Pediatrics

The biologic drug anakinra appears to be effective in treating children with secondary hemophagocytic lymphohistiocytosis (sHLH)/macrophage activation syndrome (MAS), a dangerous “cytokine storm” that can emerge from infections, cancer, and rheumatic diseases.

Children with systematic juvenile idiopathic arthritis (sJIA) and sHLH/MAS are especially good candidates for treatment with the interleukin-1 receptor antagonist anakinra (Kineret), in whom its safety and benefits have been more widely explored than in pediatric patients with sHLH/MAS related to non-sJIA underlying conditions.

In a study published in Arthritis & Rheumatology, Esraa Eloseily, MD, and colleagues at the University of Alabama at Birmingham, looked at hospitalization records for 44 children (mean age, 10 years; n = 25 females) with sHLH/MAS. The children in the study had heterogeneous underlying conditions including leukemias, infections, and rheumatic diseases. About one-third of patients had no known rheumatic or autoimmune disorder.

Dr. Eloseily and colleagues found that early initiation of anakinra (within 5 days of hospitalization) was significantly associated with improved survival across the cohort, for which mortality was 27%. Thrombocytopenia (less than 100,000/mcL) and STXBP2 mutations were both seen significantly associated with mortality.

Patients with blood cancers – even those in remission at the time of treatment – did poorly. None of the three patients in the cohort with leukemia survived.

Importantly, no deaths were seen among the 13 patients with underlying SJIA who were treated with anakinra, suggesting particular benefit for this patient group.

“In addition to the 10% risk of developing overt MAS as part of sJIA, another 30%-40% of sJIA patients may have occult or subclinical MAS during a disease flare that can eventually lead to overt MAS,” Dr. Eloseily and colleagues wrote. “This association of MAS with sJIA suggested that anakinra would also be a valuable treatment for sJIA-MAS.”

The investigators acknowledged that their study was limited by its retrospective design and “nonuniform approach to therapy, lack of treatment controls, and variable follow-up period.” The authors also acknowledged the potential for selection bias favoring anakinra use in patients who are less severely ill.

In a comment accompanying Dr. Eloseily and colleagues’ study, Sarah Nikiforow, MD, PhD, of the Dana-Farber Cancer Institute in Boston, and Nancy Berliner, MD, of Brigham & Women’s Hospital in Boston, urged clinicians not to interpret the study results as supporting anakinra as “a carte blanche approach to hyperinflammatory syndromes.”

While the study supported the use of anakinra in sJIA with MAS or sHLH, “we posit that patients [with sHLH/MAS] in sepsis, cytokine release syndrome following chimeric antigen receptor T-cell therapy, and other hyperinflammatory syndromes still require individualized approaches to therapy,” Dr. Nikiforow and Dr. Berliner wrote, adding that, “in several studies and anecdotally in our institutional practice, cytotoxic chemotherapy was/is preferred over biologic agents in patients with evidence of more severe inflammatory activity.”

Outside sJIA, Dr. Nikiforow and Dr. Berliner wrote, “early anakinra therapy should be extended to treatment of other forms of sHLH with extreme caution. Specifically, the authors’ suggestion that cytotoxic therapy should be ‘considered’ only after anakinra therapy may be dangerous for some patients.”

Two of Dr. Eloseily’s coinvestigators reported financial and research support from Sobi, the manufacturer of anakinra. No other conflicts of interest were reported.

SOURCES: Eloseily E et al. Arthritis Rheumatol. 2019 Sep 12. doi: 10.1002/art.41103; Nikiforow S, Berliner N. Arthritis Rheumatol. 2019 Sep 16. doi: 10.1002/art.41106.

The biologic drug anakinra appears to be effective in treating children with secondary hemophagocytic lymphohistiocytosis (sHLH)/macrophage activation syndrome (MAS), a dangerous “cytokine storm” that can emerge from infections, cancer, and rheumatic diseases.

Children with systematic juvenile idiopathic arthritis (sJIA) and sHLH/MAS are especially good candidates for treatment with the interleukin-1 receptor antagonist anakinra (Kineret), in whom its safety and benefits have been more widely explored than in pediatric patients with sHLH/MAS related to non-sJIA underlying conditions.

In a study published in Arthritis & Rheumatology, Esraa Eloseily, MD, and colleagues at the University of Alabama at Birmingham, looked at hospitalization records for 44 children (mean age, 10 years; n = 25 females) with sHLH/MAS. The children in the study had heterogeneous underlying conditions including leukemias, infections, and rheumatic diseases. About one-third of patients had no known rheumatic or autoimmune disorder.

Dr. Eloseily and colleagues found that early initiation of anakinra (within 5 days of hospitalization) was significantly associated with improved survival across the cohort, for which mortality was 27%. Thrombocytopenia (less than 100,000/mcL) and STXBP2 mutations were both seen significantly associated with mortality.

Patients with blood cancers – even those in remission at the time of treatment – did poorly. None of the three patients in the cohort with leukemia survived.

Importantly, no deaths were seen among the 13 patients with underlying SJIA who were treated with anakinra, suggesting particular benefit for this patient group.

“In addition to the 10% risk of developing overt MAS as part of sJIA, another 30%-40% of sJIA patients may have occult or subclinical MAS during a disease flare that can eventually lead to overt MAS,” Dr. Eloseily and colleagues wrote. “This association of MAS with sJIA suggested that anakinra would also be a valuable treatment for sJIA-MAS.”

The investigators acknowledged that their study was limited by its retrospective design and “nonuniform approach to therapy, lack of treatment controls, and variable follow-up period.” The authors also acknowledged the potential for selection bias favoring anakinra use in patients who are less severely ill.

In a comment accompanying Dr. Eloseily and colleagues’ study, Sarah Nikiforow, MD, PhD, of the Dana-Farber Cancer Institute in Boston, and Nancy Berliner, MD, of Brigham & Women’s Hospital in Boston, urged clinicians not to interpret the study results as supporting anakinra as “a carte blanche approach to hyperinflammatory syndromes.”

While the study supported the use of anakinra in sJIA with MAS or sHLH, “we posit that patients [with sHLH/MAS] in sepsis, cytokine release syndrome following chimeric antigen receptor T-cell therapy, and other hyperinflammatory syndromes still require individualized approaches to therapy,” Dr. Nikiforow and Dr. Berliner wrote, adding that, “in several studies and anecdotally in our institutional practice, cytotoxic chemotherapy was/is preferred over biologic agents in patients with evidence of more severe inflammatory activity.”

Outside sJIA, Dr. Nikiforow and Dr. Berliner wrote, “early anakinra therapy should be extended to treatment of other forms of sHLH with extreme caution. Specifically, the authors’ suggestion that cytotoxic therapy should be ‘considered’ only after anakinra therapy may be dangerous for some patients.”

Two of Dr. Eloseily’s coinvestigators reported financial and research support from Sobi, the manufacturer of anakinra. No other conflicts of interest were reported.

SOURCES: Eloseily E et al. Arthritis Rheumatol. 2019 Sep 12. doi: 10.1002/art.41103; Nikiforow S, Berliner N. Arthritis Rheumatol. 2019 Sep 16. doi: 10.1002/art.41106.

The biologic drug anakinra appears to be effective in treating children with secondary hemophagocytic lymphohistiocytosis (sHLH)/macrophage activation syndrome (MAS), a dangerous “cytokine storm” that can emerge from infections, cancer, and rheumatic diseases.

Children with systematic juvenile idiopathic arthritis (sJIA) and sHLH/MAS are especially good candidates for treatment with the interleukin-1 receptor antagonist anakinra (Kineret), in whom its safety and benefits have been more widely explored than in pediatric patients with sHLH/MAS related to non-sJIA underlying conditions.

In a study published in Arthritis & Rheumatology, Esraa Eloseily, MD, and colleagues at the University of Alabama at Birmingham, looked at hospitalization records for 44 children (mean age, 10 years; n = 25 females) with sHLH/MAS. The children in the study had heterogeneous underlying conditions including leukemias, infections, and rheumatic diseases. About one-third of patients had no known rheumatic or autoimmune disorder.

Dr. Eloseily and colleagues found that early initiation of anakinra (within 5 days of hospitalization) was significantly associated with improved survival across the cohort, for which mortality was 27%. Thrombocytopenia (less than 100,000/mcL) and STXBP2 mutations were both seen significantly associated with mortality.

Patients with blood cancers – even those in remission at the time of treatment – did poorly. None of the three patients in the cohort with leukemia survived.

Importantly, no deaths were seen among the 13 patients with underlying SJIA who were treated with anakinra, suggesting particular benefit for this patient group.

“In addition to the 10% risk of developing overt MAS as part of sJIA, another 30%-40% of sJIA patients may have occult or subclinical MAS during a disease flare that can eventually lead to overt MAS,” Dr. Eloseily and colleagues wrote. “This association of MAS with sJIA suggested that anakinra would also be a valuable treatment for sJIA-MAS.”

The investigators acknowledged that their study was limited by its retrospective design and “nonuniform approach to therapy, lack of treatment controls, and variable follow-up period.” The authors also acknowledged the potential for selection bias favoring anakinra use in patients who are less severely ill.

In a comment accompanying Dr. Eloseily and colleagues’ study, Sarah Nikiforow, MD, PhD, of the Dana-Farber Cancer Institute in Boston, and Nancy Berliner, MD, of Brigham & Women’s Hospital in Boston, urged clinicians not to interpret the study results as supporting anakinra as “a carte blanche approach to hyperinflammatory syndromes.”

While the study supported the use of anakinra in sJIA with MAS or sHLH, “we posit that patients [with sHLH/MAS] in sepsis, cytokine release syndrome following chimeric antigen receptor T-cell therapy, and other hyperinflammatory syndromes still require individualized approaches to therapy,” Dr. Nikiforow and Dr. Berliner wrote, adding that, “in several studies and anecdotally in our institutional practice, cytotoxic chemotherapy was/is preferred over biologic agents in patients with evidence of more severe inflammatory activity.”

Outside sJIA, Dr. Nikiforow and Dr. Berliner wrote, “early anakinra therapy should be extended to treatment of other forms of sHLH with extreme caution. Specifically, the authors’ suggestion that cytotoxic therapy should be ‘considered’ only after anakinra therapy may be dangerous for some patients.”

Two of Dr. Eloseily’s coinvestigators reported financial and research support from Sobi, the manufacturer of anakinra. No other conflicts of interest were reported.

SOURCES: Eloseily E et al. Arthritis Rheumatol. 2019 Sep 12. doi: 10.1002/art.41103; Nikiforow S, Berliner N. Arthritis Rheumatol. 2019 Sep 16. doi: 10.1002/art.41106.

Nearly half of children with atopic dermatitis (AD) were not getting their medications as prescribed, according to a survey of parents of children with AD.

Perceived effectiveness was the main driver of this variation, Alan Schwartz PhD, and Korey Capozza, MPH, wrote in the study, published in Pediatric Dermatology.

“Responses suggest parents may be willing to use therapies with concerning side effects if they can see a clear benefit for their child’s eczema, but when anticipated improvements fail to materialize, they may change their usage, usually in the direction of using less medication or stopping,” observed Dr. Schwartz, of the University of Illinois, Chicago, and Ms. Capozza, of Global Parents for Eczema Research.

“Addressing expectations related to effectiveness, rather than concerns about medication use, may thus be more likely to lead to taking medication as directed.”

The researchers posted a 15-question survey on the Facebook page of Global Parents for Eczema Research, an international coalition of parents of children with AD. During the month that the survey was posted, 86 parents completed it; questions pertained to adherence to medications and reasons for changing treatments. The mean age of their children was 6 years, most (about 83%) had moderate or severe eczema, and about half lived in the United States.

More than half (55%) reported using the AD medications as directed. But 30% said they took or applied less than prescribed, 13% had stopped the prescribed medication altogether, and 2% took or applied more (or more often) than prescribed.

There were several reasons stated for this variance. Concern over side effects was the most common (46%) reason for not using medications as directed. The next most common reasons were that the child’s symptoms went away (28%); or the “medication was not helping or was not helping as much,” in 23%.

A lack of physician trust or not agreeing with the physician’s recommendations accounted for 18% of the concerns. The remainder thought it wasn’t important to take the medication as prescribed, it was inconvenient or too time consuming, that they forgot, it was too expensive, or they were confused about the directions.

To the question asking “What would have made you more likely to use the medication as prescribed?” the most common answer was a clearer indication of effectiveness (56%). The next most common was “access to research or evidence about benefit and side effect profile” (14%).

A good relationship between the physician and patient was associated with taking medication as directed

“Improvement in adherence to topical treatments among children with AD could yield large gains in quality-of-life improvements and reduce exposure to costlier and potentially more toxic systemic agents,” the authors noted. “Given the large, documented gains in disease improvement, and even remission, achieved with interventions that address adherence among patients with other chronic diseases, strategies that address the underlying causes for poor adherence among parents of children with atopic dermatitis stand to provide a significant, untapped benefit.”

No financial disclosures were noted.

[email protected]

SOURCE: Pediatr Dermatol. 2019 Aug 28. doi: 10.1111/pde.13991.

Nearly half of children with atopic dermatitis (AD) were not getting their medications as prescribed, according to a survey of parents of children with AD.

Perceived effectiveness was the main driver of this variation, Alan Schwartz PhD, and Korey Capozza, MPH, wrote in the study, published in Pediatric Dermatology.

“Responses suggest parents may be willing to use therapies with concerning side effects if they can see a clear benefit for their child’s eczema, but when anticipated improvements fail to materialize, they may change their usage, usually in the direction of using less medication or stopping,” observed Dr. Schwartz, of the University of Illinois, Chicago, and Ms. Capozza, of Global Parents for Eczema Research.

“Addressing expectations related to effectiveness, rather than concerns about medication use, may thus be more likely to lead to taking medication as directed.”

The researchers posted a 15-question survey on the Facebook page of Global Parents for Eczema Research, an international coalition of parents of children with AD. During the month that the survey was posted, 86 parents completed it; questions pertained to adherence to medications and reasons for changing treatments. The mean age of their children was 6 years, most (about 83%) had moderate or severe eczema, and about half lived in the United States.

More than half (55%) reported using the AD medications as directed. But 30% said they took or applied less than prescribed, 13% had stopped the prescribed medication altogether, and 2% took or applied more (or more often) than prescribed.

There were several reasons stated for this variance. Concern over side effects was the most common (46%) reason for not using medications as directed. The next most common reasons were that the child’s symptoms went away (28%); or the “medication was not helping or was not helping as much,” in 23%.

A lack of physician trust or not agreeing with the physician’s recommendations accounted for 18% of the concerns. The remainder thought it wasn’t important to take the medication as prescribed, it was inconvenient or too time consuming, that they forgot, it was too expensive, or they were confused about the directions.

To the question asking “What would have made you more likely to use the medication as prescribed?” the most common answer was a clearer indication of effectiveness (56%). The next most common was “access to research or evidence about benefit and side effect profile” (14%).

A good relationship between the physician and patient was associated with taking medication as directed

“Improvement in adherence to topical treatments among children with AD could yield large gains in quality-of-life improvements and reduce exposure to costlier and potentially more toxic systemic agents,” the authors noted. “Given the large, documented gains in disease improvement, and even remission, achieved with interventions that address adherence among patients with other chronic diseases, strategies that address the underlying causes for poor adherence among parents of children with atopic dermatitis stand to provide a significant, untapped benefit.”

No financial disclosures were noted.

[email protected]

SOURCE: Pediatr Dermatol. 2019 Aug 28. doi: 10.1111/pde.13991.

Nearly half of children with atopic dermatitis (AD) were not getting their medications as prescribed, according to a survey of parents of children with AD.

