Almost half of black children with poorly controlled asthma showed a superior response to increased inhaled glucocorticoids over long-acting beta-agonists, based on data from 280 children aged 5-11 years with at least one grandparent identified as black.

Previous studies have suggested that long-acting beta₂-agonists (LABAs) may be more effective for patients with poorly controlled asthma, but such step-up therapy has not been well studied in black patients, wrote Michael E. Wechsler, MD, of National Jewish Health, Denver, and colleagues.

In a study published in the New England Journal of Medicine, the researchers reported results of two parallel BARD (Best African American Response to Asthma Drugs) trials conducted at nine centers between January 2014 and March 2016 of individuals with poorly controlled asthma. One trial included 280 children aged 5-11 years (average age, 8.5 years); the second trial included adolescents aged 12 years and older and adults (average age, 37 years) who had family backgrounds that were similar to those of the children.

The researchers randomized the children to four groups to compare the following protocols: doubling the dose of a glucocorticoid (fluticasone propionate) to a dose of 100 mcg, twice daily (the double-fluticasone group); doubling the dose of fluticasone to 100 mcg and adding 50 mcg of the LABA salmeterol (the salmeterol/double-fluticasone group); quintupling the dose of fluticasone to 250 mcg (the quintuple-fluticasone group); or quintupling the dose of fluticasone to 250 mcg and adding 50 mcg of salmeterol (the salmeterol/quintuple-fluticasone group). The trial consisted of a four-way crossover design with each treatment period lasting 14 weeks.

The primary outcome was a composite measure including asthma exacerbations, asthma control days, and percentage of predicted forced expiratory volume in the first second at the end of each treatment.

Overall, a superior response occurred in 53% of the salmeterol/double-fluticasone group, 41% of the double-fluticasone group, 43% of the salmeterol/quintuple fluticasone group, and 47% of the quintuple-fluticasone group.

The superior response was 46% for both groups when the researchers compared a quintupled dose of fluticasone propionate (250 mcg) with a two step–up strategy of adding salmeterol at a dose of 50 mcg and increasing the dose of fluticasone to 100 mcg.

“In contrast to black adults and white persons of all ages, almost half the children who had at least one grandparent who identified as black and who had poorly controlled asthma had a superior response to an increased dose of an inhaled glucocorticoid over the addition of a LABA,” Dr. Wechsler and coauthors wrote. No more than 12% of the children in any treatment group did not have a superior response. No significant differences in reports of respiratory tract infections or pneumonia were seen between the groups. Children younger than 8 years showed a decrease in the ratio of urinary cortisol to creatinine with an increased dose of inhaled glucocorticoids.

In the adolescent and adult study using the same treatment protocols, 20%-25% of patients did not have a differential outcome between treatments. “In adolescents and adults, the addition of a LABA was more likely to produce superior responses than increasing the dose of an inhaled glucocorticoid,” Dr. Wechsler and coauthors wrote.

The study findings were limited by several factors, including the inability to assess long-term effects on growth and inability to detect biomarkers associated with responses to specific therapies, the researchers noted. However, the results suggest that black children with poorly controlled asthma can benefit from additional inhaled glucocorticoids, and larger studies are needed to identify the best treatment for this patient population.

The study was supported by the National Heart, Lung, and Blood Institute. Dr. Wechsler reported relationships with companies including AstraZeneca, Equillium, Genentech, GlaxoSmithKline, Mylan, Novartis, Regeneron, resTORbio, Sanofi, and others. Coauthors identified relationships with numerous pharmaceutical companies.

SOURCE: Wechsler ME et al. N Engl J Med. 2019 Sep 25. doi: 10.1056/NEJMoa1905560.

Almost half of black children with poorly controlled asthma showed a superior response to increased inhaled glucocorticoids over long-acting beta-agonists, based on data from 280 children aged 5-11 years with at least one grandparent identified as black.

Previous studies have suggested that long-acting beta₂-agonists (LABAs) may be more effective for patients with poorly controlled asthma, but such step-up therapy has not been well studied in black patients, wrote Michael E. Wechsler, MD, of National Jewish Health, Denver, and colleagues.

In a study published in the New England Journal of Medicine, the researchers reported results of two parallel BARD (Best African American Response to Asthma Drugs) trials conducted at nine centers between January 2014 and March 2016 of individuals with poorly controlled asthma. One trial included 280 children aged 5-11 years (average age, 8.5 years); the second trial included adolescents aged 12 years and older and adults (average age, 37 years) who had family backgrounds that were similar to those of the children.

The researchers randomized the children to four groups to compare the following protocols: doubling the dose of a glucocorticoid (fluticasone propionate) to a dose of 100 mcg, twice daily (the double-fluticasone group); doubling the dose of fluticasone to 100 mcg and adding 50 mcg of the LABA salmeterol (the salmeterol/double-fluticasone group); quintupling the dose of fluticasone to 250 mcg (the quintuple-fluticasone group); or quintupling the dose of fluticasone to 250 mcg and adding 50 mcg of salmeterol (the salmeterol/quintuple-fluticasone group). The trial consisted of a four-way crossover design with each treatment period lasting 14 weeks.

The primary outcome was a composite measure including asthma exacerbations, asthma control days, and percentage of predicted forced expiratory volume in the first second at the end of each treatment.

Overall, a superior response occurred in 53% of the salmeterol/double-fluticasone group, 41% of the double-fluticasone group, 43% of the salmeterol/quintuple fluticasone group, and 47% of the quintuple-fluticasone group.

