, edema, pyrexia, upper respiratory infection, constipation, diarrhea, musculoskeletal pain, insomnia, and rash.
The recommended dosage for this indication is 1800 mg daratumumab and 30,000 units hyaluronidase, according to the full prescribing information.
Daratumumab and hyaluronidase-fihj, which was first approved in 2020, has a range of other indications in multiple myeloma.
A version of this article first appeared on Medscape.com.
Maternal Obesity Linked to Sudden Infant Death
More than 5% of cases of sudden infant death may be linked to maternal obesity, new research showed.
“When a parent has a child that dies of sudden unexplained infant death [SUID], it’s extremely devastating,” said Jan-Marino Ramirez, PhD, the Zain Nadella Endowed Chair in Pediatric Neurosciences at the University of Washington, Seattle, and director of the Center for Integrative Brain Research at Seattle Children’s Hospital. “And the most devastating problem is that there’s no clear answer. Understanding the mechanisms will help parents understand.”
The study was published online in JAMA Pediatrics.
In the United States, approximately 3500 cases of SUID are reported yearly. After educational campaigns in the 1990s demonstrating safe infant sleep positions, rates of these fatalities dropped but have since plateaued.
Maternal Obesity During Pregnancy
Rates of maternal obesity are increasing globally, and more than half of women of reproductive age are overweight or obese.
“Maternal obesity before pregnancy affects placental development, gene expression, and has long-term implications,” said Patrick Catalano, MD, a professor in residence at the Departments of Reproductive Endocrinology and Obstetrics and Gynecology at Massachusetts General Hospital and Harvard Medical School in Boston.
Maternal obesity is a well-documented risk factor for adverse outcomes of pregnancy including stillbirth, preterm birth, and admission to the neonatal intensive care unit. Swedish researchers in 2014 reported maternal obesity was linked to an increase in infant mortality that increased with body mass index (BMI), but that study did not look specifically at SUID.
For their new study, Dr. Ramirez and colleagues looked at data from all live births in the United States from 2015 to 2019 recorded by the Centers for Disease Control and Prevention and the National Center for Health Statistics. Of the 18,857,694 live births occurring at 28 weeks of gestation or later, 16,545 infants died of a sudden, unexplained cause.
Rates of SUID in babies born to mothers with obesity increased in a statistically significant, dose-dependent manner relative to normal weight mothers. The unadjusted absolute risks for SUID were 0.74 cases per 1000 births for normal weight mothers, 0.99 cases at BMIs between 30 and 35, 1.17 cases at BMIs between 35 and 40, and 1.47 instances at BMI ≥ 40.
After adjustment for maternal age, race, ethnicity, and level of education, the adjusted odds ratio for a case of SUID was 1.39 among women with the highest levels of obesity (95% CI, 1.31-1.47), according to the researchers.
While the study revealed an association between maternal obesity and SUID, the basis for this connection remains unknown, the investigators noted. One possibility for the link is that obesity increases the risk for obstructive sleep apnea, which can result in intermittent hypoxia. That, in turn, causes oxidative stress, which may possibly have effects on the fetus causing effects that eventually lead to SUID in the infant.
An accompanying editorial by Jacqueline Maya, MD; Marie-France Hivert, MD, MMSc; and Lydia Shook, MD, from the Massachusetts General Hospital and Harvard Medical School, suggested that the SUID is unlikely directly influenced by high maternal BMI but rather by the metabolic concerns related to obesity such as inflammation, insulin resistance, and abnormal lipid metabolism. Epigenetics may also play a role.
“We believe the evidence for this study of an association between prepregnancy obesity and SUID is a call to action for the scientific and medical community to better understand the complex interplay of biological, social, and behavioral factors that may lead to SUID, a devastating complication that no family should experience,” the authors of the editorial wrote.
Dr. Ramirez stressed the importance of not initiating guilt because there are many factors in SUID such as genetics that cannot be controlled.
“We are far from saying a baby died because you were obese; that’s an important message to parents,” he said. What he sees as important, rather, is using this new research to elucidate further mechanisms that may allow for more targeted interventions: “If we discover that it’s due to, for example, sleep apnea, that’s something we can prevent.”
The researchers reported no conflicts of interest.
A version of this article first appeared on Medscape.com.
More than 5% of cases of sudden infant death may be linked to maternal obesity, new research showed.
“When a parent has a child that dies of sudden unexplained infant death [SUID], it’s extremely devastating,” said Jan-Marino Ramirez, PhD, the Zain Nadella Endowed Chair in Pediatric Neurosciences at the University of Washington, Seattle, and director of the Center for Integrative Brain Research at Seattle Children’s Hospital. “And the most devastating problem is that there’s no clear answer. Understanding the mechanisms will help parents understand.”
The study was published online in JAMA Pediatrics.
In the United States, approximately 3500 cases of SUID are reported yearly. After educational campaigns in the 1990s demonstrating safe infant sleep positions, rates of these fatalities dropped but have since plateaued.
Maternal Obesity During Pregnancy
Rates of maternal obesity are increasing globally, and more than half of women of reproductive age are overweight or obese.
“Maternal obesity before pregnancy affects placental development, gene expression, and has long-term implications,” said Patrick Catalano, MD, a professor in residence at the Departments of Reproductive Endocrinology and Obstetrics and Gynecology at Massachusetts General Hospital and Harvard Medical School in Boston.
Maternal obesity is a well-documented risk factor for adverse outcomes of pregnancy including stillbirth, preterm birth, and admission to the neonatal intensive care unit. Swedish researchers in 2014 reported maternal obesity was linked to an increase in infant mortality that increased with body mass index (BMI), but that study did not look specifically at SUID.
For their new study, Dr. Ramirez and colleagues looked at data from all live births in the United States from 2015 to 2019 recorded by the Centers for Disease Control and Prevention and the National Center for Health Statistics. Of the 18,857,694 live births occurring at 28 weeks of gestation or later, 16,545 infants died of a sudden, unexplained cause.
Rates of SUID in babies born to mothers with obesity increased in a statistically significant, dose-dependent manner relative to normal weight mothers. The unadjusted absolute risks for SUID were 0.74 cases per 1000 births for normal weight mothers, 0.99 cases at BMIs between 30 and 35, 1.17 cases at BMIs between 35 and 40, and 1.47 instances at BMI ≥ 40.
After adjustment for maternal age, race, ethnicity, and level of education, the adjusted odds ratio for a case of SUID was 1.39 among women with the highest levels of obesity (95% CI, 1.31-1.47), according to the researchers.
While the study revealed an association between maternal obesity and SUID, the basis for this connection remains unknown, the investigators noted. One possibility for the link is that obesity increases the risk for obstructive sleep apnea, which can result in intermittent hypoxia. That, in turn, causes oxidative stress, which may possibly have effects on the fetus causing effects that eventually lead to SUID in the infant.
An accompanying editorial by Jacqueline Maya, MD; Marie-France Hivert, MD, MMSc; and Lydia Shook, MD, from the Massachusetts General Hospital and Harvard Medical School, suggested that the SUID is unlikely directly influenced by high maternal BMI but rather by the metabolic concerns related to obesity such as inflammation, insulin resistance, and abnormal lipid metabolism. Epigenetics may also play a role.
“We believe the evidence for this study of an association between prepregnancy obesity and SUID is a call to action for the scientific and medical community to better understand the complex interplay of biological, social, and behavioral factors that may lead to SUID, a devastating complication that no family should experience,” the authors of the editorial wrote.
Dr. Ramirez stressed the importance of not initiating guilt because there are many factors in SUID such as genetics that cannot be controlled.
“We are far from saying a baby died because you were obese; that’s an important message to parents,” he said. What he sees as important, rather, is using this new research to elucidate further mechanisms that may allow for more targeted interventions: “If we discover that it’s due to, for example, sleep apnea, that’s something we can prevent.”
The researchers reported no conflicts of interest.
A version of this article first appeared on Medscape.com.
More than 5% of cases of sudden infant death may be linked to maternal obesity, new research showed.
“When a parent has a child that dies of sudden unexplained infant death [SUID], it’s extremely devastating,” said Jan-Marino Ramirez, PhD, the Zain Nadella Endowed Chair in Pediatric Neurosciences at the University of Washington, Seattle, and director of the Center for Integrative Brain Research at Seattle Children’s Hospital. “And the most devastating problem is that there’s no clear answer. Understanding the mechanisms will help parents understand.”
The study was published online in JAMA Pediatrics.
In the United States, approximately 3500 cases of SUID are reported yearly. After educational campaigns in the 1990s demonstrating safe infant sleep positions, rates of these fatalities dropped but have since plateaued.
Maternal Obesity During Pregnancy
Rates of maternal obesity are increasing globally, and more than half of women of reproductive age are overweight or obese.
“Maternal obesity before pregnancy affects placental development, gene expression, and has long-term implications,” said Patrick Catalano, MD, a professor in residence at the Departments of Reproductive Endocrinology and Obstetrics and Gynecology at Massachusetts General Hospital and Harvard Medical School in Boston.
Maternal obesity is a well-documented risk factor for adverse outcomes of pregnancy including stillbirth, preterm birth, and admission to the neonatal intensive care unit. Swedish researchers in 2014 reported maternal obesity was linked to an increase in infant mortality that increased with body mass index (BMI), but that study did not look specifically at SUID.
For their new study, Dr. Ramirez and colleagues looked at data from all live births in the United States from 2015 to 2019 recorded by the Centers for Disease Control and Prevention and the National Center for Health Statistics. Of the 18,857,694 live births occurring at 28 weeks of gestation or later, 16,545 infants died of a sudden, unexplained cause.
Rates of SUID in babies born to mothers with obesity increased in a statistically significant, dose-dependent manner relative to normal weight mothers. The unadjusted absolute risks for SUID were 0.74 cases per 1000 births for normal weight mothers, 0.99 cases at BMIs between 30 and 35, 1.17 cases at BMIs between 35 and 40, and 1.47 instances at BMI ≥ 40.
After adjustment for maternal age, race, ethnicity, and level of education, the adjusted odds ratio for a case of SUID was 1.39 among women with the highest levels of obesity (95% CI, 1.31-1.47), according to the researchers.
While the study revealed an association between maternal obesity and SUID, the basis for this connection remains unknown, the investigators noted. One possibility for the link is that obesity increases the risk for obstructive sleep apnea, which can result in intermittent hypoxia. That, in turn, causes oxidative stress, which may possibly have effects on the fetus causing effects that eventually lead to SUID in the infant.
An accompanying editorial by Jacqueline Maya, MD; Marie-France Hivert, MD, MMSc; and Lydia Shook, MD, from the Massachusetts General Hospital and Harvard Medical School, suggested that the SUID is unlikely directly influenced by high maternal BMI but rather by the metabolic concerns related to obesity such as inflammation, insulin resistance, and abnormal lipid metabolism. Epigenetics may also play a role.
“We believe the evidence for this study of an association between prepregnancy obesity and SUID is a call to action for the scientific and medical community to better understand the complex interplay of biological, social, and behavioral factors that may lead to SUID, a devastating complication that no family should experience,” the authors of the editorial wrote.
Dr. Ramirez stressed the importance of not initiating guilt because there are many factors in SUID such as genetics that cannot be controlled.
“We are far from saying a baby died because you were obese; that’s an important message to parents,” he said. What he sees as important, rather, is using this new research to elucidate further mechanisms that may allow for more targeted interventions: “If we discover that it’s due to, for example, sleep apnea, that’s something we can prevent.”
The researchers reported no conflicts of interest.
A version of this article first appeared on Medscape.com.
FROM JAMA PEDIATRICS
New Drugs Could Reduce AMD Treatment Burden
STOCKHOLM — Current treatments for age-related macular degeneration (AMD) have proved effective and safe. However, these lifelong therapies involve frequent ocular injections. “It can be nerve-wracking for patients about to embark on this journey,” Lisa Olmos de Koo, MD, an ophthalmologist at the University of Washington Eye Institute at Harborview, Seattle, told this news organization.
At the American Society of Retina Specialists (ASRS) 2024 annual meeting, researchers from around the world presented results from clinical studies aiming at reducing the burden of AMD treatment by:
- Identifying patients at a higher risk for degeneration and vision loss who will be more likely to respond to treatment
- Developing gene therapies that promise to drastically reduce or eliminate the need for injections
- Testing novel drugs with mechanisms of action that use different pathways than currently available medications, offering patients more options and longer-lasting treatments
“It’s exciting to see the broad range of novel approaches in AMD treatments,” Dimitra Skondra, MD, PhD, a retina specialist at the University of Chicago, told this news organization.
Whom to Treat
Anti–vascular endothelial growth factor (anti-VEGF) therapies shook the AMD treatment scene when they were introduced in the early 2000s. “It was incredible,” Dr. Olmos de Koo said. Patients with wet AMD could finally see their vision improve with each injection. “It was a great motivator to begin therapy.”
However, patients with the advanced form of dry AMD involving geographic atrophy (GA) have had less luck. Pegcetacoplan and avacincaptad pegol, the only US Food and Drug Administration (FDA)–approved treatments for GA, slow the progression of the disease but do not restore vision. In fact, vision continues to decline. “Patients want to understand if their condition is worsening and whether treatment is necessary,” Dr. Olmos de Koo said.
Researchers are developing tools to help clinicians identify lesions that are more likely to grow and reach the fovea, causing vision loss.
For example, Cleveland Clinic’s Katherine Talcott, MD, presented an analysis of the GATHER1 and GATHER2 clinical trials that showed that spectral domain optical coherence tomography can be used to examine the integrity of the ellipsoid zone for predicting GA growth and treatment response. The retina’s ellipsoid zone contains densely packed mitochondria within the inner segments of the photoreceptor cells and plays a critical role in visual function.
Dr. Talcott and her team found that more severe baseline damage of the ellipsoid zone was associated with a faster growth rate of GA.
Another analysis of the same trials, presented by Dilraj Grewal, MD, associate professor of ophthalmology, vitreoretinal surgery, and uveitis at Duke Eye Center, Durham, North Carolina, showed that intravitreal administration of avacincaptad pegol efficiently reduced GA growth whether the treated eye developed macular neovascularization or not. Avacincaptad pegol is a complement factor inhibitor that aims to reduce complement-mediated inflammation and tissue damage in the retina.
Dr. Olmos de Koo explained that clinical trials have shown that more patients develop neovascularization when treated for dry GA than they would if left untreated. This has raised the question among clinicians whether the increased risk is a valid reason to avoid treatment. “This useful analysis tells us that there is still a rationale to continue treating GA, even while you’re concurrently treating the wet component with anti-VEGF therapies,” she said.
Another biomarker of GA growth is the position of the lesion at baseline. Daniel Muth, MD, an ophthalmology consultant at the Karolinska Institutet in Stockholm, Sweden, reported the results from a long-term, retrospective analysis of fundus autofluorescence in patients with GA. His semiautomated artificial intelligence–based analysis showed that patients affected bilaterally, but whose fovea was not yet affected, exhibited a faster GA growth rate than fovea-involving patients, with an approximate 15% risk for fovea involvement.
“Those patients whose atrophy has not yet affected the very center are the most likely to benefit from preventive therapy,” Dr. Olmos de Koo said. “Left untreated, a large proportion of them will develop atrophy that does affect their central vision — that’s their reading or facial recognition ability.”
“Potential predictors of rapid growth rates guide us clinically and allow patients to make more informed decisions about whether to pursue treatments that require frequent interventions,” Dr. Olmos de Koo said.
Forecasting the side to which the cost-benefit balance of treatment will tip for each patient is a complex decision-making process, she explained. “A patient is not a statistic, but these predictive studies are one important piece of the pie.”
The Promise of Gene Therapy
The one-and-done promise of gene therapy could rattle the field once again. Trials presented at the ASRS24 showed a drastic reduction (from 85% to 95%) in the number of anti-VEGF and complement treatments needed following gene therapy injection, improving patient vision while relieving them from the stress of monthly injections.
But researchers are still debating the optimal corticosteroid regimen that is required for reducing the inflammatory response associated with the administration of gene therapies, especially those that use viral vectors. The main controversy is whether systemic immunosuppression is necessary or if local therapies, such as topical and intravitreal administration, can suffice.
Results presented at the meeting suggest that local therapies alone can be effective, potentially reducing the need for systemic immunosuppression.
The LUNA trial evaluated the efficacy and tolerability of ixoberogene soroparvovec, a therapy that delivers an anti-VEGF gene into the eye. Investigators included various prophylactic regimens, including local corticosteroids with and without oral prednisone. They found that local corticosteroid therapy alone effectively reduced inflammation.