Perceived effectiveness was the main driver of this variation, Alan Schwartz PhD, and Korey Capozza, MPH, wrote in the study, published in Pediatric Dermatology.

“Responses suggest parents may be willing to use therapies with concerning side effects if they can see a clear benefit for their child’s eczema, but when anticipated improvements fail to materialize, they may change their usage, usually in the direction of using less medication or stopping,” observed Dr. Schwartz, of the University of Illinois, Chicago, and Ms. Capozza, of Global Parents for Eczema Research.

“Addressing expectations related to effectiveness, rather than concerns about medication use, may thus be more likely to lead to taking medication as directed.”

The researchers posted a 15-question survey on the Facebook page of Global Parents for Eczema Research, an international coalition of parents of children with AD. During the month that the survey was posted, 86 parents completed it; questions pertained to adherence to medications and reasons for changing treatments. The mean age of their children was 6 years, most (about 83%) had moderate or severe eczema, and about half lived in the United States.

More than half (55%) reported using the AD medications as directed. But 30% said they took or applied less than prescribed, 13% had stopped the prescribed medication altogether, and 2% took or applied more (or more often) than prescribed.

There were several reasons stated for this variance. Concern over side effects was the most common (46%) reason for not using medications as directed. The next most common reasons were that the child’s symptoms went away (28%); or the “medication was not helping or was not helping as much,” in 23%.

A lack of physician trust or not agreeing with the physician’s recommendations accounted for 18% of the concerns. The remainder thought it wasn’t important to take the medication as prescribed, it was inconvenient or too time consuming, that they forgot, it was too expensive, or they were confused about the directions.

To the question asking “What would have made you more likely to use the medication as prescribed?” the most common answer was a clearer indication of effectiveness (56%). The next most common was “access to research or evidence about benefit and side effect profile” (14%).

A good relationship between the physician and patient was associated with taking medication as directed

“Improvement in adherence to topical treatments among children with AD could yield large gains in quality-of-life improvements and reduce exposure to costlier and potentially more toxic systemic agents,” the authors noted. “Given the large, documented gains in disease improvement, and even remission, achieved with interventions that address adherence among patients with other chronic diseases, strategies that address the underlying causes for poor adherence among parents of children with atopic dermatitis stand to provide a significant, untapped benefit.”

No financial disclosures were noted.

[email protected]

SOURCE: Pediatr Dermatol. 2019 Aug 28. doi: 10.1111/pde.13991.

If you are still struggling to understand the ADHD phenomenon and its meteoric rise to prominence over the last 3 or 4 decades, a study published in the September 2019 Pediatrics may help you make sense of why you are spending a large part of your professional day counseling parents and treating children whose lives are disrupted by their impulsivity, distractibility, and inattentiveness (“24-hour movement behaviors and impulsivity.” doi: 10.1542/peds.2019-0187). Researchers at the Children’s Hospital of Eastern Ontario (Canada) Research Institute used data collected over 10 years in 21 sites across the United States on more than 4,500 children aged 8-11 years, looking for possible associations between impulsivity and three factors – sleep duration, screen time, and physical activity.

Goads Agency/Getty Images

They found that children who were exposed to fewer than 2 hours of recreational screen time each day and slept 9-11 hours nightly had significantly reduced scores on a range of impulsivity scores. While participating in at least 60 minutes of vigorous physical activity per day also was associated with less impulsivity, the effect added little to the benefit of the sleep/screen time combination. Although these nonpharmacologic strategies aimed at decreasing impulsivity may not be a cure-all for every child with symptoms that suggest ADHD, the data are compelling.

I hope that the associations these Canadian researchers have unearthed is not news to you. But their observation that 30% of the sample population met none of the recommendations for sleep, screen time, and activity and that only 5% of the sample did suggests that too few of us are delivering the message with sufficient enthusiasm and/or too many parents aren’t taking it seriously.

Over the last several years I have been encouraged to find sleep and screen time limits mentioned in articles on ADHD for both professionals and parents, but these potent contributors to impulsivity and distractibility always seem to be relegated to the oh-by-the-way category at the end of the article after a lengthy discussion of the relative values of medication and cognitive-behavioral therapy. And unfortunately, meeting these behavioral guidelines can be difficult to achieve and cannot be subcontracted out to a therapist or a pharmacist. They require parents to set and enforce limits. Saying no is difficult for all of us, particularly those without much prior experience.

Have you observed the association between inadequate sleep, excessive screen time, and impulsivity? How robustly have you bought into the idea that more sleep and less screen time are, if not THE answers, at least are the two we should start with? Where do your recommendations about screen time, sleep, and physical activity fit into the script when you are talking with parents about their child’s ADHD-ish behaviors? Have you put them in the oh-by-the-way category?

Do you ever say, “I know you may be expecting me to talk about medication at this visit, but I suggest you try setting and enforcing these limits on sleep and screen time for a few months and we will see how things are going”? “And I am going to give you some suggestions on how you can do this, and we will meet again as often as you feel is necessary to ease the process.”

Do you think you have the time to try this approach? Do you feel you have the skills to counsel on sleep and behavior? Do you think you can find someone with the time and experience who shares your priorities about screen time and sleep to do the parental coaching for you? It’s an approach worth considering when you step back and take the longer look at why we are living through this decades-long ADHD phenomenon.
 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

If you are still struggling to understand the ADHD phenomenon and its meteoric rise to prominence over the last 3 or 4 decades, a study published in the September 2019 Pediatrics may help you make sense of why you are spending a large part of your professional day counseling parents and treating children whose lives are disrupted by their impulsivity, distractibility, and inattentiveness (“24-hour movement behaviors and impulsivity.” doi: 10.1542/peds.2019-0187). Researchers at the Children’s Hospital of Eastern Ontario (Canada) Research Institute used data collected over 10 years in 21 sites across the United States on more than 4,500 children aged 8-11 years, looking for possible associations between impulsivity and three factors – sleep duration, screen time, and physical activity.

Goads Agency/Getty Images

They found that children who were exposed to fewer than 2 hours of recreational screen time each day and slept 9-11 hours nightly had significantly reduced scores on a range of impulsivity scores. While participating in at least 60 minutes of vigorous physical activity per day also was associated with less impulsivity, the effect added little to the benefit of the sleep/screen time combination. Although these nonpharmacologic strategies aimed at decreasing impulsivity may not be a cure-all for every child with symptoms that suggest ADHD, the data are compelling.

I hope that the associations these Canadian researchers have unearthed is not news to you. But their observation that 30% of the sample population met none of the recommendations for sleep, screen time, and activity and that only 5% of the sample did suggests that too few of us are delivering the message with sufficient enthusiasm and/or too many parents aren’t taking it seriously.

Over the last several years I have been encouraged to find sleep and screen time limits mentioned in articles on ADHD for both professionals and parents, but these potent contributors to impulsivity and distractibility always seem to be relegated to the oh-by-the-way category at the end of the article after a lengthy discussion of the relative values of medication and cognitive-behavioral therapy. And unfortunately, meeting these behavioral guidelines can be difficult to achieve and cannot be subcontracted out to a therapist or a pharmacist. They require parents to set and enforce limits. Saying no is difficult for all of us, particularly those without much prior experience.

Have you observed the association between inadequate sleep, excessive screen time, and impulsivity? How robustly have you bought into the idea that more sleep and less screen time are, if not THE answers, at least are the two we should start with? Where do your recommendations about screen time, sleep, and physical activity fit into the script when you are talking with parents about their child’s ADHD-ish behaviors? Have you put them in the oh-by-the-way category?

Do you ever say, “I know you may be expecting me to talk about medication at this visit, but I suggest you try setting and enforcing these limits on sleep and screen time for a few months and we will see how things are going”? “And I am going to give you some suggestions on how you can do this, and we will meet again as often as you feel is necessary to ease the process.”

Do you think you have the time to try this approach? Do you feel you have the skills to counsel on sleep and behavior? Do you think you can find someone with the time and experience who shares your priorities about screen time and sleep to do the parental coaching for you? It’s an approach worth considering when you step back and take the longer look at why we are living through this decades-long ADHD phenomenon.
 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

If you are still struggling to understand the ADHD phenomenon and its meteoric rise to prominence over the last 3 or 4 decades, a study published in the September 2019 Pediatrics may help you make sense of why you are spending a large part of your professional day counseling parents and treating children whose lives are disrupted by their impulsivity, distractibility, and inattentiveness (“24-hour movement behaviors and impulsivity.” doi: 10.1542/peds.2019-0187). Researchers at the Children’s Hospital of Eastern Ontario (Canada) Research Institute used data collected over 10 years in 21 sites across the United States on more than 4,500 children aged 8-11 years, looking for possible associations between impulsivity and three factors – sleep duration, screen time, and physical activity.

Goads Agency/Getty Images

They found that children who were exposed to fewer than 2 hours of recreational screen time each day and slept 9-11 hours nightly had significantly reduced scores on a range of impulsivity scores. While participating in at least 60 minutes of vigorous physical activity per day also was associated with less impulsivity, the effect added little to the benefit of the sleep/screen time combination. Although these nonpharmacologic strategies aimed at decreasing impulsivity may not be a cure-all for every child with symptoms that suggest ADHD, the data are compelling.

I hope that the associations these Canadian researchers have unearthed is not news to you. But their observation that 30% of the sample population met none of the recommendations for sleep, screen time, and activity and that only 5% of the sample did suggests that too few of us are delivering the message with sufficient enthusiasm and/or too many parents aren’t taking it seriously.

Over the last several years I have been encouraged to find sleep and screen time limits mentioned in articles on ADHD for both professionals and parents, but these potent contributors to impulsivity and distractibility always seem to be relegated to the oh-by-the-way category at the end of the article after a lengthy discussion of the relative values of medication and cognitive-behavioral therapy. And unfortunately, meeting these behavioral guidelines can be difficult to achieve and cannot be subcontracted out to a therapist or a pharmacist. They require parents to set and enforce limits. Saying no is difficult for all of us, particularly those without much prior experience.

Have you observed the association between inadequate sleep, excessive screen time, and impulsivity? How robustly have you bought into the idea that more sleep and less screen time are, if not THE answers, at least are the two we should start with? Where do your recommendations about screen time, sleep, and physical activity fit into the script when you are talking with parents about their child’s ADHD-ish behaviors? Have you put them in the oh-by-the-way category?

Do you ever say, “I know you may be expecting me to talk about medication at this visit, but I suggest you try setting and enforcing these limits on sleep and screen time for a few months and we will see how things are going”? “And I am going to give you some suggestions on how you can do this, and we will meet again as often as you feel is necessary to ease the process.”

Do you think you have the time to try this approach? Do you feel you have the skills to counsel on sleep and behavior? Do you think you can find someone with the time and experience who shares your priorities about screen time and sleep to do the parental coaching for you? It’s an approach worth considering when you step back and take the longer look at why we are living through this decades-long ADHD phenomenon.
 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

Among preschool children with cystic fibrosis, airway disease as measured by the Perth-Rotterdam Annotated Grid Morphometric Analysis for CF (PRAGMA-CF) accurately predicts bronchiectasis in grade school, reported Nynke R. Bouma, BSc, and colleagues.

“Even though bronchiectasis is present in 60% to 80% of children with CF in school age, the extent and severity of bronchiectasis in preschool children are generally lower ... however, diffuse airway abnormalities such as airway wall thickening and mucus plugging are observed in many preschool children. It is hypothesized that these preschool airway changes reflect diffuse airway disease that eventually will result in bronchiectasis in school age,” they noted.

The PRAGMA-CF image scoring system can measure airway disease and can also be used to monitor disease progression, noted Ms. Bouma of Sophia Children’s Hospital, Rotterdam, and colleagues. The study was published in Pediatric Pulmonology. PRAGMA-CF is a composite score of airway wall thickening, mucus plugging, and bronchiectasis as percent disease (%disease). “In preschool children, %disease measured by PRAGMA-CF on chest CT allows quantification of early clinically relevant morphological features of CF airway disease and it is associated with later school-age bronchiectasis,” the team wrote. “These findings support the use of %disease as a clinically relevant outcome measure in early CF lung disease.”

The team conducted a prospective cohort study of 61 children (mean age 4 years) with cystic fibrosis, following them for a mean of 5 years. A total of 122 CT scans were available from this group, in addition to spirometry data and cystic fibrosis quality of life scores.

From preschool age to school age, the %disease on PRAGMA-CF increased significantly, from a mean of 0.7% to 1.73%. Scores on another composite measuring tool (%MUPAT, a composite score of airway wall thickening and mucus plugging) went from 0.46 to 0.58 – not a significant difference.

A multivariate analysis corrected for age in each school group and the type of scanner used to acquire the images. That analysis determined that each 1% increase in %disease at preschool age resulted in an increase of 1.18% of bronchiectasis at school age.

A cross-sectional analysis of the group at school age found significant associations between the %disease and percent of forced expiratory volume and the cystic fibrosis quality of life score.

At least one pulmonary exacerbation requiring intravenous antibiotics occurred in 19 of the patients. However, the investigators didn’t find any significant interactions between the %disease in preschool and these exacerbations..

“These findings are in line with previous studies in school‐aged children that showed that mucus plugging is associated with inflammation and airway wall thickening, and that these are thought to be risk factors for later bronchiectasis,” they concluded. “On the basis of our findings, we suggest that %disease and %MUPAT could be used as a clinically relevant outcome measure in clinical studies in preschool patients with cystic fibrosis, as these measures predict later bronchiectasis. Percent disease may be preferred as it captures all the principal features of CF airways disease including bronchiectasis.”

Ms. Bouma had no financial disclosures.

[email protected]

SOURCE: Bouma NR et al. Pediatr Pulmonol. 2019 Sep 9 doi: 10.1002/ppul.24498; Rosenow et al. Am J Respir Crit Care Med. 2015 May 15. doi: 10.1164/rccm.201501-0061OC.

Among preschool children with cystic fibrosis, airway disease as measured by the Perth-Rotterdam Annotated Grid Morphometric Analysis for CF (PRAGMA-CF) accurately predicts bronchiectasis in grade school, reported Nynke R. Bouma, BSc, and colleagues.

“Even though bronchiectasis is present in 60% to 80% of children with CF in school age, the extent and severity of bronchiectasis in preschool children are generally lower ... however, diffuse airway abnormalities such as airway wall thickening and mucus plugging are observed in many preschool children. It is hypothesized that these preschool airway changes reflect diffuse airway disease that eventually will result in bronchiectasis in school age,” they noted.

The PRAGMA-CF image scoring system can measure airway disease and can also be used to monitor disease progression, noted Ms. Bouma of Sophia Children’s Hospital, Rotterdam, and colleagues. The study was published in Pediatric Pulmonology. PRAGMA-CF is a composite score of airway wall thickening, mucus plugging, and bronchiectasis as percent disease (%disease). “In preschool children, %disease measured by PRAGMA-CF on chest CT allows quantification of early clinically relevant morphological features of CF airway disease and it is associated with later school-age bronchiectasis,” the team wrote. “These findings support the use of %disease as a clinically relevant outcome measure in early CF lung disease.”

The team conducted a prospective cohort study of 61 children (mean age 4 years) with cystic fibrosis, following them for a mean of 5 years. A total of 122 CT scans were available from this group, in addition to spirometry data and cystic fibrosis quality of life scores.

From preschool age to school age, the %disease on PRAGMA-CF increased significantly, from a mean of 0.7% to 1.73%. Scores on another composite measuring tool (%MUPAT, a composite score of airway wall thickening and mucus plugging) went from 0.46 to 0.58 – not a significant difference.