Biopharma company 4DMT conducted the PRISM study, which examined a dual transgene therapy for neovascular AMD. Patients in this trial received a 20-week topical steroid taper. Only one patient (of 39) required a 6-week extension of steroid therapy. No patients experienced clinically significant intraocular inflammation, indicating that local corticosteroid therapy was effective in managing immune responses.
Currently, gene therapy clinical trials are designed for patients who have failed standard therapy or require frequent injections. “Once we figure out possible long-term side effects and how to deal with inflammation, [gene therapy] could reach many more patients,” Dr. Olmos de Koo said.
New Approaches Enter Pipeline
While gene therapy brings excitement to the field, it might not be for everyone, experts agreed at the ASRS24. New agents are being evaluated to offer a broader range of treatment options with longer-lasting efficacy. Results from early-phase trials presented at the meeting show favorable safety and efficacy signals.
“Finally, after a long time, we have a lot of exciting drugs for geographic atrophy in the pipeline that seem to be safe, with many studies also showing a functional outcome in addition to anatomical outcome,” Dr. Skondra told this news organization.
Current FDA-approved treatments for GA focus on inhibiting the humoral arm of the immune system through C3 and C5 inhibitors. However, a new approach targets both the humoral and cellular arms of the immune response by inhibiting macrophages that release pro-inflammatory cytokines. The goal is to convert these macrophages to a “resolution state,” potentially reducing the release of inflammatory cytokines and offering a more comprehensive treatment for wet and dry AMD, said Rishi Singh, MD, a retina surgeon at the Cole Eye Institute, Cleveland Clinic.
AVD-104, a sialic acid–coated nanoparticle developed by Aviceda Therapeutics, is a promising candidate in this approach. This 100-nm-in-diameter particle, which is only as heavy as 20 hydrogen atoms, is designed for better tissue penetration and has a pharmacokinetic profile lasting 3-4 months after a single intravitreal dose.
AVD-104 aims to repolarize macrophages into a resolution phenotype and decreases complement factor overamplification through direct binding to complement factor H, which downregulates C3 production in immune cells. This dual-action approach could offer a more effective and long-lasting treatment option.
Dr. Singh, who presented the phase 2/3 SIGLEC clinical trial assessing AVD-104, said a single dose resulted in significantly slower rates of disease progression as early as 1 month post-treatment and a notable decrease in junctional zone hyper-autofluorescence.
In addition, about 40% of patients gained vision, which was unexpected but a pleasant surprise, Dr. Singh said. “This is a small study. I don’t want anyone to walk away with the conclusion that we’ve figured out how to improve visual acuity in GA. But it’s promising.”
Other researchers are tackling GA by focusing on therapies that aim to intervene before the complement system is activated.
ONL1204 is a novel agent designed to inhibit the activation of the tumor necrosis factor FAS receptor, which is activated and upregulated in a disease state and is implicated in multiple cell death and inflammatory pathways.
Multiple preclinical models of AMD have shown that ONL1204 preserves retinal cells and inhibits inflammation by inhibiting the FAS receptor. Phase 1 trial results presented at the meeting showed that ONL1204 was safe and showed strong efficacy signals as early as 6 months after treatment initiation.
“We need to be cautiously optimistic,” Dr. Skondra said. “Larger studies will tell us if these signals are real. But it’s a very exciting time. I’m happy to see different mechanisms of action besides the complement because we can attack the disease from multiple fronts.”
Dr. Grewal declared interests with Eyepoint, Iveric Bio, Regeneron, Alumis, Apellis, DORC, and Genentech. Dr. Muth declared interests with Bayer, Canon, and Roche. Dr. Olmos de Koo declared interests with Alcon and Pixium Vision. Dr. Singh declared interests with Gyroscope, 4DMT, Aviceda, Eyepoint, Alcon, Bausch and Lomb, Novartis, and Regeneron. Dr. Skondra declared interests with Biogen, Iveric Bio, Allergan, and Trinity Health Science. Dr. Talcott declared interests with Bausch and Lomb, Eyepoint, Regeneron, REGENXBIO, Zeiss, Apellis, Genentech, Alimera, Outlook, and Iveric Bio.
A version of this article first appeared on Medscape.com.
STOCKHOLM — Current treatments for age-related macular degeneration (AMD) have proved effective and safe. However, these lifelong therapies involve frequent ocular injections. “It can be nerve-wracking for patients about to embark on this journey,” Lisa Olmos de Koo, MD, an ophthalmologist at the University of Washington Eye Institute at Harborview, Seattle, told this news organization.
At the American Society of Retina Specialists (ASRS) 2024 annual meeting, researchers from around the world presented results from clinical studies aiming at reducing the burden of AMD treatment by:
- Identifying patients at a higher risk for degeneration and vision loss who will be more likely to respond to treatment
- Developing gene therapies that promise to drastically reduce or eliminate the need for injections
- Testing novel drugs with mechanisms of action that use different pathways than currently available medications, offering patients more options and longer-lasting treatments
“It’s exciting to see the broad range of novel approaches in AMD treatments,” Dimitra Skondra, MD, PhD, a retina specialist at the University of Chicago, told this news organization.
Whom to Treat
Anti–vascular endothelial growth factor (anti-VEGF) therapies shook the AMD treatment scene when they were introduced in the early 2000s. “It was incredible,” Dr. Olmos de Koo said. Patients with wet AMD could finally see their vision improve with each injection. “It was a great motivator to begin therapy.”
However, patients with the advanced form of dry AMD involving geographic atrophy (GA) have had less luck. Pegcetacoplan and avacincaptad pegol, the only US Food and Drug Administration (FDA)–approved treatments for GA, slow the progression of the disease but do not restore vision. In fact, vision continues to decline. “Patients want to understand if their condition is worsening and whether treatment is necessary,” Dr. Olmos de Koo said.
Researchers are developing tools to help clinicians identify lesions that are more likely to grow and reach the fovea, causing vision loss.
For example, Cleveland Clinic’s Katherine Talcott, MD, presented an analysis of the GATHER1 and GATHER2 clinical trials that showed that spectral domain optical coherence tomography can be used to examine the integrity of the ellipsoid zone for predicting GA growth and treatment response. The retina’s ellipsoid zone contains densely packed mitochondria within the inner segments of the photoreceptor cells and plays a critical role in visual function.
Dr. Talcott and her team found that more severe baseline damage of the ellipsoid zone was associated with a faster growth rate of GA.
Another analysis of the same trials, presented by Dilraj Grewal, MD, associate professor of ophthalmology, vitreoretinal surgery, and uveitis at Duke Eye Center, Durham, North Carolina, showed that intravitreal administration of avacincaptad pegol efficiently reduced GA growth whether the treated eye developed macular neovascularization or not. Avacincaptad pegol is a complement factor inhibitor that aims to reduce complement-mediated inflammation and tissue damage in the retina.
Dr. Olmos de Koo explained that clinical trials have shown that more patients develop neovascularization when treated for dry GA than they would if left untreated. This has raised the question among clinicians whether the increased risk is a valid reason to avoid treatment. “This useful analysis tells us that there is still a rationale to continue treating GA, even while you’re concurrently treating the wet component with anti-VEGF therapies,” she said.
Another biomarker of GA growth is the position of the lesion at baseline. Daniel Muth, MD, an ophthalmology consultant at the Karolinska Institutet in Stockholm, Sweden, reported the results from a long-term, retrospective analysis of fundus autofluorescence in patients with GA. His semiautomated artificial intelligence–based analysis showed that patients affected bilaterally, but whose fovea was not yet affected, exhibited a faster GA growth rate than fovea-involving patients, with an approximate 15% risk for fovea involvement.
“Those patients whose atrophy has not yet affected the very center are the most likely to benefit from preventive therapy,” Dr. Olmos de Koo said. “Left untreated, a large proportion of them will develop atrophy that does affect their central vision — that’s their reading or facial recognition ability.”
“Potential predictors of rapid growth rates guide us clinically and allow patients to make more informed decisions about whether to pursue treatments that require frequent interventions,” Dr. Olmos de Koo said.
Forecasting the side to which the cost-benefit balance of treatment will tip for each patient is a complex decision-making process, she explained. “A patient is not a statistic, but these predictive studies are one important piece of the pie.”
The Promise of Gene Therapy
The one-and-done promise of gene therapy could rattle the field once again. Trials presented at the ASRS24 showed a drastic reduction (from 85% to 95%) in the number of anti-VEGF and complement treatments needed following gene therapy injection, improving patient vision while relieving them from the stress of monthly injections.
But researchers are still debating the optimal corticosteroid regimen that is required for reducing the inflammatory response associated with the administration of gene therapies, especially those that use viral vectors. The main controversy is whether systemic immunosuppression is necessary or if local therapies, such as topical and intravitreal administration, can suffice.
Results presented at the meeting suggest that local therapies alone can be effective, potentially reducing the need for systemic immunosuppression.
The LUNA trial evaluated the efficacy and tolerability of ixoberogene soroparvovec, a therapy that delivers an anti-VEGF gene into the eye. Investigators included various prophylactic regimens, including local corticosteroids with and without oral prednisone. They found that local corticosteroid therapy alone effectively reduced inflammation.
Biopharma company 4DMT conducted the PRISM study, which examined a dual transgene therapy for neovascular AMD. Patients in this trial received a 20-week topical steroid taper. Only one patient (of 39) required a 6-week extension of steroid therapy. No patients experienced clinically significant intraocular inflammation, indicating that local corticosteroid therapy was effective in managing immune responses.
Currently, gene therapy clinical trials are designed for patients who have failed standard therapy or require frequent injections. “Once we figure out possible long-term side effects and how to deal with inflammation, [gene therapy] could reach many more patients,” Dr. Olmos de Koo said.
New Approaches Enter Pipeline
While gene therapy brings excitement to the field, it might not be for everyone, experts agreed at the ASRS24. New agents are being evaluated to offer a broader range of treatment options with longer-lasting efficacy. Results from early-phase trials presented at the meeting show favorable safety and efficacy signals.
“Finally, after a long time, we have a lot of exciting drugs for geographic atrophy in the pipeline that seem to be safe, with many studies also showing a functional outcome in addition to anatomical outcome,” Dr. Skondra told this news organization.
Current FDA-approved treatments for GA focus on inhibiting the humoral arm of the immune system through C3 and C5 inhibitors. However, a new approach targets both the humoral and cellular arms of the immune response by inhibiting macrophages that release pro-inflammatory cytokines. The goal is to convert these macrophages to a “resolution state,” potentially reducing the release of inflammatory cytokines and offering a more comprehensive treatment for wet and dry AMD, said Rishi Singh, MD, a retina surgeon at the Cole Eye Institute, Cleveland Clinic.
AVD-104, a sialic acid–coated nanoparticle developed by Aviceda Therapeutics, is a promising candidate in this approach. This 100-nm-in-diameter particle, which is only as heavy as 20 hydrogen atoms, is designed for better tissue penetration and has a pharmacokinetic profile lasting 3-4 months after a single intravitreal dose.
AVD-104 aims to repolarize macrophages into a resolution phenotype and decreases complement factor overamplification through direct binding to complement factor H, which downregulates C3 production in immune cells. This dual-action approach could offer a more effective and long-lasting treatment option.
Dr. Singh, who presented the phase 2/3 SIGLEC clinical trial assessing AVD-104, said a single dose resulted in significantly slower rates of disease progression as early as 1 month post-treatment and a notable decrease in junctional zone hyper-autofluorescence.
In addition, about 40% of patients gained vision, which was unexpected but a pleasant surprise, Dr. Singh said. “This is a small study. I don’t want anyone to walk away with the conclusion that we’ve figured out how to improve visual acuity in GA. But it’s promising.”
Other researchers are tackling GA by focusing on therapies that aim to intervene before the complement system is activated.
ONL1204 is a novel agent designed to inhibit the activation of the tumor necrosis factor FAS receptor, which is activated and upregulated in a disease state and is implicated in multiple cell death and inflammatory pathways.
Multiple preclinical models of AMD have shown that ONL1204 preserves retinal cells and inhibits inflammation by inhibiting the FAS receptor. Phase 1 trial results presented at the meeting showed that ONL1204 was safe and showed strong efficacy signals as early as 6 months after treatment initiation.
“We need to be cautiously optimistic,” Dr. Skondra said. “Larger studies will tell us if these signals are real. But it’s a very exciting time. I’m happy to see different mechanisms of action besides the complement because we can attack the disease from multiple fronts.”
Dr. Grewal declared interests with Eyepoint, Iveric Bio, Regeneron, Alumis, Apellis, DORC, and Genentech. Dr. Muth declared interests with Bayer, Canon, and Roche. Dr. Olmos de Koo declared interests with Alcon and Pixium Vision. Dr. Singh declared interests with Gyroscope, 4DMT, Aviceda, Eyepoint, Alcon, Bausch and Lomb, Novartis, and Regeneron. Dr. Skondra declared interests with Biogen, Iveric Bio, Allergan, and Trinity Health Science. Dr. Talcott declared interests with Bausch and Lomb, Eyepoint, Regeneron, REGENXBIO, Zeiss, Apellis, Genentech, Alimera, Outlook, and Iveric Bio.
A version of this article first appeared on Medscape.com.
STOCKHOLM — Current treatments for age-related macular degeneration (AMD) have proved effective and safe. However, these lifelong therapies involve frequent ocular injections. “It can be nerve-wracking for patients about to embark on this journey,” Lisa Olmos de Koo, MD, an ophthalmologist at the University of Washington Eye Institute at Harborview, Seattle, told this news organization.
At the American Society of Retina Specialists (ASRS) 2024 annual meeting, researchers from around the world presented results from clinical studies aiming at reducing the burden of AMD treatment by:
- Identifying patients at a higher risk for degeneration and vision loss who will be more likely to respond to treatment
- Developing gene therapies that promise to drastically reduce or eliminate the need for injections
- Testing novel drugs with mechanisms of action that use different pathways than currently available medications, offering patients more options and longer-lasting treatments
“It’s exciting to see the broad range of novel approaches in AMD treatments,” Dimitra Skondra, MD, PhD, a retina specialist at the University of Chicago, told this news organization.
Whom to Treat
Anti–vascular endothelial growth factor (anti-VEGF) therapies shook the AMD treatment scene when they were introduced in the early 2000s. “It was incredible,” Dr. Olmos de Koo said. Patients with wet AMD could finally see their vision improve with each injection. “It was a great motivator to begin therapy.”
However, patients with the advanced form of dry AMD involving geographic atrophy (GA) have had less luck. Pegcetacoplan and avacincaptad pegol, the only US Food and Drug Administration (FDA)–approved treatments for GA, slow the progression of the disease but do not restore vision. In fact, vision continues to decline. “Patients want to understand if their condition is worsening and whether treatment is necessary,” Dr. Olmos de Koo said.
Researchers are developing tools to help clinicians identify lesions that are more likely to grow and reach the fovea, causing vision loss.
For example, Cleveland Clinic’s Katherine Talcott, MD, presented an analysis of the GATHER1 and GATHER2 clinical trials that showed that spectral domain optical coherence tomography can be used to examine the integrity of the ellipsoid zone for predicting GA growth and treatment response. The retina’s ellipsoid zone contains densely packed mitochondria within the inner segments of the photoreceptor cells and plays a critical role in visual function.
Dr. Talcott and her team found that more severe baseline damage of the ellipsoid zone was associated with a faster growth rate of GA.
Another analysis of the same trials, presented by Dilraj Grewal, MD, associate professor of ophthalmology, vitreoretinal surgery, and uveitis at Duke Eye Center, Durham, North Carolina, showed that intravitreal administration of avacincaptad pegol efficiently reduced GA growth whether the treated eye developed macular neovascularization or not. Avacincaptad pegol is a complement factor inhibitor that aims to reduce complement-mediated inflammation and tissue damage in the retina.
Dr. Olmos de Koo explained that clinical trials have shown that more patients develop neovascularization when treated for dry GA than they would if left untreated. This has raised the question among clinicians whether the increased risk is a valid reason to avoid treatment. “This useful analysis tells us that there is still a rationale to continue treating GA, even while you’re concurrently treating the wet component with anti-VEGF therapies,” she said.
Another biomarker of GA growth is the position of the lesion at baseline. Daniel Muth, MD, an ophthalmology consultant at the Karolinska Institutet in Stockholm, Sweden, reported the results from a long-term, retrospective analysis of fundus autofluorescence in patients with GA. His semiautomated artificial intelligence–based analysis showed that patients affected bilaterally, but whose fovea was not yet affected, exhibited a faster GA growth rate than fovea-involving patients, with an approximate 15% risk for fovea involvement.
“Those patients whose atrophy has not yet affected the very center are the most likely to benefit from preventive therapy,” Dr. Olmos de Koo said. “Left untreated, a large proportion of them will develop atrophy that does affect their central vision — that’s their reading or facial recognition ability.”
“Potential predictors of rapid growth rates guide us clinically and allow patients to make more informed decisions about whether to pursue treatments that require frequent interventions,” Dr. Olmos de Koo said.
Forecasting the side to which the cost-benefit balance of treatment will tip for each patient is a complex decision-making process, she explained. “A patient is not a statistic, but these predictive studies are one important piece of the pie.”
The Promise of Gene Therapy
The one-and-done promise of gene therapy could rattle the field once again. Trials presented at the ASRS24 showed a drastic reduction (from 85% to 95%) in the number of anti-VEGF and complement treatments needed following gene therapy injection, improving patient vision while relieving them from the stress of monthly injections.
But researchers are still debating the optimal corticosteroid regimen that is required for reducing the inflammatory response associated with the administration of gene therapies, especially those that use viral vectors. The main controversy is whether systemic immunosuppression is necessary or if local therapies, such as topical and intravitreal administration, can suffice.
Results presented at the meeting suggest that local therapies alone can be effective, potentially reducing the need for systemic immunosuppression.
The LUNA trial evaluated the efficacy and tolerability of ixoberogene soroparvovec, a therapy that delivers an anti-VEGF gene into the eye. Investigators included various prophylactic regimens, including local corticosteroids with and without oral prednisone. They found that local corticosteroid therapy alone effectively reduced inflammation.
Biopharma company 4DMT conducted the PRISM study, which examined a dual transgene therapy for neovascular AMD. Patients in this trial received a 20-week topical steroid taper. Only one patient (of 39) required a 6-week extension of steroid therapy. No patients experienced clinically significant intraocular inflammation, indicating that local corticosteroid therapy was effective in managing immune responses.
Currently, gene therapy clinical trials are designed for patients who have failed standard therapy or require frequent injections. “Once we figure out possible long-term side effects and how to deal with inflammation, [gene therapy] could reach many more patients,” Dr. Olmos de Koo said.
New Approaches Enter Pipeline
While gene therapy brings excitement to the field, it might not be for everyone, experts agreed at the ASRS24. New agents are being evaluated to offer a broader range of treatment options with longer-lasting efficacy. Results from early-phase trials presented at the meeting show favorable safety and efficacy signals.
“Finally, after a long time, we have a lot of exciting drugs for geographic atrophy in the pipeline that seem to be safe, with many studies also showing a functional outcome in addition to anatomical outcome,” Dr. Skondra told this news organization.
Current FDA-approved treatments for GA focus on inhibiting the humoral arm of the immune system through C3 and C5 inhibitors. However, a new approach targets both the humoral and cellular arms of the immune response by inhibiting macrophages that release pro-inflammatory cytokines. The goal is to convert these macrophages to a “resolution state,” potentially reducing the release of inflammatory cytokines and offering a more comprehensive treatment for wet and dry AMD, said Rishi Singh, MD, a retina surgeon at the Cole Eye Institute, Cleveland Clinic.
AVD-104, a sialic acid–coated nanoparticle developed by Aviceda Therapeutics, is a promising candidate in this approach. This 100-nm-in-diameter particle, which is only as heavy as 20 hydrogen atoms, is designed for better tissue penetration and has a pharmacokinetic profile lasting 3-4 months after a single intravitreal dose.
AVD-104 aims to repolarize macrophages into a resolution phenotype and decreases complement factor overamplification through direct binding to complement factor H, which downregulates C3 production in immune cells. This dual-action approach could offer a more effective and long-lasting treatment option.
Dr. Singh, who presented the phase 2/3 SIGLEC clinical trial assessing AVD-104, said a single dose resulted in significantly slower rates of disease progression as early as 1 month post-treatment and a notable decrease in junctional zone hyper-autofluorescence.
In addition, about 40% of patients gained vision, which was unexpected but a pleasant surprise, Dr. Singh said. “This is a small study. I don’t want anyone to walk away with the conclusion that we’ve figured out how to improve visual acuity in GA. But it’s promising.”
Other researchers are tackling GA by focusing on therapies that aim to intervene before the complement system is activated.
ONL1204 is a novel agent designed to inhibit the activation of the tumor necrosis factor FAS receptor, which is activated and upregulated in a disease state and is implicated in multiple cell death and inflammatory pathways.
Multiple preclinical models of AMD have shown that ONL1204 preserves retinal cells and inhibits inflammation by inhibiting the FAS receptor. Phase 1 trial results presented at the meeting showed that ONL1204 was safe and showed strong efficacy signals as early as 6 months after treatment initiation.
“We need to be cautiously optimistic,” Dr. Skondra said. “Larger studies will tell us if these signals are real. But it’s a very exciting time. I’m happy to see different mechanisms of action besides the complement because we can attack the disease from multiple fronts.”
Dr. Grewal declared interests with Eyepoint, Iveric Bio, Regeneron, Alumis, Apellis, DORC, and Genentech. Dr. Muth declared interests with Bayer, Canon, and Roche. Dr. Olmos de Koo declared interests with Alcon and Pixium Vision. Dr. Singh declared interests with Gyroscope, 4DMT, Aviceda, Eyepoint, Alcon, Bausch and Lomb, Novartis, and Regeneron. Dr. Skondra declared interests with Biogen, Iveric Bio, Allergan, and Trinity Health Science. Dr. Talcott declared interests with Bausch and Lomb, Eyepoint, Regeneron, REGENXBIO, Zeiss, Apellis, Genentech, Alimera, Outlook, and Iveric Bio.
A version of this article first appeared on Medscape.com.
FROM ASRS 2024
Lipedema: Current Diagnostic and Treatment Evidence
Lipedema affects about 11% of cisgender women, according to the Brazilian Society of Angiology and Vascular Surgery. Yet the condition remains wrapped in uncertainties. Despite significant advancements in understanding its physiology, diagnosis, and treatment, more clarity is needed as awareness and diagnoses increase.
At the latest International Congress on Obesity (ICO) in São Paulo, Brazil, Philipp Scherer, PhD, director of the Touchstone Diabetes Center, discussed the complexities of lipedema. “It is an extremely frustrating condition for someone like me, who has spent a lifetime studying functional and dysfunctional adipose tissue. We are trying to understand the physiology of this pathology, but it is challenging, and so far, we have not been able to find a concrete answer,” he noted.
Lipedema is characterized by the abnormal accumulation of subcutaneous adipose tissue, especially in the lower limbs, and almost exclusively affects cisgender women. The reason for this gender disparity is unclear. It could be an intrinsic characteristic of the disease or a result from clinicians’ lack of familiarity with lipedema, which often leads to misdiagnosis as obesity. This misdiagnosis results in fewer men seeking treatment.
Research has predominantly focused on women, and evidence suggests that hormones play a crucial role in the disease’s pathophysiology. Lipedema typically manifests during periods of hormonal changes, such as puberty, pregnancy, menopause, and hormone replacement therapies, reinforcing the idea that hormones significantly influence the condition’s development and progression.
Main Symptoms
Jonathan Kartt, CEO of the Lipedema Foundation, emphasized that intense pain in the areas of adipose tissue accumulation is a hallmark symptom of lipedema, setting it apart from obesity. Pain levels can vary widely among patients, ranging from moderate to severe, with unbearable peaks on certain days. Mr. Kartt stressed the importance of recognizing and addressing this often underestimated symptom.
Lipedema is characterized by a bilateral, symmetrical increase in mass compared with the rest of the body. This is commonly distinguished by the “cuff sign,” a separation between normal tissue in the feet and abnormal tissue from the ankle upward. Other frequent symptoms include a feeling of heaviness, discomfort, fatigue, frequent bruising, and tiredness. A notable sign is the presence of subcutaneous nodules with a texture similar to that of rice grains, which are crucial for differentiating lipedema from other conditions. Palpation during anamnesis is essential to identify these nodules and confirm the diagnosis.
“It is crucial to investigate the family history for genetic predisposition. Additionally, it is fundamental to ask whether, even with weight loss, the affected areas retain accumulated fat. Hormonal changes, pain symptoms, and impact on quality of life should also be carefully evaluated,” advised Mr. Kartt.
Diagnostic Tools
André Murad, MD, a clinical consultant at the Instituto Lipedema Brazil, has been exploring new diagnostic approaches for lipedema beyond traditional anamnesis. During his presentation at the ICO, he shared studies on the efficacy of imaging exams such as ultrasound, tomography, and MRI in diagnosing the characteristic lipedema-associated increase in subcutaneous tissue.
He also discussed lymphangiography and lymphoscintigraphy, highlighting the use of magnetic resonance lymphangiography to evaluate dilated lymphatic vessels often observed in patients with lipedema. “By injecting contrast into the feet, this technique allows the evaluation of vessels, which are usually dilated, indicating characteristic lymphatic system overload in lipedema. Lymphoscintigraphy is crucial for detecting associated lymphedema, revealing delayed lymphatic flow and asymmetry between limbs in cases of lipedema without lymphedema,” he explained.
Despite the various diagnostic options, Dr. Murad highlighted two highly effective studies. A Brazilian study used ultrasound to establish a cutoff point of 11.7 mm in the pretibial subcutaneous tissue thickness, achieving 96% specificity for diagnosis. Another study emphasized the value of dual-energy x-ray absorptiometry (DXA), which demonstrated 95% sensitivity. This method assesses fat distribution by correlating the amount present in the legs with the total body, providing a cost-effective and accessible option for specialists.
“DXA allows for a precise mathematical evaluation of fat distribution relative to the total body. A ratio of 0.38 in the leg-to-body relationship is a significant indicator of high suspicion of lipedema,” highlighted Dr. Murad. “In clinical practice, many patients self-diagnose with lipedema, but the clinical exam often reveals no disproportion, with the leg-to-body ratio below 0.38 being common in these cases,” he added.
Treatment Approaches
Treatments for lipedema are still evolving, with considerable debate about the best approach. While some specialists advocate exclusively for conservative treatment, others recommend combining these methods with surgical interventions, depending on the stage of the disease. The relative novelty of lipedema and the scarcity of robust, long-term studies contribute to the uncertainty around treatment efficacy.
Conservative treatment typically includes compression, lymphatic drainage techniques, and pressure therapy. An active lifestyle and a healthy diet are also recommended. Although these measures do not prevent the accumulation of adipose tissue, they help reduce inflammation and improve quality of life. “Even though the causes of lipedema are not fully known, lifestyle management is essential for controlling symptoms, starting with an anti-inflammatory diet,” emphasized Dr. Murad.
Because insulin promotes lipogenesis, a diet that avoids spikes in glycemic and insulin levels is advisable. Insulin resistance can exacerbate edema formation, so a Mediterranean diet may be beneficial. This diet limits fast-absorbing carbohydrates, such as added sugar, refined grains, and ultraprocessed foods, while promoting complex carbohydrates from whole grains and legumes.
Dr. Murad also presented a study evaluating the potential benefits of a low-carbohydrate, high-fat diet for patients with lipedema. The study demonstrated weight loss, reduced body fat, controlled leg volume, and, notably, pain relief.
For more advanced stages of lipedema, plastic surgery is often considered when conservative approaches do not yield satisfactory results. Some specialists advocate for surgery as an effective way to remove diseased adipose cells and reduce excess fat accumulation, which can improve physical appearance and associated pain. There is a growing consensus that surgical intervention should be performed early, ideally in stage I of IV, to maximize efficacy and prevent disease progression.
Fábio Masato Kamamoto, MD, a plastic surgeon and director of the Instituto Lipedema Brazil, shared insights into surgical treatments for lipedema. He discussed techniques from liposuction to advanced skin retraction and dermolipectomy, crucial for addressing more advanced stages of the condition. “It’s a complex process that demands precision to protect the lymphatic system, especially considering the characteristic nodules of lipedema,” he noted.
Dr. Kamamoto discussed a former patient with stage III lipedema. In the initial stage, he performed liposuction, removing 8 L of fat and 3.4 kg of skin. After 6 months, a follow-up procedure resulted in a total removal of 15 kg. Complementary procedures, such as microneedling, were performed to stimulate collagen production and reduce skin sagging. In addition to cosmetic improvements, the procedure also removed the distinctive lipedema nodules, which Mr. Kartt described as feeling like “rice grains.” Removing these nodules significantly alleviates pain, according to Dr. Kamamoto.
The benefits of surgical treatment for lipedema can be long lasting. Dr. Kamamoto noted that fat tends not to reaccumulate in treated areas, with patients often experiencing lower weight, reduced edema, and decreased pain over time. “While we hope that patients do not regain weight, the benefits of surgery persist even if weight is regained. Therefore, combining conservative and surgical treatments remains a valid and effective approach,” he concluded.
Dr. Scherer highlighted that despite various approaches, there is still no definitive “magic signature” that fully explains lipedema. This lack of clarity directly affects the effectiveness of diagnoses and treatments. He expressed hope that future integration of data from different studies and approaches will lead to the identification of a clinically useful molecular signature. “The true cause of lipedema remains unknown, requiring more speculation, hypothesis formulation, and testing for significant discoveries. This situation is frustrating, as the disease affects many women who lack a clear diagnosis that differentiates them from patients with obesity, as well as evidence-based recommendations,” he concluded.
This story was translated from the Medscape Portuguese edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.
Lipedema affects about 11% of cisgender women, according to the Brazilian Society of Angiology and Vascular Surgery. Yet the condition remains wrapped in uncertainties. Despite significant advancements in understanding its physiology, diagnosis, and treatment, more clarity is needed as awareness and diagnoses increase.
At the latest International Congress on Obesity (ICO) in São Paulo, Brazil, Philipp Scherer, PhD, director of the Touchstone Diabetes Center, discussed the complexities of lipedema. “It is an extremely frustrating condition for someone like me, who has spent a lifetime studying functional and dysfunctional adipose tissue. We are trying to understand the physiology of this pathology, but it is challenging, and so far, we have not been able to find a concrete answer,” he noted.
Lipedema is characterized by the abnormal accumulation of subcutaneous adipose tissue, especially in the lower limbs, and almost exclusively affects cisgender women. The reason for this gender disparity is unclear. It could be an intrinsic characteristic of the disease or a result from clinicians’ lack of familiarity with lipedema, which often leads to misdiagnosis as obesity. This misdiagnosis results in fewer men seeking treatment.
Research has predominantly focused on women, and evidence suggests that hormones play a crucial role in the disease’s pathophysiology. Lipedema typically manifests during periods of hormonal changes, such as puberty, pregnancy, menopause, and hormone replacement therapies, reinforcing the idea that hormones significantly influence the condition’s development and progression.
Main Symptoms
Jonathan Kartt, CEO of the Lipedema Foundation, emphasized that intense pain in the areas of adipose tissue accumulation is a hallmark symptom of lipedema, setting it apart from obesity. Pain levels can vary widely among patients, ranging from moderate to severe, with unbearable peaks on certain days. Mr. Kartt stressed the importance of recognizing and addressing this often underestimated symptom.
Lipedema is characterized by a bilateral, symmetrical increase in mass compared with the rest of the body. This is commonly distinguished by the “cuff sign,” a separation between normal tissue in the feet and abnormal tissue from the ankle upward. Other frequent symptoms include a feeling of heaviness, discomfort, fatigue, frequent bruising, and tiredness. A notable sign is the presence of subcutaneous nodules with a texture similar to that of rice grains, which are crucial for differentiating lipedema from other conditions. Palpation during anamnesis is essential to identify these nodules and confirm the diagnosis.
“It is crucial to investigate the family history for genetic predisposition. Additionally, it is fundamental to ask whether, even with weight loss, the affected areas retain accumulated fat. Hormonal changes, pain symptoms, and impact on quality of life should also be carefully evaluated,” advised Mr. Kartt.
Diagnostic Tools
André Murad, MD, a clinical consultant at the Instituto Lipedema Brazil, has been exploring new diagnostic approaches for lipedema beyond traditional anamnesis. During his presentation at the ICO, he shared studies on the efficacy of imaging exams such as ultrasound, tomography, and MRI in diagnosing the characteristic lipedema-associated increase in subcutaneous tissue.
He also discussed lymphangiography and lymphoscintigraphy, highlighting the use of magnetic resonance lymphangiography to evaluate dilated lymphatic vessels often observed in patients with lipedema. “By injecting contrast into the feet, this technique allows the evaluation of vessels, which are usually dilated, indicating characteristic lymphatic system overload in lipedema. Lymphoscintigraphy is crucial for detecting associated lymphedema, revealing delayed lymphatic flow and asymmetry between limbs in cases of lipedema without lymphedema,” he explained.