A multivariate analysis corrected for age in each school group and the type of scanner used to acquire the images. That analysis determined that each 1% increase in %disease at preschool age resulted in an increase of 1.18% of bronchiectasis at school age.

A cross-sectional analysis of the group at school age found significant associations between the %disease and percent of forced expiratory volume and the cystic fibrosis quality of life score.

At least one pulmonary exacerbation requiring intravenous antibiotics occurred in 19 of the patients. However, the investigators didn’t find any significant interactions between the %disease in preschool and these exacerbations..

“These findings are in line with previous studies in school‐aged children that showed that mucus plugging is associated with inflammation and airway wall thickening, and that these are thought to be risk factors for later bronchiectasis,” they concluded. “On the basis of our findings, we suggest that %disease and %MUPAT could be used as a clinically relevant outcome measure in clinical studies in preschool patients with cystic fibrosis, as these measures predict later bronchiectasis. Percent disease may be preferred as it captures all the principal features of CF airways disease including bronchiectasis.”

Ms. Bouma had no financial disclosures.

[email protected]

SOURCE: Bouma NR et al. Pediatr Pulmonol. 2019 Sep 9 doi: 10.1002/ppul.24498; Rosenow et al. Am J Respir Crit Care Med. 2015 May 15. doi: 10.1164/rccm.201501-0061OC.

Among preschool children with cystic fibrosis, airway disease as measured by the Perth-Rotterdam Annotated Grid Morphometric Analysis for CF (PRAGMA-CF) accurately predicts bronchiectasis in grade school, reported Nynke R. Bouma, BSc, and colleagues.

“Even though bronchiectasis is present in 60% to 80% of children with CF in school age, the extent and severity of bronchiectasis in preschool children are generally lower ... however, diffuse airway abnormalities such as airway wall thickening and mucus plugging are observed in many preschool children. It is hypothesized that these preschool airway changes reflect diffuse airway disease that eventually will result in bronchiectasis in school age,” they noted.

The PRAGMA-CF image scoring system can measure airway disease and can also be used to monitor disease progression, noted Ms. Bouma of Sophia Children’s Hospital, Rotterdam, and colleagues. The study was published in Pediatric Pulmonology. PRAGMA-CF is a composite score of airway wall thickening, mucus plugging, and bronchiectasis as percent disease (%disease). “In preschool children, %disease measured by PRAGMA-CF on chest CT allows quantification of early clinically relevant morphological features of CF airway disease and it is associated with later school-age bronchiectasis,” the team wrote. “These findings support the use of %disease as a clinically relevant outcome measure in early CF lung disease.”

The team conducted a prospective cohort study of 61 children (mean age 4 years) with cystic fibrosis, following them for a mean of 5 years. A total of 122 CT scans were available from this group, in addition to spirometry data and cystic fibrosis quality of life scores.

From preschool age to school age, the %disease on PRAGMA-CF increased significantly, from a mean of 0.7% to 1.73%. Scores on another composite measuring tool (%MUPAT, a composite score of airway wall thickening and mucus plugging) went from 0.46 to 0.58 – not a significant difference.

A multivariate analysis corrected for age in each school group and the type of scanner used to acquire the images. That analysis determined that each 1% increase in %disease at preschool age resulted in an increase of 1.18% of bronchiectasis at school age.

A cross-sectional analysis of the group at school age found significant associations between the %disease and percent of forced expiratory volume and the cystic fibrosis quality of life score.

At least one pulmonary exacerbation requiring intravenous antibiotics occurred in 19 of the patients. However, the investigators didn’t find any significant interactions between the %disease in preschool and these exacerbations..

“These findings are in line with previous studies in school‐aged children that showed that mucus plugging is associated with inflammation and airway wall thickening, and that these are thought to be risk factors for later bronchiectasis,” they concluded. “On the basis of our findings, we suggest that %disease and %MUPAT could be used as a clinically relevant outcome measure in clinical studies in preschool patients with cystic fibrosis, as these measures predict later bronchiectasis. Percent disease may be preferred as it captures all the principal features of CF airways disease including bronchiectasis.”

Ms. Bouma had no financial disclosures.

[email protected]

SOURCE: Bouma NR et al. Pediatr Pulmonol. 2019 Sep 9 doi: 10.1002/ppul.24498; Rosenow et al. Am J Respir Crit Care Med. 2015 May 15. doi: 10.1164/rccm.201501-0061OC.

A 54-year-old pharmacist with a history of gout, hypertension, and conjunctivitis presents for evaluation of pink eye in the summer. The morning before coming into the office, he noticed that his right eye was red and inflamed. He self-treated with saline washes and eye drops, but upon awakening the next day, he found his right eye to be crusted shut with surrounding yellow discharge. He has not had any changes to his vision but endorses a somewhat uncomfortable, “gritty” sensation. He reports no recent cough, nasal congestion, or allergies, and he has not been around any sick contacts. His blood pressure is 102/58 mm Hg, pulse is 76 bpm, and body mass index is 27.3 kg/m². His eye exam reveals unilateral conjunctival injections but no hyperemia of the conjunctiva adjacent to the cornea. Mucopurulent discharge was neither found on the undersurface of the eyelid nor emerging from the eye. Which of the following is the best treatment for this patient’s condition?

A) Erythromycin 5 mg/gram ophthalmic ointment.

B) Ofloxacin 0.3% ophthalmic drops.

C) Antihistamine drops.

D) Eye lubricant drops.

E) No treatment necessary.

This patient is an adult presenting with presumed conjunctivitis. Because he is presenting in the summer without observed purulent discharge, his condition is unlikely to be bacterial. This patient does not need treatment, although eye lubricant drops could reduce his discomfort.

Nearly 1% of primary care office visits¹ and 300 million in annual costs² are spent evaluating and treating “pink eye.” After ruling out serious eye disease, clinicians need to determine which cases of suspected conjunctivitis are most likely to be bacterial to allow for judicious use of antibiotic eye drops. This is an important undertaking as most patients assume that antibiotics are needed.

How do we know which history and clinical exam findings to lean on when attempting to categorize conjunctivitis as bacterial or not? If a patient reports purulent discharge, doesn’t that mean it is bacterial? Surprisingly, a systematic review published in 2016 by Narayana and McGee found that a patient’s self-report of “purulent drainage” is diagnostically unhelpful, but if a clinician finds it on exam, the likelihood of a bacterial etiology increases.³

Narayana and McGee analyzed three studies that enrolled a total of 281 patients with presumed conjunctivitis who underwent bacterial cultures. They then determined which findings increased the probability of positive bacterial culture. From strongest to weakest, the best indicators of a bacterial cause were found to be: complete redness of the conjunctival membrane obscuring tarsal vessels (the vessels visible on the inside of everted upper or lower eyelids) (likelihood ratio, 4.6), observed purulent discharge (LR, 3.9), matting of both eyes in the morning (LR, 3.6), and presence during winter/spring months (LR, 1.9). On the other hand, failure to observe a red eye at 20 feet (LR, 0.2), absence of morning gluing of either eye (LR, 0.3), and presentation during summer months (LR, 0.4) all decreased the probability of a bacterial cause. This review and different study by Stenson et al. unfortunately have conflicting evidence regarding whether the following findings are diagnostically helpful: qualities of eye discomfort (such as burning or itching), preauricular adenopathy, conjunctival follicles, and conjunctival papillae.^3,4 Rietveld and colleagues found that a history of conjunctivitis decreased the likelihood of bacterial conjunctivitis.⁵

Pearl: The best indicators of a bacterial cause in patients with presumed conjunctivitis are complete redness of the conjunctival membrane obscuring tarsal vessels, observed purulent discharge, and matting of both eyes in the morning. Presentation during the summer months and having a history of conjunctivitis decreases the likelihood of bacterial conjunctivitis.

Ms. Momany is a fourth-year medical student at University of Washington, Seattle. Dr. Paauw is professor of medicine in the division of general internal medicine at the University of Washington and serves as third-year medical student clerkship director at that university. Contact Dr. Paauw at [email protected].

References

1. Azari AA and Barney NP. JAMA. 2013 Oct 23; 310(16):1721-9.

2. Smith AF and Waycaster C. BMC Ophthalmol. 2009 Nov 25. doi: 10.1186/1471-2415-9-13.

3) Narayana S and McGee S. Am J Med. 2015;128(11):1220-4.e1.

4) Stenson S et al. Arch Ophthalmol. 1982;100(8):1275-7.

5) Rietveld RP et al. BMJ. 2004 Jul 24;329(7459):206-10.

A 54-year-old pharmacist with a history of gout, hypertension, and conjunctivitis presents for evaluation of pink eye in the summer. The morning before coming into the office, he noticed that his right eye was red and inflamed. He self-treated with saline washes and eye drops, but upon awakening the next day, he found his right eye to be crusted shut with surrounding yellow discharge. He has not had any changes to his vision but endorses a somewhat uncomfortable, “gritty” sensation. He reports no recent cough, nasal congestion, or allergies, and he has not been around any sick contacts. His blood pressure is 102/58 mm Hg, pulse is 76 bpm, and body mass index is 27.3 kg/m². His eye exam reveals unilateral conjunctival injections but no hyperemia of the conjunctiva adjacent to the cornea. Mucopurulent discharge was neither found on the undersurface of the eyelid nor emerging from the eye. Which of the following is the best treatment for this patient’s condition?

A) Erythromycin 5 mg/gram ophthalmic ointment.

B) Ofloxacin 0.3% ophthalmic drops.

C) Antihistamine drops.

D) Eye lubricant drops.

E) No treatment necessary.

This patient is an adult presenting with presumed conjunctivitis. Because he is presenting in the summer without observed purulent discharge, his condition is unlikely to be bacterial. This patient does not need treatment, although eye lubricant drops could reduce his discomfort.

Nearly 1% of primary care office visits¹ and 300 million in annual costs² are spent evaluating and treating “pink eye.” After ruling out serious eye disease, clinicians need to determine which cases of suspected conjunctivitis are most likely to be bacterial to allow for judicious use of antibiotic eye drops. This is an important undertaking as most patients assume that antibiotics are needed.

How do we know which history and clinical exam findings to lean on when attempting to categorize conjunctivitis as bacterial or not? If a patient reports purulent discharge, doesn’t that mean it is bacterial? Surprisingly, a systematic review published in 2016 by Narayana and McGee found that a patient’s self-report of “purulent drainage” is diagnostically unhelpful, but if a clinician finds it on exam, the likelihood of a bacterial etiology increases.³

Narayana and McGee analyzed three studies that enrolled a total of 281 patients with presumed conjunctivitis who underwent bacterial cultures. They then determined which findings increased the probability of positive bacterial culture. From strongest to weakest, the best indicators of a bacterial cause were found to be: complete redness of the conjunctival membrane obscuring tarsal vessels (the vessels visible on the inside of everted upper or lower eyelids) (likelihood ratio, 4.6), observed purulent discharge (LR, 3.9), matting of both eyes in the morning (LR, 3.6), and presence during winter/spring months (LR, 1.9). On the other hand, failure to observe a red eye at 20 feet (LR, 0.2), absence of morning gluing of either eye (LR, 0.3), and presentation during summer months (LR, 0.4) all decreased the probability of a bacterial cause. This review and different study by Stenson et al. unfortunately have conflicting evidence regarding whether the following findings are diagnostically helpful: qualities of eye discomfort (such as burning or itching), preauricular adenopathy, conjunctival follicles, and conjunctival papillae.^3,4 Rietveld and colleagues found that a history of conjunctivitis decreased the likelihood of bacterial conjunctivitis.⁵

Pearl: The best indicators of a bacterial cause in patients with presumed conjunctivitis are complete redness of the conjunctival membrane obscuring tarsal vessels, observed purulent discharge, and matting of both eyes in the morning. Presentation during the summer months and having a history of conjunctivitis decreases the likelihood of bacterial conjunctivitis.

Ms. Momany is a fourth-year medical student at University of Washington, Seattle. Dr. Paauw is professor of medicine in the division of general internal medicine at the University of Washington and serves as third-year medical student clerkship director at that university. Contact Dr. Paauw at [email protected].

References

1. Azari AA and Barney NP. JAMA. 2013 Oct 23; 310(16):1721-9.

2. Smith AF and Waycaster C. BMC Ophthalmol. 2009 Nov 25. doi: 10.1186/1471-2415-9-13.

3) Narayana S and McGee S. Am J Med. 2015;128(11):1220-4.e1.

4) Stenson S et al. Arch Ophthalmol. 1982;100(8):1275-7.

5) Rietveld RP et al. BMJ. 2004 Jul 24;329(7459):206-10.

A 54-year-old pharmacist with a history of gout, hypertension, and conjunctivitis presents for evaluation of pink eye in the summer. The morning before coming into the office, he noticed that his right eye was red and inflamed. He self-treated with saline washes and eye drops, but upon awakening the next day, he found his right eye to be crusted shut with surrounding yellow discharge. He has not had any changes to his vision but endorses a somewhat uncomfortable, “gritty” sensation. He reports no recent cough, nasal congestion, or allergies, and he has not been around any sick contacts. His blood pressure is 102/58 mm Hg, pulse is 76 bpm, and body mass index is 27.3 kg/m². His eye exam reveals unilateral conjunctival injections but no hyperemia of the conjunctiva adjacent to the cornea. Mucopurulent discharge was neither found on the undersurface of the eyelid nor emerging from the eye. Which of the following is the best treatment for this patient’s condition?

A) Erythromycin 5 mg/gram ophthalmic ointment.

B) Ofloxacin 0.3% ophthalmic drops.

C) Antihistamine drops.

D) Eye lubricant drops.

E) No treatment necessary.

This patient is an adult presenting with presumed conjunctivitis. Because he is presenting in the summer without observed purulent discharge, his condition is unlikely to be bacterial. This patient does not need treatment, although eye lubricant drops could reduce his discomfort.

Nearly 1% of primary care office visits¹ and 300 million in annual costs² are spent evaluating and treating “pink eye.” After ruling out serious eye disease, clinicians need to determine which cases of suspected conjunctivitis are most likely to be bacterial to allow for judicious use of antibiotic eye drops. This is an important undertaking as most patients assume that antibiotics are needed.

How do we know which history and clinical exam findings to lean on when attempting to categorize conjunctivitis as bacterial or not? If a patient reports purulent discharge, doesn’t that mean it is bacterial? Surprisingly, a systematic review published in 2016 by Narayana and McGee found that a patient’s self-report of “purulent drainage” is diagnostically unhelpful, but if a clinician finds it on exam, the likelihood of a bacterial etiology increases.³

Narayana and McGee analyzed three studies that enrolled a total of 281 patients with presumed conjunctivitis who underwent bacterial cultures. They then determined which findings increased the probability of positive bacterial culture. From strongest to weakest, the best indicators of a bacterial cause were found to be: complete redness of the conjunctival membrane obscuring tarsal vessels (the vessels visible on the inside of everted upper or lower eyelids) (likelihood ratio, 4.6), observed purulent discharge (LR, 3.9), matting of both eyes in the morning (LR, 3.6), and presence during winter/spring months (LR, 1.9). On the other hand, failure to observe a red eye at 20 feet (LR, 0.2), absence of morning gluing of either eye (LR, 0.3), and presentation during summer months (LR, 0.4) all decreased the probability of a bacterial cause. This review and different study by Stenson et al. unfortunately have conflicting evidence regarding whether the following findings are diagnostically helpful: qualities of eye discomfort (such as burning or itching), preauricular adenopathy, conjunctival follicles, and conjunctival papillae.^3,4 Rietveld and colleagues found that a history of conjunctivitis decreased the likelihood of bacterial conjunctivitis.⁵

Pearl: The best indicators of a bacterial cause in patients with presumed conjunctivitis are complete redness of the conjunctival membrane obscuring tarsal vessels, observed purulent discharge, and matting of both eyes in the morning. Presentation during the summer months and having a history of conjunctivitis decreases the likelihood of bacterial conjunctivitis.