Despite the various diagnostic options, Dr. Murad highlighted two highly effective studies. A Brazilian study used ultrasound to establish a cutoff point of 11.7 mm in the pretibial subcutaneous tissue thickness, achieving 96% specificity for diagnosis. Another study emphasized the value of dual-energy x-ray absorptiometry (DXA), which demonstrated 95% sensitivity. This method assesses fat distribution by correlating the amount present in the legs with the total body, providing a cost-effective and accessible option for specialists.
“DXA allows for a precise mathematical evaluation of fat distribution relative to the total body. A ratio of 0.38 in the leg-to-body relationship is a significant indicator of high suspicion of lipedema,” highlighted Dr. Murad. “In clinical practice, many patients self-diagnose with lipedema, but the clinical exam often reveals no disproportion, with the leg-to-body ratio below 0.38 being common in these cases,” he added.
Treatment Approaches
Treatments for lipedema are still evolving, with considerable debate about the best approach. While some specialists advocate exclusively for conservative treatment, others recommend combining these methods with surgical interventions, depending on the stage of the disease. The relative novelty of lipedema and the scarcity of robust, long-term studies contribute to the uncertainty around treatment efficacy.
Conservative treatment typically includes compression, lymphatic drainage techniques, and pressure therapy. An active lifestyle and a healthy diet are also recommended. Although these measures do not prevent the accumulation of adipose tissue, they help reduce inflammation and improve quality of life. “Even though the causes of lipedema are not fully known, lifestyle management is essential for controlling symptoms, starting with an anti-inflammatory diet,” emphasized Dr. Murad.
Because insulin promotes lipogenesis, a diet that avoids spikes in glycemic and insulin levels is advisable. Insulin resistance can exacerbate edema formation, so a Mediterranean diet may be beneficial. This diet limits fast-absorbing carbohydrates, such as added sugar, refined grains, and ultraprocessed foods, while promoting complex carbohydrates from whole grains and legumes.
Dr. Murad also presented a study evaluating the potential benefits of a low-carbohydrate, high-fat diet for patients with lipedema. The study demonstrated weight loss, reduced body fat, controlled leg volume, and, notably, pain relief.
For more advanced stages of lipedema, plastic surgery is often considered when conservative approaches do not yield satisfactory results. Some specialists advocate for surgery as an effective way to remove diseased adipose cells and reduce excess fat accumulation, which can improve physical appearance and associated pain. There is a growing consensus that surgical intervention should be performed early, ideally in stage I of IV, to maximize efficacy and prevent disease progression.
Fábio Masato Kamamoto, MD, a plastic surgeon and director of the Instituto Lipedema Brazil, shared insights into surgical treatments for lipedema. He discussed techniques from liposuction to advanced skin retraction and dermolipectomy, crucial for addressing more advanced stages of the condition. “It’s a complex process that demands precision to protect the lymphatic system, especially considering the characteristic nodules of lipedema,” he noted.
Dr. Kamamoto discussed a former patient with stage III lipedema. In the initial stage, he performed liposuction, removing 8 L of fat and 3.4 kg of skin. After 6 months, a follow-up procedure resulted in a total removal of 15 kg. Complementary procedures, such as microneedling, were performed to stimulate collagen production and reduce skin sagging. In addition to cosmetic improvements, the procedure also removed the distinctive lipedema nodules, which Mr. Kartt described as feeling like “rice grains.” Removing these nodules significantly alleviates pain, according to Dr. Kamamoto.
The benefits of surgical treatment for lipedema can be long lasting. Dr. Kamamoto noted that fat tends not to reaccumulate in treated areas, with patients often experiencing lower weight, reduced edema, and decreased pain over time. “While we hope that patients do not regain weight, the benefits of surgery persist even if weight is regained. Therefore, combining conservative and surgical treatments remains a valid and effective approach,” he concluded.
Dr. Scherer highlighted that despite various approaches, there is still no definitive “magic signature” that fully explains lipedema. This lack of clarity directly affects the effectiveness of diagnoses and treatments. He expressed hope that future integration of data from different studies and approaches will lead to the identification of a clinically useful molecular signature. “The true cause of lipedema remains unknown, requiring more speculation, hypothesis formulation, and testing for significant discoveries. This situation is frustrating, as the disease affects many women who lack a clear diagnosis that differentiates them from patients with obesity, as well as evidence-based recommendations,” he concluded.
This story was translated from the Medscape Portuguese edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.
Lipedema affects about 11% of cisgender women, according to the Brazilian Society of Angiology and Vascular Surgery. Yet the condition remains wrapped in uncertainties. Despite significant advancements in understanding its physiology, diagnosis, and treatment, more clarity is needed as awareness and diagnoses increase.
At the latest International Congress on Obesity (ICO) in São Paulo, Brazil, Philipp Scherer, PhD, director of the Touchstone Diabetes Center, discussed the complexities of lipedema. “It is an extremely frustrating condition for someone like me, who has spent a lifetime studying functional and dysfunctional adipose tissue. We are trying to understand the physiology of this pathology, but it is challenging, and so far, we have not been able to find a concrete answer,” he noted.
Lipedema is characterized by the abnormal accumulation of subcutaneous adipose tissue, especially in the lower limbs, and almost exclusively affects cisgender women. The reason for this gender disparity is unclear. It could be an intrinsic characteristic of the disease or a result from clinicians’ lack of familiarity with lipedema, which often leads to misdiagnosis as obesity. This misdiagnosis results in fewer men seeking treatment.
Research has predominantly focused on women, and evidence suggests that hormones play a crucial role in the disease’s pathophysiology. Lipedema typically manifests during periods of hormonal changes, such as puberty, pregnancy, menopause, and hormone replacement therapies, reinforcing the idea that hormones significantly influence the condition’s development and progression.
Main Symptoms
Jonathan Kartt, CEO of the Lipedema Foundation, emphasized that intense pain in the areas of adipose tissue accumulation is a hallmark symptom of lipedema, setting it apart from obesity. Pain levels can vary widely among patients, ranging from moderate to severe, with unbearable peaks on certain days. Mr. Kartt stressed the importance of recognizing and addressing this often underestimated symptom.
Lipedema is characterized by a bilateral, symmetrical increase in mass compared with the rest of the body. This is commonly distinguished by the “cuff sign,” a separation between normal tissue in the feet and abnormal tissue from the ankle upward. Other frequent symptoms include a feeling of heaviness, discomfort, fatigue, frequent bruising, and tiredness. A notable sign is the presence of subcutaneous nodules with a texture similar to that of rice grains, which are crucial for differentiating lipedema from other conditions. Palpation during anamnesis is essential to identify these nodules and confirm the diagnosis.
“It is crucial to investigate the family history for genetic predisposition. Additionally, it is fundamental to ask whether, even with weight loss, the affected areas retain accumulated fat. Hormonal changes, pain symptoms, and impact on quality of life should also be carefully evaluated,” advised Mr. Kartt.
Diagnostic Tools
André Murad, MD, a clinical consultant at the Instituto Lipedema Brazil, has been exploring new diagnostic approaches for lipedema beyond traditional anamnesis. During his presentation at the ICO, he shared studies on the efficacy of imaging exams such as ultrasound, tomography, and MRI in diagnosing the characteristic lipedema-associated increase in subcutaneous tissue.
He also discussed lymphangiography and lymphoscintigraphy, highlighting the use of magnetic resonance lymphangiography to evaluate dilated lymphatic vessels often observed in patients with lipedema. “By injecting contrast into the feet, this technique allows the evaluation of vessels, which are usually dilated, indicating characteristic lymphatic system overload in lipedema. Lymphoscintigraphy is crucial for detecting associated lymphedema, revealing delayed lymphatic flow and asymmetry between limbs in cases of lipedema without lymphedema,” he explained.
Despite the various diagnostic options, Dr. Murad highlighted two highly effective studies. A Brazilian study used ultrasound to establish a cutoff point of 11.7 mm in the pretibial subcutaneous tissue thickness, achieving 96% specificity for diagnosis. Another study emphasized the value of dual-energy x-ray absorptiometry (DXA), which demonstrated 95% sensitivity. This method assesses fat distribution by correlating the amount present in the legs with the total body, providing a cost-effective and accessible option for specialists.
“DXA allows for a precise mathematical evaluation of fat distribution relative to the total body. A ratio of 0.38 in the leg-to-body relationship is a significant indicator of high suspicion of lipedema,” highlighted Dr. Murad. “In clinical practice, many patients self-diagnose with lipedema, but the clinical exam often reveals no disproportion, with the leg-to-body ratio below 0.38 being common in these cases,” he added.
Treatment Approaches
Treatments for lipedema are still evolving, with considerable debate about the best approach. While some specialists advocate exclusively for conservative treatment, others recommend combining these methods with surgical interventions, depending on the stage of the disease. The relative novelty of lipedema and the scarcity of robust, long-term studies contribute to the uncertainty around treatment efficacy.
Conservative treatment typically includes compression, lymphatic drainage techniques, and pressure therapy. An active lifestyle and a healthy diet are also recommended. Although these measures do not prevent the accumulation of adipose tissue, they help reduce inflammation and improve quality of life. “Even though the causes of lipedema are not fully known, lifestyle management is essential for controlling symptoms, starting with an anti-inflammatory diet,” emphasized Dr. Murad.
Because insulin promotes lipogenesis, a diet that avoids spikes in glycemic and insulin levels is advisable. Insulin resistance can exacerbate edema formation, so a Mediterranean diet may be beneficial. This diet limits fast-absorbing carbohydrates, such as added sugar, refined grains, and ultraprocessed foods, while promoting complex carbohydrates from whole grains and legumes.
Dr. Murad also presented a study evaluating the potential benefits of a low-carbohydrate, high-fat diet for patients with lipedema. The study demonstrated weight loss, reduced body fat, controlled leg volume, and, notably, pain relief.
For more advanced stages of lipedema, plastic surgery is often considered when conservative approaches do not yield satisfactory results. Some specialists advocate for surgery as an effective way to remove diseased adipose cells and reduce excess fat accumulation, which can improve physical appearance and associated pain. There is a growing consensus that surgical intervention should be performed early, ideally in stage I of IV, to maximize efficacy and prevent disease progression.
Fábio Masato Kamamoto, MD, a plastic surgeon and director of the Instituto Lipedema Brazil, shared insights into surgical treatments for lipedema. He discussed techniques from liposuction to advanced skin retraction and dermolipectomy, crucial for addressing more advanced stages of the condition. “It’s a complex process that demands precision to protect the lymphatic system, especially considering the characteristic nodules of lipedema,” he noted.
Dr. Kamamoto discussed a former patient with stage III lipedema. In the initial stage, he performed liposuction, removing 8 L of fat and 3.4 kg of skin. After 6 months, a follow-up procedure resulted in a total removal of 15 kg. Complementary procedures, such as microneedling, were performed to stimulate collagen production and reduce skin sagging. In addition to cosmetic improvements, the procedure also removed the distinctive lipedema nodules, which Mr. Kartt described as feeling like “rice grains.” Removing these nodules significantly alleviates pain, according to Dr. Kamamoto.
The benefits of surgical treatment for lipedema can be long lasting. Dr. Kamamoto noted that fat tends not to reaccumulate in treated areas, with patients often experiencing lower weight, reduced edema, and decreased pain over time. “While we hope that patients do not regain weight, the benefits of surgery persist even if weight is regained. Therefore, combining conservative and surgical treatments remains a valid and effective approach,” he concluded.
Dr. Scherer highlighted that despite various approaches, there is still no definitive “magic signature” that fully explains lipedema. This lack of clarity directly affects the effectiveness of diagnoses and treatments. He expressed hope that future integration of data from different studies and approaches will lead to the identification of a clinically useful molecular signature. “The true cause of lipedema remains unknown, requiring more speculation, hypothesis formulation, and testing for significant discoveries. This situation is frustrating, as the disease affects many women who lack a clear diagnosis that differentiates them from patients with obesity, as well as evidence-based recommendations,” he concluded.
This story was translated from the Medscape Portuguese edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.
CMS Proposes Maternal-Health Conditions-of-Participation Standards
Federal officials intend to compel US hospitals to improve obstetrical services, with a plan that could result in a potential loss of Medicare and Medicaid funds for institutions that fail to comply with the demands.
The Centers for Medicare and Medicaid Services (CMS) on July 10 announced this proposal, tucking its plan for new conditions of participation (COP) for obstetrician services into the draft 2025 rule on Medicare payments for outpatient hospital services.
The COP requirements are considered the most powerful tool CMS has for trying to improve the quality of medical care. With the new obstetric COP requirement, CMS said it intends to address what it sees as potential shortfalls in training, staffing, transfer protocols, and emergency services readiness.
In practice, hospitals, CMS, and accrediting bodies such as the Joint Commission usually try to address deficiencies to prevent what would be a devastating financial loss for a hospital.
“CMS is using all of our tools to improve the safety, quality, and timeliness of the care that hospitals provide to pregnant women,” Dora Hughes, MD, MPH, acting chief medical officer of the agency, said in a press release about the proposal.
CMS estimated the proposal may add new annual expenses of $70,671 per hospital. For comparison, this figure would represent far less than 1% of the total $1.4 trillion spent on hospital care in the United States in 2022.
CMS said it is trying to address the reasons women in the United States face more risk in giving birth than those in other nations. There were 22 maternal deaths for every 100,000 live births in this country in 2022, compared with 8.6 deaths per 100,000 live births or lower that year in Canada, France, the United Kingdom, Germany, and Japan, CMS said.
But CMS is seeking to impose this new requirement at a time amid growing concerns about “maternity care deserts.”
Reasonable Asks?
Between 2011 and 2021, one out of every four rural hospitals in America stopped providing obstetrics services, Senate Finance Chairman Ron Wyden (D-Ore.) said at a May hearing. Mr. Wyden last year was part of a fight to try to prevent the closure of a birthing center in Baker City in rural eastern Oregon.
The federal government should focus first on helping hospitals keep obstetrical facilities open, said Elizabeth Powers, MD, MHA, the health services officer of the Winding Waters Clinic in Enterprise, Oregon.
“Until we can ensure access to services, we can’t even work on quality,” Dr. Powers told this news organization. “If you’re thinking about a Maslow’s hierarchy of achieving health outcomes, access is your foundation, and without a shift in payment, that foundation is eroded.”
In the draft rule, CMS sketched broad mandates about staffing and training. For example, the agency proposes requiring if a hospital offers obstetrical services, “the services must be well organized and provided in accordance with nationally recognized acceptable standards of practice.”
That means CMS likely will need to provide further guidance for hospitals if it proceeds with this plan for obstetric COP requirements, said Soumi Saha, PharmD, JD, senior vice president of government affairs at Premier Inc., a healthcare consultancy and purchasing organization.
Premier is among the many groups, including the American Hospital Association, that oppose the COP proposal.
Dr. Saha said a better approach would be to consolidate the work being done through the US Department of Health and Human Services (HHS), including earlier CMS projects, to address maternal health in a cohesive way. The Centers for Disease Control and Prevention has programs, as does the HHS Office on Women’s Health.
“How do we really get to a holistic, national, unified approach to addressing this issue that is led by HHS at the top level as the top agency and trickles down consistently versus having all of these kinds of disparate programs in place?” she said.
In recent years, the federal and state governments have taken many steps to try to improve maternal healthcare.
These include the extension of Medicaid benefits to new mothers out to 12 months following delivery in most states. CMS also has encouraged hospitals to participate in voluntary statewide or national programs to improve the quality of perinatal care. Last year the agency launched a “Birthing-Friendly” designation icon for qualifying hospitals on its Care Compare online tool.
Support and Opposition
CMS is accepting comments on the draft 2025 hospital outpatient rule, which includes the obstetric COP proposal, through September 9.
Supporters of the obstetric COP approach included the American Nurses Association (ANA), which urged CMS to consider how staffing shortages can undermine patient care in creating COP requirements.
“Nurses are professionals providing critical healthcare services to patients; they should not have to fight for allotted breaks and other challenges created by antiquated views of the profession and payment policies that disincentivize adequate nurse staffing,” Debbie Hatmaker, PhD, RN, ANA’s chief nursing officer, wrote in a June 7 comment to CMS.
The American College of Obstetricians and Gynecologists (ACOG) and the Association of American Medical Colleges (AAMC) also objected to the prospect of new COP for maternal healthcare. They detailed their concerns in separate comments submitted in June 2024.