Ms. Momany is a fourth-year medical student at University of Washington, Seattle. Dr. Paauw is professor of medicine in the division of general internal medicine at the University of Washington and serves as third-year medical student clerkship director at that university. Contact Dr. Paauw at [email protected].

References

1. Azari AA and Barney NP. JAMA. 2013 Oct 23; 310(16):1721-9.

2. Smith AF and Waycaster C. BMC Ophthalmol. 2009 Nov 25. doi: 10.1186/1471-2415-9-13.

3) Narayana S and McGee S. Am J Med. 2015;128(11):1220-4.e1.

4) Stenson S et al. Arch Ophthalmol. 1982;100(8):1275-7.

5) Rietveld RP et al. BMJ. 2004 Jul 24;329(7459):206-10.

With the help of a key celebrity spokesperson, the American College of Rheumatology is hoping Rheumatic Disease Awareness Month (RDAM) will continue to raise the profile of the related illnesses and help more patients recognize their symptoms and get on the road to treatment.

This year, the celebrity tapped to lead the now-annual campaign that began in September 2016 is tennis champion Venus Williams, who has been competing while battling Sjögren’s syndrome. She was diagnosed in 2011.

Having a high-profile individual like Venus Williams will “bring more awareness to not a specific illness but just rheumatic diseases in general,” Suleman Bhana, MD, chair of the ACR’s marketing and communications committee, said in an interview, adding that it is a way to help get attention from patients and their family members to be more aware of what they are facing when afflicted with a rheumatic disease.

It also helps to drive traffic to a website (simpletasks.org) set up to help build a community around rheumatic diseases, he noted.

“By signing up on the website, patients and others can get connected with wellness articles and can be informed about any advocacy opportunities in their local area or the national level,” said Dr. Bhana, a rheumatologist with Crystal Run Healthcare in Middletown, N.Y. “It helps us a lot from an ACR point of view just by getting the word out about rheumatic diseases.”

Dr. Bhana said that part of the success of the RDAM campaign is being measured by the number of people who sign up for the website. “Last year we had about 1,600 people sign up, which was more than double from the previous year and we are hoping to do even better this year now that we have Venus Williams as our spokesperson.”

But the campaign involves more than just a celebrity spokesperson and an online community to build awareness for rheumatic diseases and their associated symptoms and treatments. The ACR is working with patients to make sure their voices are heard on Capitol Hill to help ensure proper access to medical care and those treatments.

This year, the ACR – along with more than 100 advocates – met with members of Congress to push for policies that will benefit people with rheumatic diseases. One key item on the agenda targets the use of step therapy.

“This year the ACR’s Government Affairs Committee had a list of several pieces of legislation that they are looking at heavily. [One bill is about] having brakes on what is called step therapy, which is a fairly egregious practice by many insurers that forces patients to use different medications than what the physician and patient decide in their mutual doctor-patient relationship before they can use the medicine that was originally decided upon,” he said. “The reasons for this practice mostly are financial in that insurers get kickbacks from certain pharmaceutical companies, and that is what they term as preferred drugs. It’s a lot more than just cost savings. It’s about the insurers financially benefiting from patients rather than doing the right thing and preserving the doctor-patient relationship.”

One bill in particular that has been highlighted by the ACR is the Safe Step Act of 2019 (H.R. 2279), introduced by Rep. Raul Ruiz, MD (D-Calif.), and Rep. Brad Wenstrup, DPM (R-Ohio), would put limits on step therapy and create a clear process for patients and physicians to seek exemptions.

Other legislative actions that patients and the ACR were advocating for during the visits for RDAM included the EMPOWER for Health Act (H.R. 2781), which would help increase the number of pediatric subspecialists, including pediatric rheumatologists, through loan repayment for health professionals who agree to work at least 2 years in pediatric medicine, and the REDI Act (H.R. 1554), which would defer the accumulation on student loan interest while future doctors serve in a medical internship or residency program.

“The reality of pediatrics is that they don’t get well compensated as a pediatric rheumatologist subspecialist, so there is an ultimate financial disincentive for training pediatricians to go into rheumatology,” Dr. Bhana said. “So we are trying to get support for loan repayment for pediatric rheumatologists or rheumatology fellowship programs, which may help to incentivize young pediatricians to go into rheumatology to at least expand access to care.”

The RDAM campaign also provides useful contacts for an annual survey that the ACR conducts. This year’s survey found that access issues for both physicians and treatments persist, informing the agenda for the advocacy on Capitol Hill.

For example, the 2019 survey found that more than 60% of respondents had to wait at least 31 days from a physician referral to an initial rheumatologist appointment. Dr. Bhana noted that, in his practice, the wait can be 3 months or longer. A little more than 36% of respondents were able to get that initial appointment after referral in 30 days or less.

Additionally, nearly 47% of respondents were subjected to step therapy in the past year.

Treatment pricing was also raised as a concern in the survey, with a little more than 57% of respondents reporting difficulty affording treatments in the past year, and 25% reporting annual out-of-pocket spending at greater than $1,000, with more than 6% reporting annual out-of-pocket spending of greater than $5,000.

Despite all the challenges that lay ahead, Dr. Bhana believes that, as the campaign continues in its fourth year, it is having a positive impact.

“I think there is more awareness among patients to look up their symptoms and prompt their providers to look into, if not test for, rheumatic diseases, direct referrals, and I think because of these campaigns, we’ve gotten some indication that from a social media awareness that more patients are talking about it, about trying to get into seeing a physician.”

[email protected]

With the help of a key celebrity spokesperson, the American College of Rheumatology is hoping Rheumatic Disease Awareness Month (RDAM) will continue to raise the profile of the related illnesses and help more patients recognize their symptoms and get on the road to treatment.

This year, the celebrity tapped to lead the now-annual campaign that began in September 2016 is tennis champion Venus Williams, who has been competing while battling Sjögren’s syndrome. She was diagnosed in 2011.

Having a high-profile individual like Venus Williams will “bring more awareness to not a specific illness but just rheumatic diseases in general,” Suleman Bhana, MD, chair of the ACR’s marketing and communications committee, said in an interview, adding that it is a way to help get attention from patients and their family members to be more aware of what they are facing when afflicted with a rheumatic disease.

It also helps to drive traffic to a website (simpletasks.org) set up to help build a community around rheumatic diseases, he noted.

“By signing up on the website, patients and others can get connected with wellness articles and can be informed about any advocacy opportunities in their local area or the national level,” said Dr. Bhana, a rheumatologist with Crystal Run Healthcare in Middletown, N.Y. “It helps us a lot from an ACR point of view just by getting the word out about rheumatic diseases.”

Dr. Bhana said that part of the success of the RDAM campaign is being measured by the number of people who sign up for the website. “Last year we had about 1,600 people sign up, which was more than double from the previous year and we are hoping to do even better this year now that we have Venus Williams as our spokesperson.”

But the campaign involves more than just a celebrity spokesperson and an online community to build awareness for rheumatic diseases and their associated symptoms and treatments. The ACR is working with patients to make sure their voices are heard on Capitol Hill to help ensure proper access to medical care and those treatments.

This year, the ACR – along with more than 100 advocates – met with members of Congress to push for policies that will benefit people with rheumatic diseases. One key item on the agenda targets the use of step therapy.

“This year the ACR’s Government Affairs Committee had a list of several pieces of legislation that they are looking at heavily. [One bill is about] having brakes on what is called step therapy, which is a fairly egregious practice by many insurers that forces patients to use different medications than what the physician and patient decide in their mutual doctor-patient relationship before they can use the medicine that was originally decided upon,” he said. “The reasons for this practice mostly are financial in that insurers get kickbacks from certain pharmaceutical companies, and that is what they term as preferred drugs. It’s a lot more than just cost savings. It’s about the insurers financially benefiting from patients rather than doing the right thing and preserving the doctor-patient relationship.”

One bill in particular that has been highlighted by the ACR is the Safe Step Act of 2019 (H.R. 2279), introduced by Rep. Raul Ruiz, MD (D-Calif.), and Rep. Brad Wenstrup, DPM (R-Ohio), would put limits on step therapy and create a clear process for patients and physicians to seek exemptions.

Other legislative actions that patients and the ACR were advocating for during the visits for RDAM included the EMPOWER for Health Act (H.R. 2781), which would help increase the number of pediatric subspecialists, including pediatric rheumatologists, through loan repayment for health professionals who agree to work at least 2 years in pediatric medicine, and the REDI Act (H.R. 1554), which would defer the accumulation on student loan interest while future doctors serve in a medical internship or residency program.

“The reality of pediatrics is that they don’t get well compensated as a pediatric rheumatologist subspecialist, so there is an ultimate financial disincentive for training pediatricians to go into rheumatology,” Dr. Bhana said. “So we are trying to get support for loan repayment for pediatric rheumatologists or rheumatology fellowship programs, which may help to incentivize young pediatricians to go into rheumatology to at least expand access to care.”

The RDAM campaign also provides useful contacts for an annual survey that the ACR conducts. This year’s survey found that access issues for both physicians and treatments persist, informing the agenda for the advocacy on Capitol Hill.

For example, the 2019 survey found that more than 60% of respondents had to wait at least 31 days from a physician referral to an initial rheumatologist appointment. Dr. Bhana noted that, in his practice, the wait can be 3 months or longer. A little more than 36% of respondents were able to get that initial appointment after referral in 30 days or less.

Additionally, nearly 47% of respondents were subjected to step therapy in the past year.

Treatment pricing was also raised as a concern in the survey, with a little more than 57% of respondents reporting difficulty affording treatments in the past year, and 25% reporting annual out-of-pocket spending at greater than $1,000, with more than 6% reporting annual out-of-pocket spending of greater than $5,000.

Despite all the challenges that lay ahead, Dr. Bhana believes that, as the campaign continues in its fourth year, it is having a positive impact.

“I think there is more awareness among patients to look up their symptoms and prompt their providers to look into, if not test for, rheumatic diseases, direct referrals, and I think because of these campaigns, we’ve gotten some indication that from a social media awareness that more patients are talking about it, about trying to get into seeing a physician.”

[email protected]

With the help of a key celebrity spokesperson, the American College of Rheumatology is hoping Rheumatic Disease Awareness Month (RDAM) will continue to raise the profile of the related illnesses and help more patients recognize their symptoms and get on the road to treatment.

This year, the celebrity tapped to lead the now-annual campaign that began in September 2016 is tennis champion Venus Williams, who has been competing while battling Sjögren’s syndrome. She was diagnosed in 2011.

Having a high-profile individual like Venus Williams will “bring more awareness to not a specific illness but just rheumatic diseases in general,” Suleman Bhana, MD, chair of the ACR’s marketing and communications committee, said in an interview, adding that it is a way to help get attention from patients and their family members to be more aware of what they are facing when afflicted with a rheumatic disease.

It also helps to drive traffic to a website (simpletasks.org) set up to help build a community around rheumatic diseases, he noted.

“By signing up on the website, patients and others can get connected with wellness articles and can be informed about any advocacy opportunities in their local area or the national level,” said Dr. Bhana, a rheumatologist with Crystal Run Healthcare in Middletown, N.Y. “It helps us a lot from an ACR point of view just by getting the word out about rheumatic diseases.”

Dr. Bhana said that part of the success of the RDAM campaign is being measured by the number of people who sign up for the website. “Last year we had about 1,600 people sign up, which was more than double from the previous year and we are hoping to do even better this year now that we have Venus Williams as our spokesperson.”

But the campaign involves more than just a celebrity spokesperson and an online community to build awareness for rheumatic diseases and their associated symptoms and treatments. The ACR is working with patients to make sure their voices are heard on Capitol Hill to help ensure proper access to medical care and those treatments.

This year, the ACR – along with more than 100 advocates – met with members of Congress to push for policies that will benefit people with rheumatic diseases. One key item on the agenda targets the use of step therapy.

“This year the ACR’s Government Affairs Committee had a list of several pieces of legislation that they are looking at heavily. [One bill is about] having brakes on what is called step therapy, which is a fairly egregious practice by many insurers that forces patients to use different medications than what the physician and patient decide in their mutual doctor-patient relationship before they can use the medicine that was originally decided upon,” he said. “The reasons for this practice mostly are financial in that insurers get kickbacks from certain pharmaceutical companies, and that is what they term as preferred drugs. It’s a lot more than just cost savings. It’s about the insurers financially benefiting from patients rather than doing the right thing and preserving the doctor-patient relationship.”

One bill in particular that has been highlighted by the ACR is the Safe Step Act of 2019 (H.R. 2279), introduced by Rep. Raul Ruiz, MD (D-Calif.), and Rep. Brad Wenstrup, DPM (R-Ohio), would put limits on step therapy and create a clear process for patients and physicians to seek exemptions.

Other legislative actions that patients and the ACR were advocating for during the visits for RDAM included the EMPOWER for Health Act (H.R. 2781), which would help increase the number of pediatric subspecialists, including pediatric rheumatologists, through loan repayment for health professionals who agree to work at least 2 years in pediatric medicine, and the REDI Act (H.R. 1554), which would defer the accumulation on student loan interest while future doctors serve in a medical internship or residency program.

“The reality of pediatrics is that they don’t get well compensated as a pediatric rheumatologist subspecialist, so there is an ultimate financial disincentive for training pediatricians to go into rheumatology,” Dr. Bhana said. “So we are trying to get support for loan repayment for pediatric rheumatologists or rheumatology fellowship programs, which may help to incentivize young pediatricians to go into rheumatology to at least expand access to care.”

The RDAM campaign also provides useful contacts for an annual survey that the ACR conducts. This year’s survey found that access issues for both physicians and treatments persist, informing the agenda for the advocacy on Capitol Hill.

For example, the 2019 survey found that more than 60% of respondents had to wait at least 31 days from a physician referral to an initial rheumatologist appointment. Dr. Bhana noted that, in his practice, the wait can be 3 months or longer. A little more than 36% of respondents were able to get that initial appointment after referral in 30 days or less.

Additionally, nearly 47% of respondents were subjected to step therapy in the past year.

Treatment pricing was also raised as a concern in the survey, with a little more than 57% of respondents reporting difficulty affording treatments in the past year, and 25% reporting annual out-of-pocket spending at greater than $1,000, with more than 6% reporting annual out-of-pocket spending of greater than $5,000.

Despite all the challenges that lay ahead, Dr. Bhana believes that, as the campaign continues in its fourth year, it is having a positive impact.

“I think there is more awareness among patients to look up their symptoms and prompt their providers to look into, if not test for, rheumatic diseases, direct referrals, and I think because of these campaigns, we’ve gotten some indication that from a social media awareness that more patients are talking about it, about trying to get into seeing a physician.”

[email protected]

As youth suicides continue to climb nationwide, a growing body of research shows that the deaths are happening at higher rates in rural communities.

Courtesy Dr. Kriechman

In 2017, suicides reached their highest point since 2000, a trend driven by a sharp rise in male suicides and in youth aged 15-19 years, according to an analysis published recently in JAMA (2019 Jun 18. doi: 10.1001/jama.2019.5054). Among youth aged 15-19 years, the suicide rate was 12 per 100,000 in 2017 (18 per 100,000 in males and 5 per 100,000 in females), compared with 8 per 100,000 in 2000, the study found. Across all age groups, the highest suicide rates and greatest rate increases are in rural counties, according to data from the Centers for Disease Control and Prevention (CDC).

Now, a unique initiative in New Mexico is working to combat those alarming trends through an alliance of community leaders that strives to strengthen resilience and build peer support for at-risk youth.

The Alliance-Building for Suicide Prevention & Youth Resilience (ASPYR) program, created by the University of New Mexico (UNM), Albuquerque, focuses on training professionals and advocates within New Mexico communities in a strength-based, youth-directed, collaborative approach for the assessment and treatment of suicidality. A diversity of community members undergo the training, including health and behavioral health care providers, peer support and community support workers, youth and community advocates, educators, and first responders. The initiative also supports and facilitates the development of a communitywide crisis intervention plan that promotes youth safety and resilience.

“ASPYR is unique, in that we actively involve youth to guide our program, versus an adult-only led program,” says Laura Rombach, program manager for ASPYR and a senior program therapist in the department of psychiatry and behavioral sciences at UNM. “Youth offer feedback about our training and ideas about how to best prevent suicide in their schools and communities. New Mexico is underresourced, and individuals living in rural/frontier areas do not always have access to licensed behavioral health providers, so our training is developed for licensed providers as well as peers and paraprofessionals to increase the knowledge of care for individuals experiencing a suicidal crisis.”
 

Rural populations present challenges

The many rural pockets of New Mexico pose numerous obstacles for antisuicide advocates.

Of the 33 counties in New Mexico, six are identified by the Census Bureau as completely “rural,” and an additional six are defined as mostly rural, according to the University of New Mexico Bureau of Business & Economic Research. Even among counties considered “urban” however, a considerable amount of the population lives in rural areas, according to the bureau. San Juan County, for example, which is considered urban by the Census Bureau, had an estimated 34% of residents living in rural areas in 2010.

Poverty adds to the difficulty. In 2017, nearly one in five New Mexicans (20%) lived below the poverty line, and the state had the second-highest rate of children under 18 years living in poverty in the country, according to a report by the New Mexico Department of Workforce Solutions.

“New Mexico is an impoverished state with limited capacity, especially in regards to behavioral health services,” said Avi Kriechman, MD, principal investigator for ASPYR at UNM and a child, adolescent, and family psychiatrist at the university. “It is also challenging to create a truly statewide effort where there is limited public transportation, problematic Internet connection, and other barriers to involving those who live and work in rural and frontier New Mexico.”

Addressing suicide among the many native and Indigenous people in rural New Mexico presents another unique set of challenges, said Mary Roessel, MD, a Santa Fe, N.M.–based psychiatrist who specializes in cultural psychiatry. Native and Indigenous residents often have a general mistrust of outsiders and a stigma against mental illnesses, Dr. Roessel said in an interview.

“One of the problems is being able to identify when a person has attempted suicide in some of these small, private, Pueblo communities because they are very closed,” she said. “At times, we don’t get the information to go in and help them. They’re trying to address or deal with the problem themselves.”

To address the many barriers of rural New Mexico, ASPYR works hard to recognize, identify, and support preexisting community resources that are often neglected in needs assessment and stakeholder identification, Dr. Kriechman said. This can include food banks, church care committees, youth advocacy groups, local caregiving, and spiritual traditions, among others. Frequently, many community caregivers and agencies have not connected or communicated with one another and often are unaware of all they have to offer, he said.

“We try to build capacity through community trainings, which include a widely diverse group of providers, advocates, and supports,” he continued. “Our trainings involve highlighting and building upon local and cultural practices and traditions of healing, caregiving, and support. A significant part of our onsite training involves assembling a representative group of local providers in health care, behavioral health care, peer & community support and advocacy, education, first responders to community crises, and government and nonprofit agencies, then facilitating a community conversation between the panel and training attendees about how best to move forward in a synergistic and systemic manner to support youth safety and resilience.”
 

Peers support peers

While ASPYR encompasses elements of other suicide prevention models, two unique cornerstones of the program are its emphasis on resilience and promotion of peer support. The strength-based, youth-directed approach includes creating a youth-directed safety plan, enlisting peers as support and reducing access to lethal means.

Regarding the youth safety plan, Dr. Kriechman explained that, rather than being prescribed and instructed in expert-selected and expert-driven coping skills, youth are offered a menu of options that most speak to their strengths, values, experience, and preferences. Young people also select a peer who, if they wish, accompanies them to sessions, and supports and coaches them at home.

“Peers are often more influential than parents, siblings, family members, and adults regarding youth behavior,” Dr. Kriechman said. “Most often, it is a peer that a youth-at-risk turns to for support, counsel, role models, and understanding. Youth who wish to offer their peers support can quickly be trained to provide early identification of youth at risk, motivational support to seek help, and a ‘warm hand-off’ to community resources.”

In addition, a Youth Advisory Council established as part of the program draws from young people across New Mexico to participate in state and national conferences, and conduct outreach efforts to peers.

ASPYR Youth council member Serenity Gomez, a senior at the Public Academy for Performing Arts in Albuquerque, became interested in ASPYR after volunteering for the American Foundation for Suicide Prevention in 2016. As a youth council member, Ms. Gomez said she helps create projects to raise suicide awareness, whether through posters, stickers, social media, poetry, or songs.

“My experience as a youth council member has really opened my eyes and has made me more motivated to help others,” she said in an interview. “It has also showed me that talking about suicide doesn’t always have to be a slideshow of facts. You can reach people through music, poetry, storytelling, and so much more. Many people are afraid to talk about suicide because it’s such a scary idea, but if we all talk about it and bring more awareness, then we can find the support everyone needs. In ASPYR, specifically, I hope to reach youth and help all youth learn to support each other.”

Since ASPYR launched in 2017, the program has provided both onsite and online trainings to hundreds of New Mexicans, and has helped rural and frontier communities start working on collaborative approaches to promoting youth safety and resilience, Dr. Kriechman said. Following community consultations, numerous rural communities have since formed systems of care to identify, support, and treat youth at risk. In addition to the youth council, an Advisory Community Council has also been established that welcomes any New Mexico resident interested in working on the mission of preventing youth suicide.

The program’s approach of focusing on strengths, rather than deficits, has resonated strongly with community providers with whom the program partners, Dr. Kriechman added. For example, the program shifts from “no-suicide contracts” to safety planning, focusing on reasons for living rather than reasons for dying, and shifting from prescribing coping skills to strengthening preexisting coping skills in young people.

“An ultimate hope for ASPYR is emphasizing that recovery from any of life’s challenges is far more than symptom reduction or agency collaboration,” Dr. Kriechman said. “It is the understanding that a life of value and meaning, the instillation of hope and support for the unique strengths, competencies, skills, and understandings of each individual, is honored, respected, and supported.”

[email protected]

As youth suicides continue to climb nationwide, a growing body of research shows that the deaths are happening at higher rates in rural communities.

Courtesy Dr. Kriechman

In 2017, suicides reached their highest point since 2000, a trend driven by a sharp rise in male suicides and in youth aged 15-19 years, according to an analysis published recently in JAMA (2019 Jun 18. doi: 10.1001/jama.2019.5054). Among youth aged 15-19 years, the suicide rate was 12 per 100,000 in 2017 (18 per 100,000 in males and 5 per 100,000 in females), compared with 8 per 100,000 in 2000, the study found. Across all age groups, the highest suicide rates and greatest rate increases are in rural counties, according to data from the Centers for Disease Control and Prevention (CDC).

Now, a unique initiative in New Mexico is working to combat those alarming trends through an alliance of community leaders that strives to strengthen resilience and build peer support for at-risk youth.

The Alliance-Building for Suicide Prevention & Youth Resilience (ASPYR) program, created by the University of New Mexico (UNM), Albuquerque, focuses on training professionals and advocates within New Mexico communities in a strength-based, youth-directed, collaborative approach for the assessment and treatment of suicidality. A diversity of community members undergo the training, including health and behavioral health care providers, peer support and community support workers, youth and community advocates, educators, and first responders. The initiative also supports and facilitates the development of a communitywide crisis intervention plan that promotes youth safety and resilience.

“ASPYR is unique, in that we actively involve youth to guide our program, versus an adult-only led program,” says Laura Rombach, program manager for ASPYR and a senior program therapist in the department of psychiatry and behavioral sciences at UNM. “Youth offer feedback about our training and ideas about how to best prevent suicide in their schools and communities. New Mexico is underresourced, and individuals living in rural/frontier areas do not always have access to licensed behavioral health providers, so our training is developed for licensed providers as well as peers and paraprofessionals to increase the knowledge of care for individuals experiencing a suicidal crisis.”
 

Rural populations present challenges

The many rural pockets of New Mexico pose numerous obstacles for antisuicide advocates.

Of the 33 counties in New Mexico, six are identified by the Census Bureau as completely “rural,” and an additional six are defined as mostly rural, according to the University of New Mexico Bureau of Business & Economic Research. Even among counties considered “urban” however, a considerable amount of the population lives in rural areas, according to the bureau. San Juan County, for example, which is considered urban by the Census Bureau, had an estimated 34% of residents living in rural areas in 2010.

Poverty adds to the difficulty. In 2017, nearly one in five New Mexicans (20%) lived below the poverty line, and the state had the second-highest rate of children under 18 years living in poverty in the country, according to a report by the New Mexico Department of Workforce Solutions.

“New Mexico is an impoverished state with limited capacity, especially in regards to behavioral health services,” said Avi Kriechman, MD, principal investigator for ASPYR at UNM and a child, adolescent, and family psychiatrist at the university. “It is also challenging to create a truly statewide effort where there is limited public transportation, problematic Internet connection, and other barriers to involving those who live and work in rural and frontier New Mexico.”

Addressing suicide among the many native and Indigenous people in rural New Mexico presents another unique set of challenges, said Mary Roessel, MD, a Santa Fe, N.M.–based psychiatrist who specializes in cultural psychiatry. Native and Indigenous residents often have a general mistrust of outsiders and a stigma against mental illnesses, Dr. Roessel said in an interview.

“One of the problems is being able to identify when a person has attempted suicide in some of these small, private, Pueblo communities because they are very closed,” she said. “At times, we don’t get the information to go in and help them. They’re trying to address or deal with the problem themselves.”

To address the many barriers of rural New Mexico, ASPYR works hard to recognize, identify, and support preexisting community resources that are often neglected in needs assessment and stakeholder identification, Dr. Kriechman said. This can include food banks, church care committees, youth advocacy groups, local caregiving, and spiritual traditions, among others. Frequently, many community caregivers and agencies have not connected or communicated with one another and often are unaware of all they have to offer, he said.

“We try to build capacity through community trainings, which include a widely diverse group of providers, advocates, and supports,” he continued. “Our trainings involve highlighting and building upon local and cultural practices and traditions of healing, caregiving, and support. A significant part of our onsite training involves assembling a representative group of local providers in health care, behavioral health care, peer & community support and advocacy, education, first responders to community crises, and government and nonprofit agencies, then facilitating a community conversation between the panel and training attendees about how best to move forward in a synergistic and systemic manner to support youth safety and resilience.”
 

Peers support peers

While ASPYR encompasses elements of other suicide prevention models, two unique cornerstones of the program are its emphasis on resilience and promotion of peer support. The strength-based, youth-directed approach includes creating a youth-directed safety plan, enlisting peers as support and reducing access to lethal means.

Regarding the youth safety plan, Dr. Kriechman explained that, rather than being prescribed and instructed in expert-selected and expert-driven coping skills, youth are offered a menu of options that most speak to their strengths, values, experience, and preferences. Young people also select a peer who, if they wish, accompanies them to sessions, and supports and coaches them at home.

“Peers are often more influential than parents, siblings, family members, and adults regarding youth behavior,” Dr. Kriechman said. “Most often, it is a peer that a youth-at-risk turns to for support, counsel, role models, and understanding. Youth who wish to offer their peers support can quickly be trained to provide early identification of youth at risk, motivational support to seek help, and a ‘warm hand-off’ to community resources.”

In addition, a Youth Advisory Council established as part of the program draws from young people across New Mexico to participate in state and national conferences, and conduct outreach efforts to peers.

ASPYR Youth council member Serenity Gomez, a senior at the Public Academy for Performing Arts in Albuquerque, became interested in ASPYR after volunteering for the American Foundation for Suicide Prevention in 2016. As a youth council member, Ms. Gomez said she helps create projects to raise suicide awareness, whether through posters, stickers, social media, poetry, or songs.

“My experience as a youth council member has really opened my eyes and has made me more motivated to help others,” she said in an interview. “It has also showed me that talking about suicide doesn’t always have to be a slideshow of facts. You can reach people through music, poetry, storytelling, and so much more. Many people are afraid to talk about suicide because it’s such a scary idea, but if we all talk about it and bring more awareness, then we can find the support everyone needs. In ASPYR, specifically, I hope to reach youth and help all youth learn to support each other.”

Since ASPYR launched in 2017, the program has provided both onsite and online trainings to hundreds of New Mexicans, and has helped rural and frontier communities start working on collaborative approaches to promoting youth safety and resilience, Dr. Kriechman said. Following community consultations, numerous rural communities have since formed systems of care to identify, support, and treat youth at risk. In addition to the youth council, an Advisory Community Council has also been established that welcomes any New Mexico resident interested in working on the mission of preventing youth suicide.

The program’s approach of focusing on strengths, rather than deficits, has resonated strongly with community providers with whom the program partners, Dr. Kriechman added. For example, the program shifts from “no-suicide contracts” to safety planning, focusing on reasons for living rather than reasons for dying, and shifting from prescribing coping skills to strengthening preexisting coping skills in young people.

“An ultimate hope for ASPYR is emphasizing that recovery from any of life’s challenges is far more than symptom reduction or agency collaboration,” Dr. Kriechman said. “It is the understanding that a life of value and meaning, the instillation of hope and support for the unique strengths, competencies, skills, and understandings of each individual, is honored, respected, and supported.”

[email protected]

As youth suicides continue to climb nationwide, a growing body of research shows that the deaths are happening at higher rates in rural communities.

Courtesy Dr. Kriechman

In 2017, suicides reached their highest point since 2000, a trend driven by a sharp rise in male suicides and in youth aged 15-19 years, according to an analysis published recently in JAMA (2019 Jun 18. doi: 10.1001/jama.2019.5054). Among youth aged 15-19 years, the suicide rate was 12 per 100,000 in 2017 (18 per 100,000 in males and 5 per 100,000 in females), compared with 8 per 100,000 in 2000, the study found. Across all age groups, the highest suicide rates and greatest rate increases are in rural counties, according to data from the Centers for Disease Control and Prevention (CDC).

Now, a unique initiative in New Mexico is working to combat those alarming trends through an alliance of community leaders that strives to strengthen resilience and build peer support for at-risk youth.

The Alliance-Building for Suicide Prevention & Youth Resilience (ASPYR) program, created by the University of New Mexico (UNM), Albuquerque, focuses on training professionals and advocates within New Mexico communities in a strength-based, youth-directed, collaborative approach for the assessment and treatment of suicidality. A diversity of community members undergo the training, including health and behavioral health care providers, peer support and community support workers, youth and community advocates, educators, and first responders. The initiative also supports and facilitates the development of a communitywide crisis intervention plan that promotes youth safety and resilience.

“ASPYR is unique, in that we actively involve youth to guide our program, versus an adult-only led program,” says Laura Rombach, program manager for ASPYR and a senior program therapist in the department of psychiatry and behavioral sciences at UNM. “Youth offer feedback about our training and ideas about how to best prevent suicide in their schools and communities. New Mexico is underresourced, and individuals living in rural/frontier areas do not always have access to licensed behavioral health providers, so our training is developed for licensed providers as well as peers and paraprofessionals to increase the knowledge of care for individuals experiencing a suicidal crisis.”
 

Rural populations present challenges

The many rural pockets of New Mexico pose numerous obstacles for antisuicide advocates.