ACOG said it feared many hospitals might opt to close labor and delivery (L&D) units due to new CMS COP requirements, especially if these take effect “without important and direct stakeholder engagement and buy-in.” More than 200 rural hospitals across the United States stopped providing L&D services in the last decade, Christopher M. Zahn, MD, ACOG’s interim chief executive officer, wrote in a comment to CMS.
“The reason for these closures is varied. Many rural hospitals that still have L&D units continue to lose money on patient services overall, and their ability to continue to deliver maternity care is at risk,” Dr. Zahn wrote.
The AAMC urged CMS to focus on using other strategies such as quality measures to try to improve maternal health and to drop the COP approach. CMS must consider how many clinicians play a role in successful births, including those who see patients during their pregnancies, Jonathan Jaffery, MD, MS, AAMC’s chief healthcare officer, wrote in a comment to the agency.
“Hospitals do have a critical role in improving maternal healthcare equity, especially for labor and delivery outcomes,” he wrote, “but cannot be held solely responsible for implementing much-needed improvements and solutions.”
A version of this article first appeared on Medscape.com.
Federal officials intend to compel US hospitals to improve obstetrical services, with a plan that could result in a potential loss of Medicare and Medicaid funds for institutions that fail to comply with the demands.
The Centers for Medicare and Medicaid Services (CMS) on July 10 announced this proposal, tucking its plan for new conditions of participation (COP) for obstetrician services into the draft 2025 rule on Medicare payments for outpatient hospital services.
The COP requirements are considered the most powerful tool CMS has for trying to improve the quality of medical care. With the new obstetric COP requirement, CMS said it intends to address what it sees as potential shortfalls in training, staffing, transfer protocols, and emergency services readiness.
In practice, hospitals, CMS, and accrediting bodies such as the Joint Commission usually try to address deficiencies to prevent what would be a devastating financial loss for a hospital.
“CMS is using all of our tools to improve the safety, quality, and timeliness of the care that hospitals provide to pregnant women,” Dora Hughes, MD, MPH, acting chief medical officer of the agency, said in a press release about the proposal.
CMS estimated the proposal may add new annual expenses of $70,671 per hospital. For comparison, this figure would represent far less than 1% of the total $1.4 trillion spent on hospital care in the United States in 2022.
CMS said it is trying to address the reasons women in the United States face more risk in giving birth than those in other nations. There were 22 maternal deaths for every 100,000 live births in this country in 2022, compared with 8.6 deaths per 100,000 live births or lower that year in Canada, France, the United Kingdom, Germany, and Japan, CMS said.
But CMS is seeking to impose this new requirement at a time amid growing concerns about “maternity care deserts.”
Reasonable Asks?
Between 2011 and 2021, one out of every four rural hospitals in America stopped providing obstetrics services, Senate Finance Chairman Ron Wyden (D-Ore.) said at a May hearing. Mr. Wyden last year was part of a fight to try to prevent the closure of a birthing center in Baker City in rural eastern Oregon.
The federal government should focus first on helping hospitals keep obstetrical facilities open, said Elizabeth Powers, MD, MHA, the health services officer of the Winding Waters Clinic in Enterprise, Oregon.
“Until we can ensure access to services, we can’t even work on quality,” Dr. Powers told this news organization. “If you’re thinking about a Maslow’s hierarchy of achieving health outcomes, access is your foundation, and without a shift in payment, that foundation is eroded.”
In the draft rule, CMS sketched broad mandates about staffing and training. For example, the agency proposes requiring if a hospital offers obstetrical services, “the services must be well organized and provided in accordance with nationally recognized acceptable standards of practice.”
That means CMS likely will need to provide further guidance for hospitals if it proceeds with this plan for obstetric COP requirements, said Soumi Saha, PharmD, JD, senior vice president of government affairs at Premier Inc., a healthcare consultancy and purchasing organization.
Premier is among the many groups, including the American Hospital Association, that oppose the COP proposal.
Dr. Saha said a better approach would be to consolidate the work being done through the US Department of Health and Human Services (HHS), including earlier CMS projects, to address maternal health in a cohesive way. The Centers for Disease Control and Prevention has programs, as does the HHS Office on Women’s Health.
“How do we really get to a holistic, national, unified approach to addressing this issue that is led by HHS at the top level as the top agency and trickles down consistently versus having all of these kinds of disparate programs in place?” she said.
In recent years, the federal and state governments have taken many steps to try to improve maternal healthcare.
These include the extension of Medicaid benefits to new mothers out to 12 months following delivery in most states. CMS also has encouraged hospitals to participate in voluntary statewide or national programs to improve the quality of perinatal care. Last year the agency launched a “Birthing-Friendly” designation icon for qualifying hospitals on its Care Compare online tool.
Support and Opposition
CMS is accepting comments on the draft 2025 hospital outpatient rule, which includes the obstetric COP proposal, through September 9.
Supporters of the obstetric COP approach included the American Nurses Association (ANA), which urged CMS to consider how staffing shortages can undermine patient care in creating COP requirements.
“Nurses are professionals providing critical healthcare services to patients; they should not have to fight for allotted breaks and other challenges created by antiquated views of the profession and payment policies that disincentivize adequate nurse staffing,” Debbie Hatmaker, PhD, RN, ANA’s chief nursing officer, wrote in a June 7 comment to CMS.
The American College of Obstetricians and Gynecologists (ACOG) and the Association of American Medical Colleges (AAMC) also objected to the prospect of new COP for maternal healthcare. They detailed their concerns in separate comments submitted in June 2024.
ACOG said it feared many hospitals might opt to close labor and delivery (L&D) units due to new CMS COP requirements, especially if these take effect “without important and direct stakeholder engagement and buy-in.” More than 200 rural hospitals across the United States stopped providing L&D services in the last decade, Christopher M. Zahn, MD, ACOG’s interim chief executive officer, wrote in a comment to CMS.
“The reason for these closures is varied. Many rural hospitals that still have L&D units continue to lose money on patient services overall, and their ability to continue to deliver maternity care is at risk,” Dr. Zahn wrote.
The AAMC urged CMS to focus on using other strategies such as quality measures to try to improve maternal health and to drop the COP approach. CMS must consider how many clinicians play a role in successful births, including those who see patients during their pregnancies, Jonathan Jaffery, MD, MS, AAMC’s chief healthcare officer, wrote in a comment to the agency.
“Hospitals do have a critical role in improving maternal healthcare equity, especially for labor and delivery outcomes,” he wrote, “but cannot be held solely responsible for implementing much-needed improvements and solutions.”
A version of this article first appeared on Medscape.com.
Federal officials intend to compel US hospitals to improve obstetrical services, with a plan that could result in a potential loss of Medicare and Medicaid funds for institutions that fail to comply with the demands.
The Centers for Medicare and Medicaid Services (CMS) on July 10 announced this proposal, tucking its plan for new conditions of participation (COP) for obstetrician services into the draft 2025 rule on Medicare payments for outpatient hospital services.
The COP requirements are considered the most powerful tool CMS has for trying to improve the quality of medical care. With the new obstetric COP requirement, CMS said it intends to address what it sees as potential shortfalls in training, staffing, transfer protocols, and emergency services readiness.
In practice, hospitals, CMS, and accrediting bodies such as the Joint Commission usually try to address deficiencies to prevent what would be a devastating financial loss for a hospital.
“CMS is using all of our tools to improve the safety, quality, and timeliness of the care that hospitals provide to pregnant women,” Dora Hughes, MD, MPH, acting chief medical officer of the agency, said in a press release about the proposal.
CMS estimated the proposal may add new annual expenses of $70,671 per hospital. For comparison, this figure would represent far less than 1% of the total $1.4 trillion spent on hospital care in the United States in 2022.
CMS said it is trying to address the reasons women in the United States face more risk in giving birth than those in other nations. There were 22 maternal deaths for every 100,000 live births in this country in 2022, compared with 8.6 deaths per 100,000 live births or lower that year in Canada, France, the United Kingdom, Germany, and Japan, CMS said.
But CMS is seeking to impose this new requirement at a time amid growing concerns about “maternity care deserts.”
Reasonable Asks?
Between 2011 and 2021, one out of every four rural hospitals in America stopped providing obstetrics services, Senate Finance Chairman Ron Wyden (D-Ore.) said at a May hearing. Mr. Wyden last year was part of a fight to try to prevent the closure of a birthing center in Baker City in rural eastern Oregon.
The federal government should focus first on helping hospitals keep obstetrical facilities open, said Elizabeth Powers, MD, MHA, the health services officer of the Winding Waters Clinic in Enterprise, Oregon.
“Until we can ensure access to services, we can’t even work on quality,” Dr. Powers told this news organization. “If you’re thinking about a Maslow’s hierarchy of achieving health outcomes, access is your foundation, and without a shift in payment, that foundation is eroded.”
In the draft rule, CMS sketched broad mandates about staffing and training. For example, the agency proposes requiring if a hospital offers obstetrical services, “the services must be well organized and provided in accordance with nationally recognized acceptable standards of practice.”
That means CMS likely will need to provide further guidance for hospitals if it proceeds with this plan for obstetric COP requirements, said Soumi Saha, PharmD, JD, senior vice president of government affairs at Premier Inc., a healthcare consultancy and purchasing organization.
Premier is among the many groups, including the American Hospital Association, that oppose the COP proposal.
Dr. Saha said a better approach would be to consolidate the work being done through the US Department of Health and Human Services (HHS), including earlier CMS projects, to address maternal health in a cohesive way. The Centers for Disease Control and Prevention has programs, as does the HHS Office on Women’s Health.
“How do we really get to a holistic, national, unified approach to addressing this issue that is led by HHS at the top level as the top agency and trickles down consistently versus having all of these kinds of disparate programs in place?” she said.
In recent years, the federal and state governments have taken many steps to try to improve maternal healthcare.
These include the extension of Medicaid benefits to new mothers out to 12 months following delivery in most states. CMS also has encouraged hospitals to participate in voluntary statewide or national programs to improve the quality of perinatal care. Last year the agency launched a “Birthing-Friendly” designation icon for qualifying hospitals on its Care Compare online tool.
Support and Opposition
CMS is accepting comments on the draft 2025 hospital outpatient rule, which includes the obstetric COP proposal, through September 9.
Supporters of the obstetric COP approach included the American Nurses Association (ANA), which urged CMS to consider how staffing shortages can undermine patient care in creating COP requirements.
“Nurses are professionals providing critical healthcare services to patients; they should not have to fight for allotted breaks and other challenges created by antiquated views of the profession and payment policies that disincentivize adequate nurse staffing,” Debbie Hatmaker, PhD, RN, ANA’s chief nursing officer, wrote in a June 7 comment to CMS.
The American College of Obstetricians and Gynecologists (ACOG) and the Association of American Medical Colleges (AAMC) also objected to the prospect of new COP for maternal healthcare. They detailed their concerns in separate comments submitted in June 2024.
ACOG said it feared many hospitals might opt to close labor and delivery (L&D) units due to new CMS COP requirements, especially if these take effect “without important and direct stakeholder engagement and buy-in.” More than 200 rural hospitals across the United States stopped providing L&D services in the last decade, Christopher M. Zahn, MD, ACOG’s interim chief executive officer, wrote in a comment to CMS.
“The reason for these closures is varied. Many rural hospitals that still have L&D units continue to lose money on patient services overall, and their ability to continue to deliver maternity care is at risk,” Dr. Zahn wrote.
The AAMC urged CMS to focus on using other strategies such as quality measures to try to improve maternal health and to drop the COP approach. CMS must consider how many clinicians play a role in successful births, including those who see patients during their pregnancies, Jonathan Jaffery, MD, MS, AAMC’s chief healthcare officer, wrote in a comment to the agency.
“Hospitals do have a critical role in improving maternal healthcare equity, especially for labor and delivery outcomes,” he wrote, “but cannot be held solely responsible for implementing much-needed improvements and solutions.”
A version of this article first appeared on Medscape.com.
Identifying, Treating Lyme Disease in Primary Care
Geographic spread of the ticks that most often cause Lyme disease in the United States and a rise in incidence of bites, resulting in 476,000 new US cases a year, have increased the chances that physicians who have never encountered a patient with Lyme disease will see their first cases.
“It’s increasing in areas where it was not seen before,” Steven E. Schutzer, MD, with the Department of Medicine, Rutgers New Jersey Medical School, Newark, said in an interview. Dr. Schutzer coauthored a report on diagnosing and treating Lyme disease with Patricia K. Coyle, MD, Department of Neurology, Renaissance School of Medicine at Stony Brook University, Stony Brook, New York.
The report, a Curbside Consult published in New England Journal of Medicine Evidence, comes amid high season for Lyme disease. Bites from an ixodid (hard shield) tick — almost always the source of the disease in the United States — are most common from April through October.
Identifying the Bite
About 70%-90% of the time, Lyme disease will be signaled by erythema migrans (EM) or lesion expanding from the tick bite site, the authors wrote. The “classic” presentation looks like a bullseye, but most of the time the skin will show a variation of that, the authors noted.
“The presence of EM is considered the best clinical diagnostic marker for Lyme disease,” they wrote.
Other dermatologic conditions, however, can complicate diagnosis: “EM mimickers include contact dermatitis, other arthropod bites, fixed drug eruptions, granuloma annulare, cellulitis, dermatophytosis, and systemic lupus erythematosus,” they wrote.
Testing Steps
“The current recommendation is to do two-step testing almost simultaneously,” Dr. Schutzer said in an interview. The first, he said, is an ELISA (enzyme-linked immunosorbent assay)-type test and the second one, used for years, has been a pictoral view of a Western immunoblot showing which antigens of the Lyme bacteria, Borrelia burgdorferi, the antibodies are reacting to.
However, the pictoral view is subjective and some of the antigens could be cross-reactive. So the U.S. Food and Drug Administration (FDA) “has been allowing newer substitutes like a second ELISA-like assay that often uses more recombinant, less cross-reactive antigen targets,” he said. The authors advised that, “The second-tier test should not be performed alone without the first tier.”
Dr. Schutzer advised physicians to check with the lab they plan to use before sending samples.
“If you’re a practicing physician and you know you’re using a particular laboratory, you should familiarize yourself with them, talking to one of the clinical pathologists involved in advance to know what the limitations are.” Take the time to talk with the person overseeing the test and get tips on how they want the sample transported and how the cases should be reported, he said.
If the patient has neurological symptoms, he said, before treating talk with a neurologist who can advise whether, for instance, a spinal tap is in order or whether an emergency department visit is appropriate.
“If you just start proceeding you may mess up the diagnostic signs that could show up in a lab test. Don’t be hesitant to ask for extra input from colleagues,” Dr. Schutzer said.
Suspicion in Endemic Areas
On Long Island, New York, where Lyme disease is endemic, internist Ian Storch, DO, said he sees “a few cases a season.
“We have a lot of people over the summer going to the Hamptons and areas out east for the weekend and tick bites are not uncommon,” he said. “People panic.”
He said one thing it’s important to tell patients is that the tick has to be on the skin for 48-72 hours to transmit the disease. If individuals were in a wooded area and were fine before they got there and the tick was attached for less than 2 days, “they’re usually fine.”
Another issue, Dr. Storch said, is patients sometimes want to get tested for Lyme disease immediately after a tick bite. But the antibody test doesn’t turn positive for weeks, he noted, and you can get a false-negative result. “If you’re worried and you really want to test, you need to wait 6 weeks to do the blood test.”
In his region, he said that although a tick bite is a red flag, he may also suspect Lyme disease when a patient presents with otherwise unexplained joint pain, weakness, lethargy, or fever. “In our area, those are things that would make you test for Lyme.”
He also urged consideration of Lyme in this new age of long COVID. Weakness, fatigue, and lethargy are also classic symptoms of long COVID, he noted. “Keep Lyme disease in your differential because there is a lot of overlap with chronic Lyme disease,” Dr. Storch said.
Discerning Lyme from Southern Tick–Associated Rash Illness
Bonnie M. Word, MD, director of the Houston Travel Medicine Clinic in Texas, where Lyme disease is not endemic, said Lyme disease “will not and should not be on the initial differential diagnosis for those residing in nonendemic areas unless a history of travel to an endemic area is obtained.”
She noted the typical EM rash may not be as distinct or easy to discern on black and brown skin. In addition, she said, EM may have many variations in presentation, such as a crusted center or faint borders, which could lead to a delay in diagnosis and treatment. She suggested consulting the CDC guidance on Lyme disease rashes.
Another challenge in diagnosis, she said, is the patient who presents with what appears to be a classic EM lesion but does not live in a Lyme-endemic area. In Texas, Southern Tick–Associated Rash Illness (STARI) may present with a similar lesion, she said.
“It is transmitted by the Lone Star Tick, which is found in the southeast and south-central US,” Dr. Word said. “However, its habitat is moving northward and westerly,” she said.
Adding Lyme disease to the differential diagnosis is reasonable, she said, if a patient presents with neurologic symptoms “such as a facial palsy, meningitis, radiculitis, and carditis if in addition to their symptoms there is evidence of an epidemiologic link to a Lyme-endemic region.”
She noted that a detailed travel history is important as “Lyme is also endemic in Eastern Canada, Europe, states of the former Soviet Union, China, Mongolia, and Japan.”
Primary care physicians play a critical role in evaluating, diagnosing, and treating most cases of early Lyme disease, thus limiting the number of people who will develop disseminated or late Lyme disease, she said. “The two latter manifestations are most often treated by infectious disease, neurology, or rheumatology specialists.”
Treatment*
Treatment is tailored to the clinical situation, Dr. Schutzer and Dr. Coyle write. A watch-and-wait approach may be appropriate in an asymptomatic but concerned person, even in an endemic area if the person has no known tick bite and no EM lesion.
If there is high risk of an infected ixodid tick bite in a high-incidence area and the tick was attached for at least 36 hours but less than 72 hours, one dose of doxycycline has been recommended as prophylaxis.
When a diagnosis of early nondisseminated Lyme disease is made after observation of an EM lesion, oral antibiotics are typically used to treat for 10 to 14 days. Suggested oral antibiotics and doses are 100 mg of doxycycline twice a day, 500 mg of amoxicillin three times a day, or 500 mg of cefuroxime twice a day, the authors write.
Dr. Schutzer said he hopes the paper serves as a refresher for those physicians who regularly see Lyme disease cases and also helps those newly included in the disease’s spreading regions.
“The earlier you diagnose it, the earlier you can treat it and the better the chance for a favorable outcome,” he said.
Dr. Schutzer, Dr. Coyle, Dr. Storch, and Dr. Word reported no relevant financial relationships.
*This story was updated on August, 2, 2024.
Geographic spread of the ticks that most often cause Lyme disease in the United States and a rise in incidence of bites, resulting in 476,000 new US cases a year, have increased the chances that physicians who have never encountered a patient with Lyme disease will see their first cases.
“It’s increasing in areas where it was not seen before,” Steven E. Schutzer, MD, with the Department of Medicine, Rutgers New Jersey Medical School, Newark, said in an interview. Dr. Schutzer coauthored a report on diagnosing and treating Lyme disease with Patricia K. Coyle, MD, Department of Neurology, Renaissance School of Medicine at Stony Brook University, Stony Brook, New York.
The report, a Curbside Consult published in New England Journal of Medicine Evidence, comes amid high season for Lyme disease. Bites from an ixodid (hard shield) tick — almost always the source of the disease in the United States — are most common from April through October.
Identifying the Bite
About 70%-90% of the time, Lyme disease will be signaled by erythema migrans (EM) or lesion expanding from the tick bite site, the authors wrote. The “classic” presentation looks like a bullseye, but most of the time the skin will show a variation of that, the authors noted.
“The presence of EM is considered the best clinical diagnostic marker for Lyme disease,” they wrote.
Other dermatologic conditions, however, can complicate diagnosis: “EM mimickers include contact dermatitis, other arthropod bites, fixed drug eruptions, granuloma annulare, cellulitis, dermatophytosis, and systemic lupus erythematosus,” they wrote.
Testing Steps
“The current recommendation is to do two-step testing almost simultaneously,” Dr. Schutzer said in an interview. The first, he said, is an ELISA (enzyme-linked immunosorbent assay)-type test and the second one, used for years, has been a pictoral view of a Western immunoblot showing which antigens of the Lyme bacteria, Borrelia burgdorferi, the antibodies are reacting to.
However, the pictoral view is subjective and some of the antigens could be cross-reactive. So the U.S. Food and Drug Administration (FDA) “has been allowing newer substitutes like a second ELISA-like assay that often uses more recombinant, less cross-reactive antigen targets,” he said. The authors advised that, “The second-tier test should not be performed alone without the first tier.”
Dr. Schutzer advised physicians to check with the lab they plan to use before sending samples.
“If you’re a practicing physician and you know you’re using a particular laboratory, you should familiarize yourself with them, talking to one of the clinical pathologists involved in advance to know what the limitations are.” Take the time to talk with the person overseeing the test and get tips on how they want the sample transported and how the cases should be reported, he said.
If the patient has neurological symptoms, he said, before treating talk with a neurologist who can advise whether, for instance, a spinal tap is in order or whether an emergency department visit is appropriate.
“If you just start proceeding you may mess up the diagnostic signs that could show up in a lab test. Don’t be hesitant to ask for extra input from colleagues,” Dr. Schutzer said.
Suspicion in Endemic Areas
On Long Island, New York, where Lyme disease is endemic, internist Ian Storch, DO, said he sees “a few cases a season.
“We have a lot of people over the summer going to the Hamptons and areas out east for the weekend and tick bites are not uncommon,” he said. “People panic.”
He said one thing it’s important to tell patients is that the tick has to be on the skin for 48-72 hours to transmit the disease. If individuals were in a wooded area and were fine before they got there and the tick was attached for less than 2 days, “they’re usually fine.”
Another issue, Dr. Storch said, is patients sometimes want to get tested for Lyme disease immediately after a tick bite. But the antibody test doesn’t turn positive for weeks, he noted, and you can get a false-negative result. “If you’re worried and you really want to test, you need to wait 6 weeks to do the blood test.”
In his region, he said that although a tick bite is a red flag, he may also suspect Lyme disease when a patient presents with otherwise unexplained joint pain, weakness, lethargy, or fever. “In our area, those are things that would make you test for Lyme.”
He also urged consideration of Lyme in this new age of long COVID. Weakness, fatigue, and lethargy are also classic symptoms of long COVID, he noted. “Keep Lyme disease in your differential because there is a lot of overlap with chronic Lyme disease,” Dr. Storch said.
Discerning Lyme from Southern Tick–Associated Rash Illness
Bonnie M. Word, MD, director of the Houston Travel Medicine Clinic in Texas, where Lyme disease is not endemic, said Lyme disease “will not and should not be on the initial differential diagnosis for those residing in nonendemic areas unless a history of travel to an endemic area is obtained.”
She noted the typical EM rash may not be as distinct or easy to discern on black and brown skin. In addition, she said, EM may have many variations in presentation, such as a crusted center or faint borders, which could lead to a delay in diagnosis and treatment. She suggested consulting the CDC guidance on Lyme disease rashes.
Another challenge in diagnosis, she said, is the patient who presents with what appears to be a classic EM lesion but does not live in a Lyme-endemic area. In Texas, Southern Tick–Associated Rash Illness (STARI) may present with a similar lesion, she said.
“It is transmitted by the Lone Star Tick, which is found in the southeast and south-central US,” Dr. Word said. “However, its habitat is moving northward and westerly,” she said.
Adding Lyme disease to the differential diagnosis is reasonable, she said, if a patient presents with neurologic symptoms “such as a facial palsy, meningitis, radiculitis, and carditis if in addition to their symptoms there is evidence of an epidemiologic link to a Lyme-endemic region.”
She noted that a detailed travel history is important as “Lyme is also endemic in Eastern Canada, Europe, states of the former Soviet Union, China, Mongolia, and Japan.”
Primary care physicians play a critical role in evaluating, diagnosing, and treating most cases of early Lyme disease, thus limiting the number of people who will develop disseminated or late Lyme disease, she said. “The two latter manifestations are most often treated by infectious disease, neurology, or rheumatology specialists.”
Treatment*
Treatment is tailored to the clinical situation, Dr. Schutzer and Dr. Coyle write. A watch-and-wait approach may be appropriate in an asymptomatic but concerned person, even in an endemic area if the person has no known tick bite and no EM lesion.
If there is high risk of an infected ixodid tick bite in a high-incidence area and the tick was attached for at least 36 hours but less than 72 hours, one dose of doxycycline has been recommended as prophylaxis.
When a diagnosis of early nondisseminated Lyme disease is made after observation of an EM lesion, oral antibiotics are typically used to treat for 10 to 14 days. Suggested oral antibiotics and doses are 100 mg of doxycycline twice a day, 500 mg of amoxicillin three times a day, or 500 mg of cefuroxime twice a day, the authors write.
Dr. Schutzer said he hopes the paper serves as a refresher for those physicians who regularly see Lyme disease cases and also helps those newly included in the disease’s spreading regions.
“The earlier you diagnose it, the earlier you can treat it and the better the chance for a favorable outcome,” he said.
Dr. Schutzer, Dr. Coyle, Dr. Storch, and Dr. Word reported no relevant financial relationships.
*This story was updated on August, 2, 2024.
Geographic spread of the ticks that most often cause Lyme disease in the United States and a rise in incidence of bites, resulting in 476,000 new US cases a year, have increased the chances that physicians who have never encountered a patient with Lyme disease will see their first cases.
“It’s increasing in areas where it was not seen before,” Steven E. Schutzer, MD, with the Department of Medicine, Rutgers New Jersey Medical School, Newark, said in an interview. Dr. Schutzer coauthored a report on diagnosing and treating Lyme disease with Patricia K. Coyle, MD, Department of Neurology, Renaissance School of Medicine at Stony Brook University, Stony Brook, New York.
The report, a Curbside Consult published in New England Journal of Medicine Evidence, comes amid high season for Lyme disease. Bites from an ixodid (hard shield) tick — almost always the source of the disease in the United States — are most common from April through October.
Identifying the Bite
About 70%-90% of the time, Lyme disease will be signaled by erythema migrans (EM) or lesion expanding from the tick bite site, the authors wrote. The “classic” presentation looks like a bullseye, but most of the time the skin will show a variation of that, the authors noted.
“The presence of EM is considered the best clinical diagnostic marker for Lyme disease,” they wrote.
Other dermatologic conditions, however, can complicate diagnosis: “EM mimickers include contact dermatitis, other arthropod bites, fixed drug eruptions, granuloma annulare, cellulitis, dermatophytosis, and systemic lupus erythematosus,” they wrote.
Testing Steps
“The current recommendation is to do two-step testing almost simultaneously,” Dr. Schutzer said in an interview. The first, he said, is an ELISA (enzyme-linked immunosorbent assay)-type test and the second one, used for years, has been a pictoral view of a Western immunoblot showing which antigens of the Lyme bacteria, Borrelia burgdorferi, the antibodies are reacting to.
However, the pictoral view is subjective and some of the antigens could be cross-reactive. So the U.S. Food and Drug Administration (FDA) “has been allowing newer substitutes like a second ELISA-like assay that often uses more recombinant, less cross-reactive antigen targets,” he said. The authors advised that, “The second-tier test should not be performed alone without the first tier.”
Dr. Schutzer advised physicians to check with the lab they plan to use before sending samples.
“If you’re a practicing physician and you know you’re using a particular laboratory, you should familiarize yourself with them, talking to one of the clinical pathologists involved in advance to know what the limitations are.” Take the time to talk with the person overseeing the test and get tips on how they want the sample transported and how the cases should be reported, he said.
If the patient has neurological symptoms, he said, before treating talk with a neurologist who can advise whether, for instance, a spinal tap is in order or whether an emergency department visit is appropriate.
“If you just start proceeding you may mess up the diagnostic signs that could show up in a lab test. Don’t be hesitant to ask for extra input from colleagues,” Dr. Schutzer said.
Suspicion in Endemic Areas
On Long Island, New York, where Lyme disease is endemic, internist Ian Storch, DO, said he sees “a few cases a season.
“We have a lot of people over the summer going to the Hamptons and areas out east for the weekend and tick bites are not uncommon,” he said. “People panic.”
He said one thing it’s important to tell patients is that the tick has to be on the skin for 48-72 hours to transmit the disease. If individuals were in a wooded area and were fine before they got there and the tick was attached for less than 2 days, “they’re usually fine.”
Another issue, Dr. Storch said, is patients sometimes want to get tested for Lyme disease immediately after a tick bite. But the antibody test doesn’t turn positive for weeks, he noted, and you can get a false-negative result. “If you’re worried and you really want to test, you need to wait 6 weeks to do the blood test.”
In his region, he said that although a tick bite is a red flag, he may also suspect Lyme disease when a patient presents with otherwise unexplained joint pain, weakness, lethargy, or fever. “In our area, those are things that would make you test for Lyme.”
He also urged consideration of Lyme in this new age of long COVID. Weakness, fatigue, and lethargy are also classic symptoms of long COVID, he noted. “Keep Lyme disease in your differential because there is a lot of overlap with chronic Lyme disease,” Dr. Storch said.
Discerning Lyme from Southern Tick–Associated Rash Illness
Bonnie M. Word, MD, director of the Houston Travel Medicine Clinic in Texas, where Lyme disease is not endemic, said Lyme disease “will not and should not be on the initial differential diagnosis for those residing in nonendemic areas unless a history of travel to an endemic area is obtained.”
She noted the typical EM rash may not be as distinct or easy to discern on black and brown skin. In addition, she said, EM may have many variations in presentation, such as a crusted center or faint borders, which could lead to a delay in diagnosis and treatment. She suggested consulting the CDC guidance on Lyme disease rashes.
Another challenge in diagnosis, she said, is the patient who presents with what appears to be a classic EM lesion but does not live in a Lyme-endemic area. In Texas, Southern Tick–Associated Rash Illness (STARI) may present with a similar lesion, she said.
“It is transmitted by the Lone Star Tick, which is found in the southeast and south-central US,” Dr. Word said. “However, its habitat is moving northward and westerly,” she said.
Adding Lyme disease to the differential diagnosis is reasonable, she said, if a patient presents with neurologic symptoms “such as a facial palsy, meningitis, radiculitis, and carditis if in addition to their symptoms there is evidence of an epidemiologic link to a Lyme-endemic region.”
She noted that a detailed travel history is important as “Lyme is also endemic in Eastern Canada, Europe, states of the former Soviet Union, China, Mongolia, and Japan.”
Primary care physicians play a critical role in evaluating, diagnosing, and treating most cases of early Lyme disease, thus limiting the number of people who will develop disseminated or late Lyme disease, she said. “The two latter manifestations are most often treated by infectious disease, neurology, or rheumatology specialists.”
Treatment*
Treatment is tailored to the clinical situation, Dr. Schutzer and Dr. Coyle write. A watch-and-wait approach may be appropriate in an asymptomatic but concerned person, even in an endemic area if the person has no known tick bite and no EM lesion.
If there is high risk of an infected ixodid tick bite in a high-incidence area and the tick was attached for at least 36 hours but less than 72 hours, one dose of doxycycline has been recommended as prophylaxis.
When a diagnosis of early nondisseminated Lyme disease is made after observation of an EM lesion, oral antibiotics are typically used to treat for 10 to 14 days. Suggested oral antibiotics and doses are 100 mg of doxycycline twice a day, 500 mg of amoxicillin three times a day, or 500 mg of cefuroxime twice a day, the authors write.
Dr. Schutzer said he hopes the paper serves as a refresher for those physicians who regularly see Lyme disease cases and also helps those newly included in the disease’s spreading regions.
“The earlier you diagnose it, the earlier you can treat it and the better the chance for a favorable outcome,” he said.
Dr. Schutzer, Dr. Coyle, Dr. Storch, and Dr. Word reported no relevant financial relationships.
*This story was updated on August, 2, 2024.
Maternity Care in Rural Areas Is in Crisis. Can More Doulas Help?
When Bristeria Clark went into labor with her son in 2015, her contractions were steady at first. Then, they stalled. Her cervix stopped dilating. After a few hours, doctors at Phoebe Putney Memorial Hospital in Albany, Georgia, prepped Ms. Clark for an emergency cesarean section.
It wasn’t the vaginal birth Ms. Clark had hoped for during her pregnancy.
“I was freaking out. That was my first child. Like, of course you don’t plan that,” she said. “I just remember the gas pulling up to my face and I ended up going to sleep.”
She remembered feeling a rush of relief when she woke to see that her baby boy was healthy.
Ms. Clark, a 33-year-old nursing student who also works full-time in county government, had another C-section when her second child was born in 2020. This time, the cesarean was planned.
Ms. Clark said she’s grateful the physicians and nurses who delivered both her babies were kind and caring during her labor and delivery. But looking back, she said, she wishes she had had a doula for one-on-one support through pregnancy, childbirth, and the postpartum period. Now she wants to give other women the option she didn’t have.