Of the 33 counties in New Mexico, six are identified by the Census Bureau as completely “rural,” and an additional six are defined as mostly rural, according to the University of New Mexico Bureau of Business & Economic Research. Even among counties considered “urban” however, a considerable amount of the population lives in rural areas, according to the bureau. San Juan County, for example, which is considered urban by the Census Bureau, had an estimated 34% of residents living in rural areas in 2010.

Poverty adds to the difficulty. In 2017, nearly one in five New Mexicans (20%) lived below the poverty line, and the state had the second-highest rate of children under 18 years living in poverty in the country, according to a report by the New Mexico Department of Workforce Solutions.

“New Mexico is an impoverished state with limited capacity, especially in regards to behavioral health services,” said Avi Kriechman, MD, principal investigator for ASPYR at UNM and a child, adolescent, and family psychiatrist at the university. “It is also challenging to create a truly statewide effort where there is limited public transportation, problematic Internet connection, and other barriers to involving those who live and work in rural and frontier New Mexico.”

Addressing suicide among the many native and Indigenous people in rural New Mexico presents another unique set of challenges, said Mary Roessel, MD, a Santa Fe, N.M.–based psychiatrist who specializes in cultural psychiatry. Native and Indigenous residents often have a general mistrust of outsiders and a stigma against mental illnesses, Dr. Roessel said in an interview.

“One of the problems is being able to identify when a person has attempted suicide in some of these small, private, Pueblo communities because they are very closed,” she said. “At times, we don’t get the information to go in and help them. They’re trying to address or deal with the problem themselves.”

To address the many barriers of rural New Mexico, ASPYR works hard to recognize, identify, and support preexisting community resources that are often neglected in needs assessment and stakeholder identification, Dr. Kriechman said. This can include food banks, church care committees, youth advocacy groups, local caregiving, and spiritual traditions, among others. Frequently, many community caregivers and agencies have not connected or communicated with one another and often are unaware of all they have to offer, he said.

“We try to build capacity through community trainings, which include a widely diverse group of providers, advocates, and supports,” he continued. “Our trainings involve highlighting and building upon local and cultural practices and traditions of healing, caregiving, and support. A significant part of our onsite training involves assembling a representative group of local providers in health care, behavioral health care, peer & community support and advocacy, education, first responders to community crises, and government and nonprofit agencies, then facilitating a community conversation between the panel and training attendees about how best to move forward in a synergistic and systemic manner to support youth safety and resilience.”
 

Peers support peers

While ASPYR encompasses elements of other suicide prevention models, two unique cornerstones of the program are its emphasis on resilience and promotion of peer support. The strength-based, youth-directed approach includes creating a youth-directed safety plan, enlisting peers as support and reducing access to lethal means.

Regarding the youth safety plan, Dr. Kriechman explained that, rather than being prescribed and instructed in expert-selected and expert-driven coping skills, youth are offered a menu of options that most speak to their strengths, values, experience, and preferences. Young people also select a peer who, if they wish, accompanies them to sessions, and supports and coaches them at home.

“Peers are often more influential than parents, siblings, family members, and adults regarding youth behavior,” Dr. Kriechman said. “Most often, it is a peer that a youth-at-risk turns to for support, counsel, role models, and understanding. Youth who wish to offer their peers support can quickly be trained to provide early identification of youth at risk, motivational support to seek help, and a ‘warm hand-off’ to community resources.”

In addition, a Youth Advisory Council established as part of the program draws from young people across New Mexico to participate in state and national conferences, and conduct outreach efforts to peers.

ASPYR Youth council member Serenity Gomez, a senior at the Public Academy for Performing Arts in Albuquerque, became interested in ASPYR after volunteering for the American Foundation for Suicide Prevention in 2016. As a youth council member, Ms. Gomez said she helps create projects to raise suicide awareness, whether through posters, stickers, social media, poetry, or songs.

“My experience as a youth council member has really opened my eyes and has made me more motivated to help others,” she said in an interview. “It has also showed me that talking about suicide doesn’t always have to be a slideshow of facts. You can reach people through music, poetry, storytelling, and so much more. Many people are afraid to talk about suicide because it’s such a scary idea, but if we all talk about it and bring more awareness, then we can find the support everyone needs. In ASPYR, specifically, I hope to reach youth and help all youth learn to support each other.”

Since ASPYR launched in 2017, the program has provided both onsite and online trainings to hundreds of New Mexicans, and has helped rural and frontier communities start working on collaborative approaches to promoting youth safety and resilience, Dr. Kriechman said. Following community consultations, numerous rural communities have since formed systems of care to identify, support, and treat youth at risk. In addition to the youth council, an Advisory Community Council has also been established that welcomes any New Mexico resident interested in working on the mission of preventing youth suicide.

The program’s approach of focusing on strengths, rather than deficits, has resonated strongly with community providers with whom the program partners, Dr. Kriechman added. For example, the program shifts from “no-suicide contracts” to safety planning, focusing on reasons for living rather than reasons for dying, and shifting from prescribing coping skills to strengthening preexisting coping skills in young people.

“An ultimate hope for ASPYR is emphasizing that recovery from any of life’s challenges is far more than symptom reduction or agency collaboration,” Dr. Kriechman said. “It is the understanding that a life of value and meaning, the instillation of hope and support for the unique strengths, competencies, skills, and understandings of each individual, is honored, respected, and supported.”

[email protected]

You are seeing a 9-year-old for her annual health maintenance visit. A quick look at her growth chart easily confirms your first impression that she is obese. How are you going to address the weight that you know, and she probably suspects, is going to make her vulnerable to a myriad of health problems as she gets older?

SolStock/iStock/Getty Image

If she has been your patient since she was in preschool, this is certainly not the first time that her growth chart has been concerning. When did you first start discussing her weight with her parents? What words did you use? What strategies have you suggested? What referrals have you made? Maybe you have already given up and decided to not even “go there” at this visit because your experience with overweight patients has been so disappointing.

In her op ed in the New York Times, Dr. Perri Klass reconsiders these kinds of questions as she reviews an article in the journal Childhood Obesity (“Let’s Not Just Dismiss the Weight Watchers Kurbo App,” by Michelle I. Cardel, PhD, MS, RD, and Elsie M. Taveras, MD, MPH, August 2019) written by a nutrition scientist and a pediatrician who are concerned about a new weight loss app for children recently released by Weight Watchers. (The Checkup, “Helping Children Learn to Eat Well,” The New York Times, Aug. 26, 2019). Although the authors of the journal article question some of the science behind the app, their primary concerns are that the app is aimed at children without a way to guarantee parental involvement, and in their opinion the app also places too much emphasis on weight loss.

Their concerns go right to the heart of what troubles me about managing obesity in children. How should I talk to a child about her weight? What words can I choose without shaming? Maybe I shouldn’t be talking to her at all. When a child is 18 months old, we don’t talk to her about her growth chart. Not because she couldn’t understand, but because the solution rests not with her but with her parents.

At what age does a child’s obesity cease being simply a parenting problem and instead become something the child must solve? Does that point come when we have given up on the parents’ ability to create and maintain an environment that discourages obesity? Is that the point when we begin asking the child to unlearn a complex set of behaviors that have been enabled or at least tolerated and poorly modeled at home?

When we begin to talk to a child about his weight do we begin by telling him that he may not have been a contributor to the problem when it began but from now on he needs to be a major player in its management? Of course we don’t share that reality with an 8-year-old, but sometime during his struggle to manage his weight he will connect the dots.

If you are beginning to suspect that I have built my pediatric career around a scaffolding of parent blaming and shaming you are wrong. I know that there are children who have inherited a suite of genes that make them vulnerable to obesity. And I know that too many children grow up in environments in which their parents are powerless to control the family diet for economic reasons. But I am sure that like me you mutter to yourself and your colleagues about the number of patients you are seeing each day whose growth charts are a clear reflection of less than optimal parenting.

Does all of this mean we throw in the towel and stop trying to help overweight children after they turn 6 years old? Of course not. But, it does mean we must redouble our efforts to help parents manage their children’s diets and activity levels in those first critical preschool years.
 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

You are seeing a 9-year-old for her annual health maintenance visit. A quick look at her growth chart easily confirms your first impression that she is obese. How are you going to address the weight that you know, and she probably suspects, is going to make her vulnerable to a myriad of health problems as she gets older?

SolStock/iStock/Getty Image

If she has been your patient since she was in preschool, this is certainly not the first time that her growth chart has been concerning. When did you first start discussing her weight with her parents? What words did you use? What strategies have you suggested? What referrals have you made? Maybe you have already given up and decided to not even “go there” at this visit because your experience with overweight patients has been so disappointing.

In her op ed in the New York Times, Dr. Perri Klass reconsiders these kinds of questions as she reviews an article in the journal Childhood Obesity (“Let’s Not Just Dismiss the Weight Watchers Kurbo App,” by Michelle I. Cardel, PhD, MS, RD, and Elsie M. Taveras, MD, MPH, August 2019) written by a nutrition scientist and a pediatrician who are concerned about a new weight loss app for children recently released by Weight Watchers. (The Checkup, “Helping Children Learn to Eat Well,” The New York Times, Aug. 26, 2019). Although the authors of the journal article question some of the science behind the app, their primary concerns are that the app is aimed at children without a way to guarantee parental involvement, and in their opinion the app also places too much emphasis on weight loss.

Their concerns go right to the heart of what troubles me about managing obesity in children. How should I talk to a child about her weight? What words can I choose without shaming? Maybe I shouldn’t be talking to her at all. When a child is 18 months old, we don’t talk to her about her growth chart. Not because she couldn’t understand, but because the solution rests not with her but with her parents.

At what age does a child’s obesity cease being simply a parenting problem and instead become something the child must solve? Does that point come when we have given up on the parents’ ability to create and maintain an environment that discourages obesity? Is that the point when we begin asking the child to unlearn a complex set of behaviors that have been enabled or at least tolerated and poorly modeled at home?

When we begin to talk to a child about his weight do we begin by telling him that he may not have been a contributor to the problem when it began but from now on he needs to be a major player in its management? Of course we don’t share that reality with an 8-year-old, but sometime during his struggle to manage his weight he will connect the dots.

If you are beginning to suspect that I have built my pediatric career around a scaffolding of parent blaming and shaming you are wrong. I know that there are children who have inherited a suite of genes that make them vulnerable to obesity. And I know that too many children grow up in environments in which their parents are powerless to control the family diet for economic reasons. But I am sure that like me you mutter to yourself and your colleagues about the number of patients you are seeing each day whose growth charts are a clear reflection of less than optimal parenting.

Does all of this mean we throw in the towel and stop trying to help overweight children after they turn 6 years old? Of course not. But, it does mean we must redouble our efforts to help parents manage their children’s diets and activity levels in those first critical preschool years.
 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

You are seeing a 9-year-old for her annual health maintenance visit. A quick look at her growth chart easily confirms your first impression that she is obese. How are you going to address the weight that you know, and she probably suspects, is going to make her vulnerable to a myriad of health problems as she gets older?

SolStock/iStock/Getty Image

If she has been your patient since she was in preschool, this is certainly not the first time that her growth chart has been concerning. When did you first start discussing her weight with her parents? What words did you use? What strategies have you suggested? What referrals have you made? Maybe you have already given up and decided to not even “go there” at this visit because your experience with overweight patients has been so disappointing.

In her op ed in the New York Times, Dr. Perri Klass reconsiders these kinds of questions as she reviews an article in the journal Childhood Obesity (“Let’s Not Just Dismiss the Weight Watchers Kurbo App,” by Michelle I. Cardel, PhD, MS, RD, and Elsie M. Taveras, MD, MPH, August 2019) written by a nutrition scientist and a pediatrician who are concerned about a new weight loss app for children recently released by Weight Watchers. (The Checkup, “Helping Children Learn to Eat Well,” The New York Times, Aug. 26, 2019). Although the authors of the journal article question some of the science behind the app, their primary concerns are that the app is aimed at children without a way to guarantee parental involvement, and in their opinion the app also places too much emphasis on weight loss.

Their concerns go right to the heart of what troubles me about managing obesity in children. How should I talk to a child about her weight? What words can I choose without shaming? Maybe I shouldn’t be talking to her at all. When a child is 18 months old, we don’t talk to her about her growth chart. Not because she couldn’t understand, but because the solution rests not with her but with her parents.

At what age does a child’s obesity cease being simply a parenting problem and instead become something the child must solve? Does that point come when we have given up on the parents’ ability to create and maintain an environment that discourages obesity? Is that the point when we begin asking the child to unlearn a complex set of behaviors that have been enabled or at least tolerated and poorly modeled at home?

When we begin to talk to a child about his weight do we begin by telling him that he may not have been a contributor to the problem when it began but from now on he needs to be a major player in its management? Of course we don’t share that reality with an 8-year-old, but sometime during his struggle to manage his weight he will connect the dots.

If you are beginning to suspect that I have built my pediatric career around a scaffolding of parent blaming and shaming you are wrong. I know that there are children who have inherited a suite of genes that make them vulnerable to obesity. And I know that too many children grow up in environments in which their parents are powerless to control the family diet for economic reasons. But I am sure that like me you mutter to yourself and your colleagues about the number of patients you are seeing each day whose growth charts are a clear reflection of less than optimal parenting.

Does all of this mean we throw in the towel and stop trying to help overweight children after they turn 6 years old? Of course not. But, it does mean we must redouble our efforts to help parents manage their children’s diets and activity levels in those first critical preschool years.
 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Email him at [email protected].

Vaccines are marvelous, and there are many well documented success stories, including rotavirus (RV) vaccines, where a live vaccine is administered to the gastrointestinal mucosa via oral drops. Antigens presented at the mucosal/epithelial surface not only induce systemic serum IgG – as do injectable vaccines – but also induce secretory IgA (sIgA), which is most helpful in diseases that directly affect the mucosa.

Mucosal vs. systemic immunity

Antibody being present on mucosal surfaces (point of initial pathogen contact) has a chance to neutralize the pathogen before it gains a foothold. Pathogen-specific mucosal lymphoid elements (e.g. in Peyer’s patches in the gut) also appear critical for optimal protection.¹ The presence of both mucosal immune elements means that infection is severely limited or at times entirely prevented. So virus entering the GI tract causes minimal to no gut lining injury. Hence, there is no or mostly reduced vomiting/diarrhea. A downside of mucosally-administered live vaccines is that preexisting antibody to the vaccine antigens can reduce or block vaccine virus replication in the vaccinee, blunting or preventing protection. Note: Preexisting antibody also affects injectable live vaccines, such as the measles vaccine, similarly.

Classic injectable live or nonlive vaccines provide their most potent protection via systemic cellular responses antibody and/or antibodies in serum and extracellular fluid (ECF) where IgG and IgM are in highest concentrations. So even successful injectable vaccines still allow mucosal infection to start but then intercept further spread and prevent most of the downstream damage (think pertussis) or neutralize an infection-generated toxin (pertussis or tetanus). It usually is only after infection-induced damage occurs that systemic IgG and IgM gain better access to respiratory epithelial surfaces, but still only at a fraction of circulating concentrations. Indeed, pertussis vaccine–induced systemic immunity allows the pathogen to attack and replicate in/on host surface cells, causing toxin release and variable amounts of local mucosal injury/inflammation before vaccine-induced systemic immunity gains adequate access to the pathogen and/or to its toxin which may enter systemic circulation.
 

Live attenuated influenza vaccine (LAIV) induces mucosal immunity

Another “standard” vaccine that induces mucosal immunity – LAIV – was developed to improve on protection afforded by injectable influenza vaccines (IIVs), but LAIV has had hiccups in the United States. One example is several years of negligible protection against H1N1 disease. As long as LAIV’s vaccine strain had reasonably matched the circulating strains, LAIV worked at least as well as injectable influenza vaccine, and even offered some cross-protection against mildly mismatched strains. But after a number of years of LAIV use, vaccine effectiveness in the United States vs. H1N1 strains appeared to fade due to previously undetected but significant changes in the circulating H1N1 strain. The lesson is that mucosal immunity’s advantages are lost if too much change occurs in the pathogen target for sIgA and mucosally-associated lymphoid tissue cells (MALT)).

Other vaccines likely need to induce mucosal immunity

Protection at the mucosal level will likely be needed for success against norovirus, parainfluenza, respiratory syncytial virus (RSV), Neisseria gonorrhea, and chlamydia. Another helpful aspect of mucosal immunity is that immune cells and sIgA not only reside on the mucosa where the antigen was originally presented, but there is also a reasonable chance that these components will traffic to other mucosal surfaces.²

MDedge News

So intranasal vaccine could be expected to protect distant mucosal surfaces (urogenital, GI, and respiratory), leading to vaccine-induced systemic antibody plus mucosal immunity (sIGA and MALT responses) at each site.

Let’s look at a novel “two-site” chlamydia vaccine

Recently a phase 1 chlamydia vaccine that used a novel two-pronged administration site/schedule was successful at inducing both mucosal and systemic immunity in a proof-of-concept study – achieving the best of both worlds.³ This may be a template for vaccines in years to come. British investigators studied 50 healthy women aged 19-45 years in a double-blind, parallel, randomized, placebo-controlled trial that used a recombinant chlamydia protein subunit antigen (CTH522). The vaccine schedule involved three injectable priming doses followed soon thereafter by two intranasal boosting doses. There were three groups:

1. CTH522 adjuvanted with CAF01 liposomes (CTH522:CAF01).

2. CTH522 adjuvanted with aluminum hydroxide (CTH522:AH).

3. Placebo (saline).

The intramuscular (IM) priming schedule was 0, 1, and 4 months. The intranasal vaccine booster doses or placebo were given at 4.5 and 5 months. No related serious adverse reactions occurred. For injectable dosing, the most frequent adverse event was mild local injection-site reactions in all subjects in both vaccine groups vs. in 60% of placebo recipients (P = .053). The adjuvants were the likely cause for local reactions. Intranasal doses had local reactions in 47% of both vaccine groups and 60% of placebo recipients; P = 1.000).

Both vaccines produced systemic IgG seroconversion (including neutralizing antibody) plus small amounts of IgG in the nasal cavity and genital tract in all vaccine recipients; no placebo recipient seroconverted. Interestingly, liposomally-adjuvanted vaccine produced a more rapid systemic IgG response and higher serum titers than the alum-adjuvanted vaccine. Likewise, the IM liposomal vaccine also induced higher but still small mucosal IgG antibody responses (P = .0091). Intranasal IM-induced IgG titers were not boosted by later intranasal vaccine dosing.

Subjects getting liposomal vaccine (but not alum vaccine or placebo) boosters had detectable sIgA titers in both nasal and genital tract secretions. Liposomal vaccine recipients also had fivefold to sixfold higher median titers than alum vaccine recipients after the priming dose, and these higher titers persisted to the end of the study. All liposomal vaccine recipients developed antichlamydial cell-mediated responses vs. 57% alum-adjuvanted vaccine recipients. (P = .01). So both use of two-site dosing and the liposomal adjuvant appeared critical to better responses.

In summary

While this candidate vaccine has hurdles to overcome before coming into routine use, the proof-of-principle that a combination injectable-intranasal vaccine schedule can induce robust systemic and mucosal immunity when given with an appropriate adjuvant is very promising. One day we may be able to successfully immunize against more troublesome mucosal pathogens. Adding more vaccines to the schedule then becomes an issue, but that is one of those “good” problems we can deal with later.

Dr. Harrison is professor of pediatrics and pediatric infectious diseases at Children’s Mercy Hospital-Kansas City, Mo. Children’s Mercy Hospital receives grant funding to study two candidate RSV vaccines, receives funding from GlaxoSmithKline for studies on pneumococcal and rotavirus vaccines, and from Pfizer for a study on pneumococcal vaccine on which Dr. Harrison is a sub-investigator. The hospital also receives Centers for Disease Control and Prevention funding under the New Vaccine Surveillance Network for multicenter surveillance of acute respiratory infections, including influenza, RSV, and parainfluenza virus, and also for rotavirus. Email Dr. Harrison at [email protected].

References

1. PLOS Biology. 2012 Sep 1. doi: 10.1371/journal.pbio.1001397.

2. Mucosal Immunity in the Human Female Reproductive Tract in “Mucosal Immunology,” 4th ed., Volume 2 (Cambridge, MA: Academic Press, 2015, pp. 2097-124).

3. Lancet Infect Dis. 2019. doi: 10.1016/S1473-3099(19)30279-8.

Vaccines are marvelous, and there are many well documented success stories, including rotavirus (RV) vaccines, where a live vaccine is administered to the gastrointestinal mucosa via oral drops. Antigens presented at the mucosal/epithelial surface not only induce systemic serum IgG – as do injectable vaccines – but also induce secretory IgA (sIgA), which is most helpful in diseases that directly affect the mucosa.

Mucosal vs. systemic immunity

Antibody being present on mucosal surfaces (point of initial pathogen contact) has a chance to neutralize the pathogen before it gains a foothold. Pathogen-specific mucosal lymphoid elements (e.g. in Peyer’s patches in the gut) also appear critical for optimal protection.¹ The presence of both mucosal immune elements means that infection is severely limited or at times entirely prevented. So virus entering the GI tract causes minimal to no gut lining injury. Hence, there is no or mostly reduced vomiting/diarrhea. A downside of mucosally-administered live vaccines is that preexisting antibody to the vaccine antigens can reduce or block vaccine virus replication in the vaccinee, blunting or preventing protection. Note: Preexisting antibody also affects injectable live vaccines, such as the measles vaccine, similarly.

Classic injectable live or nonlive vaccines provide their most potent protection via systemic cellular responses antibody and/or antibodies in serum and extracellular fluid (ECF) where IgG and IgM are in highest concentrations. So even successful injectable vaccines still allow mucosal infection to start but then intercept further spread and prevent most of the downstream damage (think pertussis) or neutralize an infection-generated toxin (pertussis or tetanus). It usually is only after infection-induced damage occurs that systemic IgG and IgM gain better access to respiratory epithelial surfaces, but still only at a fraction of circulating concentrations. Indeed, pertussis vaccine–induced systemic immunity allows the pathogen to attack and replicate in/on host surface cells, causing toxin release and variable amounts of local mucosal injury/inflammation before vaccine-induced systemic immunity gains adequate access to the pathogen and/or to its toxin which may enter systemic circulation.
 

Live attenuated influenza vaccine (LAIV) induces mucosal immunity

Another “standard” vaccine that induces mucosal immunity – LAIV – was developed to improve on protection afforded by injectable influenza vaccines (IIVs), but LAIV has had hiccups in the United States. One example is several years of negligible protection against H1N1 disease. As long as LAIV’s vaccine strain had reasonably matched the circulating strains, LAIV worked at least as well as injectable influenza vaccine, and even offered some cross-protection against mildly mismatched strains. But after a number of years of LAIV use, vaccine effectiveness in the United States vs. H1N1 strains appeared to fade due to previously undetected but significant changes in the circulating H1N1 strain. The lesson is that mucosal immunity’s advantages are lost if too much change occurs in the pathogen target for sIgA and mucosally-associated lymphoid tissue cells (MALT)).

Other vaccines likely need to induce mucosal immunity

Protection at the mucosal level will likely be needed for success against norovirus, parainfluenza, respiratory syncytial virus (RSV), Neisseria gonorrhea, and chlamydia. Another helpful aspect of mucosal immunity is that immune cells and sIgA not only reside on the mucosa where the antigen was originally presented, but there is also a reasonable chance that these components will traffic to other mucosal surfaces.²

MDedge News

So intranasal vaccine could be expected to protect distant mucosal surfaces (urogenital, GI, and respiratory), leading to vaccine-induced systemic antibody plus mucosal immunity (sIGA and MALT responses) at each site.

Let’s look at a novel “two-site” chlamydia vaccine

Recently a phase 1 chlamydia vaccine that used a novel two-pronged administration site/schedule was successful at inducing both mucosal and systemic immunity in a proof-of-concept study – achieving the best of both worlds.³ This may be a template for vaccines in years to come. British investigators studied 50 healthy women aged 19-45 years in a double-blind, parallel, randomized, placebo-controlled trial that used a recombinant chlamydia protein subunit antigen (CTH522). The vaccine schedule involved three injectable priming doses followed soon thereafter by two intranasal boosting doses. There were three groups:

1. CTH522 adjuvanted with CAF01 liposomes (CTH522:CAF01).

2. CTH522 adjuvanted with aluminum hydroxide (CTH522:AH).

3. Placebo (saline).

The intramuscular (IM) priming schedule was 0, 1, and 4 months. The intranasal vaccine booster doses or placebo were given at 4.5 and 5 months. No related serious adverse reactions occurred. For injectable dosing, the most frequent adverse event was mild local injection-site reactions in all subjects in both vaccine groups vs. in 60% of placebo recipients (P = .053). The adjuvants were the likely cause for local reactions. Intranasal doses had local reactions in 47% of both vaccine groups and 60% of placebo recipients; P = 1.000).

Both vaccines produced systemic IgG seroconversion (including neutralizing antibody) plus small amounts of IgG in the nasal cavity and genital tract in all vaccine recipients; no placebo recipient seroconverted. Interestingly, liposomally-adjuvanted vaccine produced a more rapid systemic IgG response and higher serum titers than the alum-adjuvanted vaccine. Likewise, the IM liposomal vaccine also induced higher but still small mucosal IgG antibody responses (P = .0091). Intranasal IM-induced IgG titers were not boosted by later intranasal vaccine dosing.

Subjects getting liposomal vaccine (but not alum vaccine or placebo) boosters had detectable sIgA titers in both nasal and genital tract secretions. Liposomal vaccine recipients also had fivefold to sixfold higher median titers than alum vaccine recipients after the priming dose, and these higher titers persisted to the end of the study. All liposomal vaccine recipients developed antichlamydial cell-mediated responses vs. 57% alum-adjuvanted vaccine recipients. (P = .01). So both use of two-site dosing and the liposomal adjuvant appeared critical to better responses.

In summary

While this candidate vaccine has hurdles to overcome before coming into routine use, the proof-of-principle that a combination injectable-intranasal vaccine schedule can induce robust systemic and mucosal immunity when given with an appropriate adjuvant is very promising. One day we may be able to successfully immunize against more troublesome mucosal pathogens. Adding more vaccines to the schedule then becomes an issue, but that is one of those “good” problems we can deal with later.

Dr. Harrison is professor of pediatrics and pediatric infectious diseases at Children’s Mercy Hospital-Kansas City, Mo. Children’s Mercy Hospital receives grant funding to study two candidate RSV vaccines, receives funding from GlaxoSmithKline for studies on pneumococcal and rotavirus vaccines, and from Pfizer for a study on pneumococcal vaccine on which Dr. Harrison is a sub-investigator. The hospital also receives Centers for Disease Control and Prevention funding under the New Vaccine Surveillance Network for multicenter surveillance of acute respiratory infections, including influenza, RSV, and parainfluenza virus, and also for rotavirus. Email Dr. Harrison at [email protected].

References

1. PLOS Biology. 2012 Sep 1. doi: 10.1371/journal.pbio.1001397.

2. Mucosal Immunity in the Human Female Reproductive Tract in “Mucosal Immunology,” 4th ed., Volume 2 (Cambridge, MA: Academic Press, 2015, pp. 2097-124).

3. Lancet Infect Dis. 2019. doi: 10.1016/S1473-3099(19)30279-8.

Vaccines are marvelous, and there are many well documented success stories, including rotavirus (RV) vaccines, where a live vaccine is administered to the gastrointestinal mucosa via oral drops. Antigens presented at the mucosal/epithelial surface not only induce systemic serum IgG – as do injectable vaccines – but also induce secretory IgA (sIgA), which is most helpful in diseases that directly affect the mucosa.

Mucosal vs. systemic immunity

Antibody being present on mucosal surfaces (point of initial pathogen contact) has a chance to neutralize the pathogen before it gains a foothold. Pathogen-specific mucosal lymphoid elements (e.g. in Peyer’s patches in the gut) also appear critical for optimal protection.¹ The presence of both mucosal immune elements means that infection is severely limited or at times entirely prevented. So virus entering the GI tract causes minimal to no gut lining injury. Hence, there is no or mostly reduced vomiting/diarrhea. A downside of mucosally-administered live vaccines is that preexisting antibody to the vaccine antigens can reduce or block vaccine virus replication in the vaccinee, blunting or preventing protection. Note: Preexisting antibody also affects injectable live vaccines, such as the measles vaccine, similarly.

Classic injectable live or nonlive vaccines provide their most potent protection via systemic cellular responses antibody and/or antibodies in serum and extracellular fluid (ECF) where IgG and IgM are in highest concentrations. So even successful injectable vaccines still allow mucosal infection to start but then intercept further spread and prevent most of the downstream damage (think pertussis) or neutralize an infection-generated toxin (pertussis or tetanus). It usually is only after infection-induced damage occurs that systemic IgG and IgM gain better access to respiratory epithelial surfaces, but still only at a fraction of circulating concentrations. Indeed, pertussis vaccine–induced systemic immunity allows the pathogen to attack and replicate in/on host surface cells, causing toxin release and variable amounts of local mucosal injury/inflammation before vaccine-induced systemic immunity gains adequate access to the pathogen and/or to its toxin which may enter systemic circulation.
 

Live attenuated influenza vaccine (LAIV) induces mucosal immunity

Another “standard” vaccine that induces mucosal immunity – LAIV – was developed to improve on protection afforded by injectable influenza vaccines (IIVs), but LAIV has had hiccups in the United States. One example is several years of negligible protection against H1N1 disease. As long as LAIV’s vaccine strain had reasonably matched the circulating strains, LAIV worked at least as well as injectable influenza vaccine, and even offered some cross-protection against mildly mismatched strains. But after a number of years of LAIV use, vaccine effectiveness in the United States vs. H1N1 strains appeared to fade due to previously undetected but significant changes in the circulating H1N1 strain. The lesson is that mucosal immunity’s advantages are lost if too much change occurs in the pathogen target for sIgA and mucosally-associated lymphoid tissue cells (MALT)).

Other vaccines likely need to induce mucosal immunity

Protection at the mucosal level will likely be needed for success against norovirus, parainfluenza, respiratory syncytial virus (RSV), Neisseria gonorrhea, and chlamydia. Another helpful aspect of mucosal immunity is that immune cells and sIgA not only reside on the mucosa where the antigen was originally presented, but there is also a reasonable chance that these components will traffic to other mucosal surfaces.²

MDedge News

So intranasal vaccine could be expected to protect distant mucosal surfaces (urogenital, GI, and respiratory), leading to vaccine-induced systemic antibody plus mucosal immunity (sIGA and MALT responses) at each site.