Ms. Clark is a member of Morehouse School of Medicine’s first class of rural doulas, called Perinatal Patient Navigators.
The program recently graduated a dozen participants, all Black women from southwestern Georgia. They have completed more than 5 months of training and are scheduled to begin working with pregnant and postpartum patients this year.
“We’re developing a workforce that’s going to be providing the support that Black women and birthing people need,” Natalie Hernandez-Green, an associate professor of obstetrics and gynecology at Morehouse School of Medicine, Atlanta, Georgia, said at the doula commencement ceremony in Albany, Georgia.
Albany is Morehouse School of Medicine’s second Perinatal Patient Navigator program site. The first has been up and running in Atlanta since training began in the fall of 2022.
Georgia has one of the highest rates of maternal mortality in the country, according to an analysis by KFF, a health information nonprofit that includes KFF Health News. And Black Georgians are more than twice as likely as White Georgians to die of causes related to pregnancy.
“It doesn’t matter whether you’re rich or poor. Black women are dying at [an] alarming rate from pregnancy-related complications,” said Dr. Hernandez-Green, who is also executive director of the Center for Maternal Health Equity at Morehouse School of Medicine. “And we’re about to change that, one person at a time.”
The presence of a doula, along with regular nursing care, is associated with improved labor and delivery outcomes, reduced stress, and higher rates of patient satisfaction, according to the American College of Obstetricians and Gynecologists.
Multiple studies also link doulas to fewer expensive childbirth interventions, including cesarean births.
Doulas are not medical professionals. They are trained to offer education about the pregnancy and postpartum periods, to guide patients through the healthcare system, and to provide emotional and physical support before, during, and after childbirth.
Morehouse School of Medicine’s program is among a growing number of similar efforts being introduced across the country as more communities look to doulas to help address maternal mortality and poor maternal health outcomes, particularly for Black women and other women of color.
Now that she has graduated, Ms. Clark said she’s looking forward to helping other women in her community as a doula. “To be that person that would be there for my clients, treat them like a sister or like a mother, in a sense of just treating them with utmost respect,” she said. “The ultimate goal is to make them feel comfortable and let them know ‘I’m here to support you.’ ” Her training has inspired her to become an advocate for maternal health issues in southwestern Georgia.
Grants fund Morehouse School of Medicine’s doula program, which costs $350,000 a year to operate. Graduates are given a $2,000 training stipend and the program places five graduates with healthcare providers in southwestern Georgia. Grant money also pays the doulas’ salaries for 1 year.
“It’s not sustainable if you’re chasing the next grant to fund it,” said Rachel Hardeman, a professor of health and racial equity at the University of Minnesota School of Public Health.
Thirteen states cover doulas through Medicaid, according to the Georgetown University Center for Children and Families.
Dr. Hardeman and others have found that when Medicaid programs cover doula care, states save millions of dollars in healthcare costs. “We were able to calculate the return on investment if Medicaid decided to reimburse doulas for pregnant people who are Medicaid beneficiaries,” she said.
That’s because doulas can help reduce the number of expensive medical interventions during and after birth, and improving delivery outcomes, including reduced cesarean sections.
Doulas can even reduce the likelihood of preterm birth.
“An infant that is born at a very, very early gestational age is going to require a great deal of resources and interventions to ensure that they survive and then continue to thrive,” Dr. Hardeman said.
There is growing demand for doula services in Georgia, said Fowzio Jama, director of research for Healthy Mothers, Healthy Babies Coalition of Georgia. Her group recently completed a pilot study that offered doula services to about 170 Georgians covered under Medicaid. “We had a wait-list of over 200 clients and we wanted to give them the support that they needed, but we just couldn’t with the given resources that we had,” Ms. Jama said.
Doula services can cost hundreds or thousands of dollars out-of-pocket, making it too expensive for many low-income people, rural communities, and communities of color, many of which suffer from shortages in maternity care, according to the March of Dimes.
The Healthy Mothers, Healthy Babies study found that matching high-risk patients with doulas — particularly doulas from similar racial and ethnic backgrounds — had a positive effect on patients.
“There was a reduced use of pitocin to induce labor. We saw fewer requests for pain medication. And with our infants, only 6% were low birth weight,” Ms. Jama said.
Still, she and others acknowledge that doulas alone can’t fix the problem of high maternal mortality and morbidity rates.
States, including Georgia, need to do more to bring comprehensive maternity care to communities that need more options, Dr. Hardeman said.
“I think it’s important to understand that doulas are not going to save us, and we should not put that expectation on them. Doulas are a tool,” she said. “They are a piece of the puzzle that is helping to impact a really, really complex issue.”
In the meantime, Joan Anderson, 55, said she’s excited to get to work supporting patients, especially from rural areas around Albany.
“I feel like I’m equipped to go out and be that voice, be that person that our community needs so bad,” said Ms. Anderson, a graduate of the Morehouse School of Medicine doula program. “I am encouraged to know that I will be joining in that mission, that fight for us, as far as maternal health is concerned.”
Ms. Anderson said that someday she wants to open a birthing center to provide maternity care. “We do not have one here in southwest Georgia at all,” Ms. Anderson said.
In addition to providing support during and after childbirth, Ms. Anderson and her fellow graduates are trained to assess their patients’ needs and connect them to services such as food assistance, mental health care, transportation to prenatal appointments, and breastfeeding assistance.
Their work is likely to have ripple effects across a largely rural corner of Georgia, said Sherrell Byrd, who co-founded and directs SOWEGA Rising, a nonprofit organization in southwestern Georgia.
“So many of the graduates are part of church networks, they are part of community organizations, some of them are our government workers. They’re very connected,” Ms. Byrd said. “And I think that connectedness is what’s going to help them be successful moving forward.”
This reporting is part of a fellowship with the Association of Health Care Journalists supported by The Commonwealth Fund. It comes from a partnership that includes WABE, NPR, and KFF Health News.
A version of this article first appeared on KFF Health News.
When Bristeria Clark went into labor with her son in 2015, her contractions were steady at first. Then, they stalled. Her cervix stopped dilating. After a few hours, doctors at Phoebe Putney Memorial Hospital in Albany, Georgia, prepped Ms. Clark for an emergency cesarean section.
It wasn’t the vaginal birth Ms. Clark had hoped for during her pregnancy.
“I was freaking out. That was my first child. Like, of course you don’t plan that,” she said. “I just remember the gas pulling up to my face and I ended up going to sleep.”
She remembered feeling a rush of relief when she woke to see that her baby boy was healthy.
Ms. Clark, a 33-year-old nursing student who also works full-time in county government, had another C-section when her second child was born in 2020. This time, the cesarean was planned.
Ms. Clark said she’s grateful the physicians and nurses who delivered both her babies were kind and caring during her labor and delivery. But looking back, she said, she wishes she had had a doula for one-on-one support through pregnancy, childbirth, and the postpartum period. Now she wants to give other women the option she didn’t have.
Ms. Clark is a member of Morehouse School of Medicine’s first class of rural doulas, called Perinatal Patient Navigators.
The program recently graduated a dozen participants, all Black women from southwestern Georgia. They have completed more than 5 months of training and are scheduled to begin working with pregnant and postpartum patients this year.
“We’re developing a workforce that’s going to be providing the support that Black women and birthing people need,” Natalie Hernandez-Green, an associate professor of obstetrics and gynecology at Morehouse School of Medicine, Atlanta, Georgia, said at the doula commencement ceremony in Albany, Georgia.
Albany is Morehouse School of Medicine’s second Perinatal Patient Navigator program site. The first has been up and running in Atlanta since training began in the fall of 2022.
Georgia has one of the highest rates of maternal mortality in the country, according to an analysis by KFF, a health information nonprofit that includes KFF Health News. And Black Georgians are more than twice as likely as White Georgians to die of causes related to pregnancy.
“It doesn’t matter whether you’re rich or poor. Black women are dying at [an] alarming rate from pregnancy-related complications,” said Dr. Hernandez-Green, who is also executive director of the Center for Maternal Health Equity at Morehouse School of Medicine. “And we’re about to change that, one person at a time.”
The presence of a doula, along with regular nursing care, is associated with improved labor and delivery outcomes, reduced stress, and higher rates of patient satisfaction, according to the American College of Obstetricians and Gynecologists.
Multiple studies also link doulas to fewer expensive childbirth interventions, including cesarean births.
Doulas are not medical professionals. They are trained to offer education about the pregnancy and postpartum periods, to guide patients through the healthcare system, and to provide emotional and physical support before, during, and after childbirth.
Morehouse School of Medicine’s program is among a growing number of similar efforts being introduced across the country as more communities look to doulas to help address maternal mortality and poor maternal health outcomes, particularly for Black women and other women of color.
Now that she has graduated, Ms. Clark said she’s looking forward to helping other women in her community as a doula. “To be that person that would be there for my clients, treat them like a sister or like a mother, in a sense of just treating them with utmost respect,” she said. “The ultimate goal is to make them feel comfortable and let them know ‘I’m here to support you.’ ” Her training has inspired her to become an advocate for maternal health issues in southwestern Georgia.
Grants fund Morehouse School of Medicine’s doula program, which costs $350,000 a year to operate. Graduates are given a $2,000 training stipend and the program places five graduates with healthcare providers in southwestern Georgia. Grant money also pays the doulas’ salaries for 1 year.
“It’s not sustainable if you’re chasing the next grant to fund it,” said Rachel Hardeman, a professor of health and racial equity at the University of Minnesota School of Public Health.
Thirteen states cover doulas through Medicaid, according to the Georgetown University Center for Children and Families.
Dr. Hardeman and others have found that when Medicaid programs cover doula care, states save millions of dollars in healthcare costs. “We were able to calculate the return on investment if Medicaid decided to reimburse doulas for pregnant people who are Medicaid beneficiaries,” she said.
That’s because doulas can help reduce the number of expensive medical interventions during and after birth, and improving delivery outcomes, including reduced cesarean sections.
Doulas can even reduce the likelihood of preterm birth.
“An infant that is born at a very, very early gestational age is going to require a great deal of resources and interventions to ensure that they survive and then continue to thrive,” Dr. Hardeman said.
There is growing demand for doula services in Georgia, said Fowzio Jama, director of research for Healthy Mothers, Healthy Babies Coalition of Georgia. Her group recently completed a pilot study that offered doula services to about 170 Georgians covered under Medicaid. “We had a wait-list of over 200 clients and we wanted to give them the support that they needed, but we just couldn’t with the given resources that we had,” Ms. Jama said.
Doula services can cost hundreds or thousands of dollars out-of-pocket, making it too expensive for many low-income people, rural communities, and communities of color, many of which suffer from shortages in maternity care, according to the March of Dimes.
The Healthy Mothers, Healthy Babies study found that matching high-risk patients with doulas — particularly doulas from similar racial and ethnic backgrounds — had a positive effect on patients.
“There was a reduced use of pitocin to induce labor. We saw fewer requests for pain medication. And with our infants, only 6% were low birth weight,” Ms. Jama said.
Still, she and others acknowledge that doulas alone can’t fix the problem of high maternal mortality and morbidity rates.
States, including Georgia, need to do more to bring comprehensive maternity care to communities that need more options, Dr. Hardeman said.
“I think it’s important to understand that doulas are not going to save us, and we should not put that expectation on them. Doulas are a tool,” she said. “They are a piece of the puzzle that is helping to impact a really, really complex issue.”
In the meantime, Joan Anderson, 55, said she’s excited to get to work supporting patients, especially from rural areas around Albany.
“I feel like I’m equipped to go out and be that voice, be that person that our community needs so bad,” said Ms. Anderson, a graduate of the Morehouse School of Medicine doula program. “I am encouraged to know that I will be joining in that mission, that fight for us, as far as maternal health is concerned.”
Ms. Anderson said that someday she wants to open a birthing center to provide maternity care. “We do not have one here in southwest Georgia at all,” Ms. Anderson said.
In addition to providing support during and after childbirth, Ms. Anderson and her fellow graduates are trained to assess their patients’ needs and connect them to services such as food assistance, mental health care, transportation to prenatal appointments, and breastfeeding assistance.
Their work is likely to have ripple effects across a largely rural corner of Georgia, said Sherrell Byrd, who co-founded and directs SOWEGA Rising, a nonprofit organization in southwestern Georgia.
“So many of the graduates are part of church networks, they are part of community organizations, some of them are our government workers. They’re very connected,” Ms. Byrd said. “And I think that connectedness is what’s going to help them be successful moving forward.”
This reporting is part of a fellowship with the Association of Health Care Journalists supported by The Commonwealth Fund. It comes from a partnership that includes WABE, NPR, and KFF Health News.
A version of this article first appeared on KFF Health News.
When Bristeria Clark went into labor with her son in 2015, her contractions were steady at first. Then, they stalled. Her cervix stopped dilating. After a few hours, doctors at Phoebe Putney Memorial Hospital in Albany, Georgia, prepped Ms. Clark for an emergency cesarean section.
It wasn’t the vaginal birth Ms. Clark had hoped for during her pregnancy.
“I was freaking out. That was my first child. Like, of course you don’t plan that,” she said. “I just remember the gas pulling up to my face and I ended up going to sleep.”
She remembered feeling a rush of relief when she woke to see that her baby boy was healthy.
Ms. Clark, a 33-year-old nursing student who also works full-time in county government, had another C-section when her second child was born in 2020. This time, the cesarean was planned.
Ms. Clark said she’s grateful the physicians and nurses who delivered both her babies were kind and caring during her labor and delivery. But looking back, she said, she wishes she had had a doula for one-on-one support through pregnancy, childbirth, and the postpartum period. Now she wants to give other women the option she didn’t have.
Ms. Clark is a member of Morehouse School of Medicine’s first class of rural doulas, called Perinatal Patient Navigators.
The program recently graduated a dozen participants, all Black women from southwestern Georgia. They have completed more than 5 months of training and are scheduled to begin working with pregnant and postpartum patients this year.
“We’re developing a workforce that’s going to be providing the support that Black women and birthing people need,” Natalie Hernandez-Green, an associate professor of obstetrics and gynecology at Morehouse School of Medicine, Atlanta, Georgia, said at the doula commencement ceremony in Albany, Georgia.
Albany is Morehouse School of Medicine’s second Perinatal Patient Navigator program site. The first has been up and running in Atlanta since training began in the fall of 2022.
Georgia has one of the highest rates of maternal mortality in the country, according to an analysis by KFF, a health information nonprofit that includes KFF Health News. And Black Georgians are more than twice as likely as White Georgians to die of causes related to pregnancy.
“It doesn’t matter whether you’re rich or poor. Black women are dying at [an] alarming rate from pregnancy-related complications,” said Dr. Hernandez-Green, who is also executive director of the Center for Maternal Health Equity at Morehouse School of Medicine. “And we’re about to change that, one person at a time.”
The presence of a doula, along with regular nursing care, is associated with improved labor and delivery outcomes, reduced stress, and higher rates of patient satisfaction, according to the American College of Obstetricians and Gynecologists.
Multiple studies also link doulas to fewer expensive childbirth interventions, including cesarean births.
Doulas are not medical professionals. They are trained to offer education about the pregnancy and postpartum periods, to guide patients through the healthcare system, and to provide emotional and physical support before, during, and after childbirth.
Morehouse School of Medicine’s program is among a growing number of similar efforts being introduced across the country as more communities look to doulas to help address maternal mortality and poor maternal health outcomes, particularly for Black women and other women of color.
Now that she has graduated, Ms. Clark said she’s looking forward to helping other women in her community as a doula. “To be that person that would be there for my clients, treat them like a sister or like a mother, in a sense of just treating them with utmost respect,” she said. “The ultimate goal is to make them feel comfortable and let them know ‘I’m here to support you.’ ” Her training has inspired her to become an advocate for maternal health issues in southwestern Georgia.
Grants fund Morehouse School of Medicine’s doula program, which costs $350,000 a year to operate. Graduates are given a $2,000 training stipend and the program places five graduates with healthcare providers in southwestern Georgia. Grant money also pays the doulas’ salaries for 1 year.
“It’s not sustainable if you’re chasing the next grant to fund it,” said Rachel Hardeman, a professor of health and racial equity at the University of Minnesota School of Public Health.
Thirteen states cover doulas through Medicaid, according to the Georgetown University Center for Children and Families.
Dr. Hardeman and others have found that when Medicaid programs cover doula care, states save millions of dollars in healthcare costs. “We were able to calculate the return on investment if Medicaid decided to reimburse doulas for pregnant people who are Medicaid beneficiaries,” she said.