Let’s look at a novel “two-site” chlamydia vaccine

Recently a phase 1 chlamydia vaccine that used a novel two-pronged administration site/schedule was successful at inducing both mucosal and systemic immunity in a proof-of-concept study – achieving the best of both worlds.³ This may be a template for vaccines in years to come. British investigators studied 50 healthy women aged 19-45 years in a double-blind, parallel, randomized, placebo-controlled trial that used a recombinant chlamydia protein subunit antigen (CTH522). The vaccine schedule involved three injectable priming doses followed soon thereafter by two intranasal boosting doses. There were three groups:

1. CTH522 adjuvanted with CAF01 liposomes (CTH522:CAF01).

2. CTH522 adjuvanted with aluminum hydroxide (CTH522:AH).

3. Placebo (saline).

The intramuscular (IM) priming schedule was 0, 1, and 4 months. The intranasal vaccine booster doses or placebo were given at 4.5 and 5 months. No related serious adverse reactions occurred. For injectable dosing, the most frequent adverse event was mild local injection-site reactions in all subjects in both vaccine groups vs. in 60% of placebo recipients (P = .053). The adjuvants were the likely cause for local reactions. Intranasal doses had local reactions in 47% of both vaccine groups and 60% of placebo recipients; P = 1.000).

Both vaccines produced systemic IgG seroconversion (including neutralizing antibody) plus small amounts of IgG in the nasal cavity and genital tract in all vaccine recipients; no placebo recipient seroconverted. Interestingly, liposomally-adjuvanted vaccine produced a more rapid systemic IgG response and higher serum titers than the alum-adjuvanted vaccine. Likewise, the IM liposomal vaccine also induced higher but still small mucosal IgG antibody responses (P = .0091). Intranasal IM-induced IgG titers were not boosted by later intranasal vaccine dosing.

Subjects getting liposomal vaccine (but not alum vaccine or placebo) boosters had detectable sIgA titers in both nasal and genital tract secretions. Liposomal vaccine recipients also had fivefold to sixfold higher median titers than alum vaccine recipients after the priming dose, and these higher titers persisted to the end of the study. All liposomal vaccine recipients developed antichlamydial cell-mediated responses vs. 57% alum-adjuvanted vaccine recipients. (P = .01). So both use of two-site dosing and the liposomal adjuvant appeared critical to better responses.

In summary

While this candidate vaccine has hurdles to overcome before coming into routine use, the proof-of-principle that a combination injectable-intranasal vaccine schedule can induce robust systemic and mucosal immunity when given with an appropriate adjuvant is very promising. One day we may be able to successfully immunize against more troublesome mucosal pathogens. Adding more vaccines to the schedule then becomes an issue, but that is one of those “good” problems we can deal with later.

Dr. Harrison is professor of pediatrics and pediatric infectious diseases at Children’s Mercy Hospital-Kansas City, Mo. Children’s Mercy Hospital receives grant funding to study two candidate RSV vaccines, receives funding from GlaxoSmithKline for studies on pneumococcal and rotavirus vaccines, and from Pfizer for a study on pneumococcal vaccine on which Dr. Harrison is a sub-investigator. The hospital also receives Centers for Disease Control and Prevention funding under the New Vaccine Surveillance Network for multicenter surveillance of acute respiratory infections, including influenza, RSV, and parainfluenza virus, and also for rotavirus. Email Dr. Harrison at [email protected].

References

1. PLOS Biology. 2012 Sep 1. doi: 10.1371/journal.pbio.1001397.

2. Mucosal Immunity in the Human Female Reproductive Tract in “Mucosal Immunology,” 4th ed., Volume 2 (Cambridge, MA: Academic Press, 2015, pp. 2097-124).

3. Lancet Infect Dis. 2019. doi: 10.1016/S1473-3099(19)30279-8.

Early results indicate experimental gene therapies could illicit a cure for sickle cell disease (SCD), but many barriers to access remain, namely cost, experts reported during a recent webinar sponsored by the National Heart, Lung, and Blood Institute.

Pogonic/Getty Images

At present, allogeneic hematopoietic stem cell transplant remains the only curative therapy available for patients with SCD. Newer transplant techniques include the use of mobilized blood stem cells, where stem cells are collected from the circulation using blood cell growth factors, explained Mark Walters, MD, of UCSF Benioff Children’s Hospital Oakland in California.

The most promising experimental gene therapies currently undergoing clinical development are gene-addition and gene-editing therapies, he said. Another technique, in vivo gene editing to correct the sickle mutation, is also being investigated, but has not yet reached clinical development.
 

Gene-addition therapy

Gene-addition therapy is a technique where a fetal hemoglobin (HbF) or anti-sickling beta-hemoglobin gene is inserted into a hematopoietic stem cell to illicit a curative effect. In this technique, the corrective gene is harvested from a patient’s own blood stem cells.

In patients with SCD, when HbF levels are elevated, the likelihood of sickling is reduced, resulting in a milder form of disease. As a result, raising HbF levels is a therapeutic target that forms the basis of several ongoing clinical studies.

The technique involves packaging an HbF rescue gene into a viral vector and coincubating the vector with a patient’s own blood stem cells. Subsequently, the corrected stem cells are injected back into the patient to produce higher levels of HbF.

The ongoing phase 1/2 HGB-206 clinical study is evaluating this technique in patients aged 12-50 years with severe SCD in multiple centers throughout Europe and the United States.

In those treated thus far, initial results appear promising, Dr. Walters reported, with one patient experiencing a rise in Hb levels from 10.7 g/dL at 3 months to 15.0 g/dL at 15 months follow-up.

Dr. Walters also reported that some of these patients no longer exhibit any signs or symptoms of SCD, such as anemia or painful adverse events. While these initial findings are compelling, whether these benefits will be maintained is still unknown.

“While it’s too early to call this a cure, if [these results] could be extended for 5, 10, or 15 years, I think everyone would agree that this would be a cure,” he said.

This technique could be universally available, he said, since a patient’s own blood stem cells are used. Other complications, such as graft-versus-host disease (GVHD) or immune-related reactions, are negated with this form of therapy, he said.

Recent evidence has demonstrated that only about 20% of donor stem cells need to be corrected to illicit a very strong effect. This principle is now being applied in gene-editing techniques, as correcting every gene in every stem cell would be very challenging, Dr. Walters explained.
 

Gene editing

Another technique being investigated in SCD is gene editing, in which the fetal hemoglobin gene is “reawakened,” or other techniques are used to correct the sickle gene directly, such as CRISPR-Cas9 technology, Dr. Walters said.

In this technique, the Cas9 protein makes a cut and repairs an individual’s genomic DNA by inserting a strand of corrected donor DNA. The novel technology would allow for targeted genome editing that is specific to the SCD patient.

Currently, this experimental therapy is being investigated in preclinical studies. Dr. Walters said that he and his colleagues hope to begin enrolling patients in clinical trials within the next 1-2 years.

But while some gene therapies have been approved in other disorders, such as spinal muscle atrophy, a limiting factor to widespread availability is cost. Despite promising initial results in SCD, the affordability of future gene therapies will be a key factor to universal access, Dr. Walters said.
 

The Cure Sickle Cell Initiative

Traci Mondoro, PhD, chief of the Translational Blood Science and Resources Branch at NHLBI, explained that the NHLBI has funded a large proportion of the research that has formed the basis of several genetically based clinical studies.

One of the primary goals of the Cure Sickle Cell Initiative is to bridge the gap between new research and the SCD community. Their aim is to improve access for patients to participate in genetically based studies to advance cures.

The comprehensive approach is intended to fill in existing gaps by funding breakthrough research in both academic and private settings.

By establishing partnerships with key stakeholders, institutions, and patient groups, Dr. Mondoro said they hope to increase patient participation in clinical trials involving curative therapies. In the future, they also intend to establish a large body of evidence to provide adequate safety data to study these therapies in pediatric populations.

Dr. Walters and Dr. Mondoro did not provide information on financial disclosures.

Early results indicate experimental gene therapies could illicit a cure for sickle cell disease (SCD), but many barriers to access remain, namely cost, experts reported during a recent webinar sponsored by the National Heart, Lung, and Blood Institute.

Pogonic/Getty Images

At present, allogeneic hematopoietic stem cell transplant remains the only curative therapy available for patients with SCD. Newer transplant techniques include the use of mobilized blood stem cells, where stem cells are collected from the circulation using blood cell growth factors, explained Mark Walters, MD, of UCSF Benioff Children’s Hospital Oakland in California.

The most promising experimental gene therapies currently undergoing clinical development are gene-addition and gene-editing therapies, he said. Another technique, in vivo gene editing to correct the sickle mutation, is also being investigated, but has not yet reached clinical development.
 

Gene-addition therapy

Gene-addition therapy is a technique where a fetal hemoglobin (HbF) or anti-sickling beta-hemoglobin gene is inserted into a hematopoietic stem cell to illicit a curative effect. In this technique, the corrective gene is harvested from a patient’s own blood stem cells.

In patients with SCD, when HbF levels are elevated, the likelihood of sickling is reduced, resulting in a milder form of disease. As a result, raising HbF levels is a therapeutic target that forms the basis of several ongoing clinical studies.

The technique involves packaging an HbF rescue gene into a viral vector and coincubating the vector with a patient’s own blood stem cells. Subsequently, the corrected stem cells are injected back into the patient to produce higher levels of HbF.

The ongoing phase 1/2 HGB-206 clinical study is evaluating this technique in patients aged 12-50 years with severe SCD in multiple centers throughout Europe and the United States.

In those treated thus far, initial results appear promising, Dr. Walters reported, with one patient experiencing a rise in Hb levels from 10.7 g/dL at 3 months to 15.0 g/dL at 15 months follow-up.

Dr. Walters also reported that some of these patients no longer exhibit any signs or symptoms of SCD, such as anemia or painful adverse events. While these initial findings are compelling, whether these benefits will be maintained is still unknown.

“While it’s too early to call this a cure, if [these results] could be extended for 5, 10, or 15 years, I think everyone would agree that this would be a cure,” he said.

This technique could be universally available, he said, since a patient’s own blood stem cells are used. Other complications, such as graft-versus-host disease (GVHD) or immune-related reactions, are negated with this form of therapy, he said.

Recent evidence has demonstrated that only about 20% of donor stem cells need to be corrected to illicit a very strong effect. This principle is now being applied in gene-editing techniques, as correcting every gene in every stem cell would be very challenging, Dr. Walters explained.
 

Gene editing

Another technique being investigated in SCD is gene editing, in which the fetal hemoglobin gene is “reawakened,” or other techniques are used to correct the sickle gene directly, such as CRISPR-Cas9 technology, Dr. Walters said.

In this technique, the Cas9 protein makes a cut and repairs an individual’s genomic DNA by inserting a strand of corrected donor DNA. The novel technology would allow for targeted genome editing that is specific to the SCD patient.

Currently, this experimental therapy is being investigated in preclinical studies. Dr. Walters said that he and his colleagues hope to begin enrolling patients in clinical trials within the next 1-2 years.

But while some gene therapies have been approved in other disorders, such as spinal muscle atrophy, a limiting factor to widespread availability is cost. Despite promising initial results in SCD, the affordability of future gene therapies will be a key factor to universal access, Dr. Walters said.
 

The Cure Sickle Cell Initiative

Traci Mondoro, PhD, chief of the Translational Blood Science and Resources Branch at NHLBI, explained that the NHLBI has funded a large proportion of the research that has formed the basis of several genetically based clinical studies.

One of the primary goals of the Cure Sickle Cell Initiative is to bridge the gap between new research and the SCD community. Their aim is to improve access for patients to participate in genetically based studies to advance cures.

The comprehensive approach is intended to fill in existing gaps by funding breakthrough research in both academic and private settings.

By establishing partnerships with key stakeholders, institutions, and patient groups, Dr. Mondoro said they hope to increase patient participation in clinical trials involving curative therapies. In the future, they also intend to establish a large body of evidence to provide adequate safety data to study these therapies in pediatric populations.

Dr. Walters and Dr. Mondoro did not provide information on financial disclosures.

Early results indicate experimental gene therapies could illicit a cure for sickle cell disease (SCD), but many barriers to access remain, namely cost, experts reported during a recent webinar sponsored by the National Heart, Lung, and Blood Institute.

Pogonic/Getty Images

At present, allogeneic hematopoietic stem cell transplant remains the only curative therapy available for patients with SCD. Newer transplant techniques include the use of mobilized blood stem cells, where stem cells are collected from the circulation using blood cell growth factors, explained Mark Walters, MD, of UCSF Benioff Children’s Hospital Oakland in California.

The most promising experimental gene therapies currently undergoing clinical development are gene-addition and gene-editing therapies, he said. Another technique, in vivo gene editing to correct the sickle mutation, is also being investigated, but has not yet reached clinical development.
 

Gene-addition therapy

Gene-addition therapy is a technique where a fetal hemoglobin (HbF) or anti-sickling beta-hemoglobin gene is inserted into a hematopoietic stem cell to illicit a curative effect. In this technique, the corrective gene is harvested from a patient’s own blood stem cells.

In patients with SCD, when HbF levels are elevated, the likelihood of sickling is reduced, resulting in a milder form of disease. As a result, raising HbF levels is a therapeutic target that forms the basis of several ongoing clinical studies.

The technique involves packaging an HbF rescue gene into a viral vector and coincubating the vector with a patient’s own blood stem cells. Subsequently, the corrected stem cells are injected back into the patient to produce higher levels of HbF.

The ongoing phase 1/2 HGB-206 clinical study is evaluating this technique in patients aged 12-50 years with severe SCD in multiple centers throughout Europe and the United States.

In those treated thus far, initial results appear promising, Dr. Walters reported, with one patient experiencing a rise in Hb levels from 10.7 g/dL at 3 months to 15.0 g/dL at 15 months follow-up.

Dr. Walters also reported that some of these patients no longer exhibit any signs or symptoms of SCD, such as anemia or painful adverse events. While these initial findings are compelling, whether these benefits will be maintained is still unknown.

“While it’s too early to call this a cure, if [these results] could be extended for 5, 10, or 15 years, I think everyone would agree that this would be a cure,” he said.

This technique could be universally available, he said, since a patient’s own blood stem cells are used. Other complications, such as graft-versus-host disease (GVHD) or immune-related reactions, are negated with this form of therapy, he said.

Recent evidence has demonstrated that only about 20% of donor stem cells need to be corrected to illicit a very strong effect. This principle is now being applied in gene-editing techniques, as correcting every gene in every stem cell would be very challenging, Dr. Walters explained.
 

Gene editing

Another technique being investigated in SCD is gene editing, in which the fetal hemoglobin gene is “reawakened,” or other techniques are used to correct the sickle gene directly, such as CRISPR-Cas9 technology, Dr. Walters said.

In this technique, the Cas9 protein makes a cut and repairs an individual’s genomic DNA by inserting a strand of corrected donor DNA. The novel technology would allow for targeted genome editing that is specific to the SCD patient.

Currently, this experimental therapy is being investigated in preclinical studies. Dr. Walters said that he and his colleagues hope to begin enrolling patients in clinical trials within the next 1-2 years.

But while some gene therapies have been approved in other disorders, such as spinal muscle atrophy, a limiting factor to widespread availability is cost. Despite promising initial results in SCD, the affordability of future gene therapies will be a key factor to universal access, Dr. Walters said.
 

The Cure Sickle Cell Initiative

Traci Mondoro, PhD, chief of the Translational Blood Science and Resources Branch at NHLBI, explained that the NHLBI has funded a large proportion of the research that has formed the basis of several genetically based clinical studies.

One of the primary goals of the Cure Sickle Cell Initiative is to bridge the gap between new research and the SCD community. Their aim is to improve access for patients to participate in genetically based studies to advance cures.

The comprehensive approach is intended to fill in existing gaps by funding breakthrough research in both academic and private settings.

By establishing partnerships with key stakeholders, institutions, and patient groups, Dr. Mondoro said they hope to increase patient participation in clinical trials involving curative therapies. In the future, they also intend to establish a large body of evidence to provide adequate safety data to study these therapies in pediatric populations.

Dr. Walters and Dr. Mondoro did not provide information on financial disclosures.