That’s because doulas can help reduce the number of expensive medical interventions during and after birth, and improving delivery outcomes, including reduced cesarean sections.
Doulas can even reduce the likelihood of preterm birth.
“An infant that is born at a very, very early gestational age is going to require a great deal of resources and interventions to ensure that they survive and then continue to thrive,” Dr. Hardeman said.
There is growing demand for doula services in Georgia, said Fowzio Jama, director of research for Healthy Mothers, Healthy Babies Coalition of Georgia. Her group recently completed a pilot study that offered doula services to about 170 Georgians covered under Medicaid. “We had a wait-list of over 200 clients and we wanted to give them the support that they needed, but we just couldn’t with the given resources that we had,” Ms. Jama said.
Doula services can cost hundreds or thousands of dollars out-of-pocket, making it too expensive for many low-income people, rural communities, and communities of color, many of which suffer from shortages in maternity care, according to the March of Dimes.
The Healthy Mothers, Healthy Babies study found that matching high-risk patients with doulas — particularly doulas from similar racial and ethnic backgrounds — had a positive effect on patients.
“There was a reduced use of pitocin to induce labor. We saw fewer requests for pain medication. And with our infants, only 6% were low birth weight,” Ms. Jama said.
Still, she and others acknowledge that doulas alone can’t fix the problem of high maternal mortality and morbidity rates.
States, including Georgia, need to do more to bring comprehensive maternity care to communities that need more options, Dr. Hardeman said.
“I think it’s important to understand that doulas are not going to save us, and we should not put that expectation on them. Doulas are a tool,” she said. “They are a piece of the puzzle that is helping to impact a really, really complex issue.”
In the meantime, Joan Anderson, 55, said she’s excited to get to work supporting patients, especially from rural areas around Albany.
“I feel like I’m equipped to go out and be that voice, be that person that our community needs so bad,” said Ms. Anderson, a graduate of the Morehouse School of Medicine doula program. “I am encouraged to know that I will be joining in that mission, that fight for us, as far as maternal health is concerned.”
Ms. Anderson said that someday she wants to open a birthing center to provide maternity care. “We do not have one here in southwest Georgia at all,” Ms. Anderson said.
In addition to providing support during and after childbirth, Ms. Anderson and her fellow graduates are trained to assess their patients’ needs and connect them to services such as food assistance, mental health care, transportation to prenatal appointments, and breastfeeding assistance.
Their work is likely to have ripple effects across a largely rural corner of Georgia, said Sherrell Byrd, who co-founded and directs SOWEGA Rising, a nonprofit organization in southwestern Georgia.
“So many of the graduates are part of church networks, they are part of community organizations, some of them are our government workers. They’re very connected,” Ms. Byrd said. “And I think that connectedness is what’s going to help them be successful moving forward.”
This reporting is part of a fellowship with the Association of Health Care Journalists supported by The Commonwealth Fund. It comes from a partnership that includes WABE, NPR, and KFF Health News.
A version of this article first appeared on KFF Health News.
Breakthrough Blood Test for Colorectal Cancer Gets Green Light
The FDA on July 29 approved the test, called Shield, which can accurately detect tumors in the colon or rectum about 87% of the time when the cancer is in treatable early stages. The approval was announced July 29 by the test’s maker, Guardant Health, and comes just months after promising clinical trial results were published in The New England Journal of Medicine.
Colorectal cancer is among the most common types of cancer diagnosed in the United States each year, along with being one of the leading causes of cancer deaths. The condition is treatable in early stages, but about 1 in 3 people don’t stay up to date on regular screenings, which should begin at age 45.
The simplicity of a blood test could make it more likely for people to be screened for and, ultimately, survive the disease. Other primary screening options include feces-based tests or colonoscopy. The 5-year survival rate for colorectal cancer is 64%.
While highly accurate at detecting DNA shed by tumors during treatable stages of colorectal cancer, the Shield test was not as effective at detecting precancerous areas of tissue, which are typically removed after being detected.
In its news release, Guardant Health officials said they anticipate the test to be covered under Medicare. The out-of-pocket cost for people whose insurance does not cover the test has not yet been announced. The test is expected to be available by next week, The New York Times reported.
If someone’s Shield test comes back positive, the person would then get more tests to confirm the result. Shield was shown in trials to have a 10% false positive rate.
“I was in for a routine physical, and my doctor asked when I had my last colonoscopy,” said John Gormly, a 77-year-old business executive in Newport Beach, California, according to a Guardant Health news release. “I said it’s been a long time, so he offered to give me the Shield blood test. A few days later, the result came back positive, so he referred me for a colonoscopy. It turned out I had stage II colon cancer. The tumor was removed, and I recovered very quickly. Thank God I had taken that blood test.”
A version of this article appeared on WebMD.com.
The FDA on July 29 approved the test, called Shield, which can accurately detect tumors in the colon or rectum about 87% of the time when the cancer is in treatable early stages. The approval was announced July 29 by the test’s maker, Guardant Health, and comes just months after promising clinical trial results were published in The New England Journal of Medicine.
Colorectal cancer is among the most common types of cancer diagnosed in the United States each year, along with being one of the leading causes of cancer deaths. The condition is treatable in early stages, but about 1 in 3 people don’t stay up to date on regular screenings, which should begin at age 45.
The simplicity of a blood test could make it more likely for people to be screened for and, ultimately, survive the disease. Other primary screening options include feces-based tests or colonoscopy. The 5-year survival rate for colorectal cancer is 64%.
While highly accurate at detecting DNA shed by tumors during treatable stages of colorectal cancer, the Shield test was not as effective at detecting precancerous areas of tissue, which are typically removed after being detected.
In its news release, Guardant Health officials said they anticipate the test to be covered under Medicare. The out-of-pocket cost for people whose insurance does not cover the test has not yet been announced. The test is expected to be available by next week, The New York Times reported.
If someone’s Shield test comes back positive, the person would then get more tests to confirm the result. Shield was shown in trials to have a 10% false positive rate.
“I was in for a routine physical, and my doctor asked when I had my last colonoscopy,” said John Gormly, a 77-year-old business executive in Newport Beach, California, according to a Guardant Health news release. “I said it’s been a long time, so he offered to give me the Shield blood test. A few days later, the result came back positive, so he referred me for a colonoscopy. It turned out I had stage II colon cancer. The tumor was removed, and I recovered very quickly. Thank God I had taken that blood test.”
A version of this article appeared on WebMD.com.
The FDA on July 29 approved the test, called Shield, which can accurately detect tumors in the colon or rectum about 87% of the time when the cancer is in treatable early stages. The approval was announced July 29 by the test’s maker, Guardant Health, and comes just months after promising clinical trial results were published in The New England Journal of Medicine.
Colorectal cancer is among the most common types of cancer diagnosed in the United States each year, along with being one of the leading causes of cancer deaths. The condition is treatable in early stages, but about 1 in 3 people don’t stay up to date on regular screenings, which should begin at age 45.
The simplicity of a blood test could make it more likely for people to be screened for and, ultimately, survive the disease. Other primary screening options include feces-based tests or colonoscopy. The 5-year survival rate for colorectal cancer is 64%.
While highly accurate at detecting DNA shed by tumors during treatable stages of colorectal cancer, the Shield test was not as effective at detecting precancerous areas of tissue, which are typically removed after being detected.
In its news release, Guardant Health officials said they anticipate the test to be covered under Medicare. The out-of-pocket cost for people whose insurance does not cover the test has not yet been announced. The test is expected to be available by next week, The New York Times reported.
If someone’s Shield test comes back positive, the person would then get more tests to confirm the result. Shield was shown in trials to have a 10% false positive rate.
“I was in for a routine physical, and my doctor asked when I had my last colonoscopy,” said John Gormly, a 77-year-old business executive in Newport Beach, California, according to a Guardant Health news release. “I said it’s been a long time, so he offered to give me the Shield blood test. A few days later, the result came back positive, so he referred me for a colonoscopy. It turned out I had stage II colon cancer. The tumor was removed, and I recovered very quickly. Thank God I had taken that blood test.”
A version of this article appeared on WebMD.com.
FDA Calls AstraZeneca’s NSCLC Trial Design Into Question
The trial in question, AEGEAN, investigated perioperative durvalumab for resectable NSCLC tumors across 802 patients. Patients without EGFR or ALK mutations were randomly assigned to receive durvalumab before surgery alongside platinum-containing chemotherapy and after surgery for a year as monotherapy or to receive chemotherapy and surgery alone.
Patients receiving durvalumab demonstrated better event-free survival at 1 year (73.4% vs 64.5% without durvalumab) and a better pathologic complete response rate (17.2% vs 4.3% without). Currently, AstraZeneca is seeking to add the indication for durvalumab to those the agent already has.
However, at the July 25 ODAC meeting, the committee explained that the AEGEAN trial design makes it impossible to tell whether patients benefited from durvalumab before surgery, after it, or at both points.
Mounting evidence, including from AstraZeneca’s own studies, suggests that the benefit of immune checkpoint inhibitors, such as durvalumab, comes before surgery. That means prescribing durvalumab after surgery could be exposing patients to serious side effects and financial toxicity, with potentially no clinical benefit, “magnifying the risk of potential overtreatment,” the committee cautioned.
When AEGEAN was being designed in 2018, FDA requested that AstraZeneca address the uncertainty surrounding when to use durvalumab by including separate neoadjuvant and adjuvant arms, or at least an arm where patients were treated with neoadjuvant durvalumab alone to compare with treatment both before and after surgery.
The company didn’t follow through and, during the July 25 meeting, the committee wanted answers. “Why did you not comply with this?” asked ODAC committee acting chair Daniel Spratt, MD, a radiation oncologist at Case Western Reserve University in Cleveland, Ohio.
AstraZeneca personnel explained that doing so would have required many more subjects, made the trial more expensive, and added about 2 years to AEGEAN.
One speaker noted that the company, which makes more than $4 billion a year on durvalumab, would have taken about 2 days to recoup that added cost. Others wondered whether the motive was to sell durvalumab for as long as possible across a patient’s course of treatment.
Perhaps the biggest reason the company ignored the request is that “it wasn’t our understanding at that time that this was a barrier to approval,” an AstraZeneca regulatory affairs specialist said.
To this end, the agency asked its advisory panel to vote on whether it should require — instead of simply request, as it did with AstraZeneca — companies to prove that patients need immunotherapy both before and after surgery in resectable NSCLC.
The 11-member panel voted unanimously that it should make this a requirement, and several members said it should do so in other cancers as well.
However, when the agency asked whether durvalumab’s resectable NSCLC approval should be delayed until AstraZeneca conducts a trial to answer the neoadjuvant vs adjuvant question, the panel members didn’t think so.
The consensus was that because AEGEAN showed a decent benefit, patients and physicians should have it as an option, and approval shouldn’t be delayed. The panel said that the bigger question about the benefit of maintenance therapy should be left to future studies.
FDA usually follows the advice of its advisory panels.
A version of this article appeared on Medscape.com.
The trial in question, AEGEAN, investigated perioperative durvalumab for resectable NSCLC tumors across 802 patients. Patients without EGFR or ALK mutations were randomly assigned to receive durvalumab before surgery alongside platinum-containing chemotherapy and after surgery for a year as monotherapy or to receive chemotherapy and surgery alone.
Patients receiving durvalumab demonstrated better event-free survival at 1 year (73.4% vs 64.5% without durvalumab) and a better pathologic complete response rate (17.2% vs 4.3% without). Currently, AstraZeneca is seeking to add the indication for durvalumab to those the agent already has.
However, at the July 25 ODAC meeting, the committee explained that the AEGEAN trial design makes it impossible to tell whether patients benefited from durvalumab before surgery, after it, or at both points.
Mounting evidence, including from AstraZeneca’s own studies, suggests that the benefit of immune checkpoint inhibitors, such as durvalumab, comes before surgery. That means prescribing durvalumab after surgery could be exposing patients to serious side effects and financial toxicity, with potentially no clinical benefit, “magnifying the risk of potential overtreatment,” the committee cautioned.
When AEGEAN was being designed in 2018, FDA requested that AstraZeneca address the uncertainty surrounding when to use durvalumab by including separate neoadjuvant and adjuvant arms, or at least an arm where patients were treated with neoadjuvant durvalumab alone to compare with treatment both before and after surgery.
The company didn’t follow through and, during the July 25 meeting, the committee wanted answers. “Why did you not comply with this?” asked ODAC committee acting chair Daniel Spratt, MD, a radiation oncologist at Case Western Reserve University in Cleveland, Ohio.
AstraZeneca personnel explained that doing so would have required many more subjects, made the trial more expensive, and added about 2 years to AEGEAN.
One speaker noted that the company, which makes more than $4 billion a year on durvalumab, would have taken about 2 days to recoup that added cost. Others wondered whether the motive was to sell durvalumab for as long as possible across a patient’s course of treatment.
Perhaps the biggest reason the company ignored the request is that “it wasn’t our understanding at that time that this was a barrier to approval,” an AstraZeneca regulatory affairs specialist said.
To this end, the agency asked its advisory panel to vote on whether it should require — instead of simply request, as it did with AstraZeneca — companies to prove that patients need immunotherapy both before and after surgery in resectable NSCLC.
The 11-member panel voted unanimously that it should make this a requirement, and several members said it should do so in other cancers as well.
However, when the agency asked whether durvalumab’s resectable NSCLC approval should be delayed until AstraZeneca conducts a trial to answer the neoadjuvant vs adjuvant question, the panel members didn’t think so.
The consensus was that because AEGEAN showed a decent benefit, patients and physicians should have it as an option, and approval shouldn’t be delayed. The panel said that the bigger question about the benefit of maintenance therapy should be left to future studies.
FDA usually follows the advice of its advisory panels.
A version of this article appeared on Medscape.com.
The trial in question, AEGEAN, investigated perioperative durvalumab for resectable NSCLC tumors across 802 patients. Patients without EGFR or ALK mutations were randomly assigned to receive durvalumab before surgery alongside platinum-containing chemotherapy and after surgery for a year as monotherapy or to receive chemotherapy and surgery alone.
Patients receiving durvalumab demonstrated better event-free survival at 1 year (73.4% vs 64.5% without durvalumab) and a better pathologic complete response rate (17.2% vs 4.3% without). Currently, AstraZeneca is seeking to add the indication for durvalumab to those the agent already has.
However, at the July 25 ODAC meeting, the committee explained that the AEGEAN trial design makes it impossible to tell whether patients benefited from durvalumab before surgery, after it, or at both points.
Mounting evidence, including from AstraZeneca’s own studies, suggests that the benefit of immune checkpoint inhibitors, such as durvalumab, comes before surgery. That means prescribing durvalumab after surgery could be exposing patients to serious side effects and financial toxicity, with potentially no clinical benefit, “magnifying the risk of potential overtreatment,” the committee cautioned.
When AEGEAN was being designed in 2018, FDA requested that AstraZeneca address the uncertainty surrounding when to use durvalumab by including separate neoadjuvant and adjuvant arms, or at least an arm where patients were treated with neoadjuvant durvalumab alone to compare with treatment both before and after surgery.
The company didn’t follow through and, during the July 25 meeting, the committee wanted answers. “Why did you not comply with this?” asked ODAC committee acting chair Daniel Spratt, MD, a radiation oncologist at Case Western Reserve University in Cleveland, Ohio.
AstraZeneca personnel explained that doing so would have required many more subjects, made the trial more expensive, and added about 2 years to AEGEAN.
One speaker noted that the company, which makes more than $4 billion a year on durvalumab, would have taken about 2 days to recoup that added cost. Others wondered whether the motive was to sell durvalumab for as long as possible across a patient’s course of treatment.
Perhaps the biggest reason the company ignored the request is that “it wasn’t our understanding at that time that this was a barrier to approval,” an AstraZeneca regulatory affairs specialist said.
To this end, the agency asked its advisory panel to vote on whether it should require — instead of simply request, as it did with AstraZeneca — companies to prove that patients need immunotherapy both before and after surgery in resectable NSCLC.
The 11-member panel voted unanimously that it should make this a requirement, and several members said it should do so in other cancers as well.
However, when the agency asked whether durvalumab’s resectable NSCLC approval should be delayed until AstraZeneca conducts a trial to answer the neoadjuvant vs adjuvant question, the panel members didn’t think so.
The consensus was that because AEGEAN showed a decent benefit, patients and physicians should have it as an option, and approval shouldn’t be delayed. The panel said that the bigger question about the benefit of maintenance therapy should be left to future studies.
FDA usually follows the advice of its advisory panels.
A version of this article appeared on Medscape.com.