The superior response was 46% for both groups when the researchers compared a quintupled dose of fluticasone propionate (250 mcg) with a two step–up strategy of adding salmeterol at a dose of 50 mcg and increasing the dose of fluticasone to 100 mcg.

“In contrast to black adults and white persons of all ages, almost half the children who had at least one grandparent who identified as black and who had poorly controlled asthma had a superior response to an increased dose of an inhaled glucocorticoid over the addition of a LABA,” Dr. Wechsler and coauthors wrote. No more than 12% of the children in any treatment group did not have a superior response. No significant differences in reports of respiratory tract infections or pneumonia were seen between the groups. Children younger than 8 years showed a decrease in the ratio of urinary cortisol to creatinine with an increased dose of inhaled glucocorticoids.

In the adolescent and adult study using the same treatment protocols, 20%-25% of patients did not have a differential outcome between treatments. “In adolescents and adults, the addition of a LABA was more likely to produce superior responses than increasing the dose of an inhaled glucocorticoid,” Dr. Wechsler and coauthors wrote.

The study findings were limited by several factors, including the inability to assess long-term effects on growth and inability to detect biomarkers associated with responses to specific therapies, the researchers noted. However, the results suggest that black children with poorly controlled asthma can benefit from additional inhaled glucocorticoids, and larger studies are needed to identify the best treatment for this patient population.

The study was supported by the National Heart, Lung, and Blood Institute. Dr. Wechsler reported relationships with companies including AstraZeneca, Equillium, Genentech, GlaxoSmithKline, Mylan, Novartis, Regeneron, resTORbio, Sanofi, and others. Coauthors identified relationships with numerous pharmaceutical companies.

SOURCE: Wechsler ME et al. N Engl J Med. 2019 Sep 25. doi: 10.1056/NEJMoa1905560.

Almost half of black children with poorly controlled asthma showed a superior response to increased inhaled glucocorticoids over long-acting beta-agonists, based on data from 280 children aged 5-11 years with at least one grandparent identified as black.

Previous studies have suggested that long-acting beta₂-agonists (LABAs) may be more effective for patients with poorly controlled asthma, but such step-up therapy has not been well studied in black patients, wrote Michael E. Wechsler, MD, of National Jewish Health, Denver, and colleagues.

In a study published in the New England Journal of Medicine, the researchers reported results of two parallel BARD (Best African American Response to Asthma Drugs) trials conducted at nine centers between January 2014 and March 2016 of individuals with poorly controlled asthma. One trial included 280 children aged 5-11 years (average age, 8.5 years); the second trial included adolescents aged 12 years and older and adults (average age, 37 years) who had family backgrounds that were similar to those of the children.

The researchers randomized the children to four groups to compare the following protocols: doubling the dose of a glucocorticoid (fluticasone propionate) to a dose of 100 mcg, twice daily (the double-fluticasone group); doubling the dose of fluticasone to 100 mcg and adding 50 mcg of the LABA salmeterol (the salmeterol/double-fluticasone group); quintupling the dose of fluticasone to 250 mcg (the quintuple-fluticasone group); or quintupling the dose of fluticasone to 250 mcg and adding 50 mcg of salmeterol (the salmeterol/quintuple-fluticasone group). The trial consisted of a four-way crossover design with each treatment period lasting 14 weeks.

The primary outcome was a composite measure including asthma exacerbations, asthma control days, and percentage of predicted forced expiratory volume in the first second at the end of each treatment.

Overall, a superior response occurred in 53% of the salmeterol/double-fluticasone group, 41% of the double-fluticasone group, 43% of the salmeterol/quintuple fluticasone group, and 47% of the quintuple-fluticasone group.

The superior response was 46% for both groups when the researchers compared a quintupled dose of fluticasone propionate (250 mcg) with a two step–up strategy of adding salmeterol at a dose of 50 mcg and increasing the dose of fluticasone to 100 mcg.

“In contrast to black adults and white persons of all ages, almost half the children who had at least one grandparent who identified as black and who had poorly controlled asthma had a superior response to an increased dose of an inhaled glucocorticoid over the addition of a LABA,” Dr. Wechsler and coauthors wrote. No more than 12% of the children in any treatment group did not have a superior response. No significant differences in reports of respiratory tract infections or pneumonia were seen between the groups. Children younger than 8 years showed a decrease in the ratio of urinary cortisol to creatinine with an increased dose of inhaled glucocorticoids.

In the adolescent and adult study using the same treatment protocols, 20%-25% of patients did not have a differential outcome between treatments. “In adolescents and adults, the addition of a LABA was more likely to produce superior responses than increasing the dose of an inhaled glucocorticoid,” Dr. Wechsler and coauthors wrote.

The study findings were limited by several factors, including the inability to assess long-term effects on growth and inability to detect biomarkers associated with responses to specific therapies, the researchers noted. However, the results suggest that black children with poorly controlled asthma can benefit from additional inhaled glucocorticoids, and larger studies are needed to identify the best treatment for this patient population.

The study was supported by the National Heart, Lung, and Blood Institute. Dr. Wechsler reported relationships with companies including AstraZeneca, Equillium, Genentech, GlaxoSmithKline, Mylan, Novartis, Regeneron, resTORbio, Sanofi, and others. Coauthors identified relationships with numerous pharmaceutical companies.

SOURCE: Wechsler ME et al. N Engl J Med. 2019 Sep 25. doi: 10.1056/NEJMoa1905560.

The AGA Education & Training Committee sponsors a session during Digestive Disease Week^® (DDW) entitled “Advancing Clinical Practice: GI Fellow-Directed Quality Improvement (QI) Projects.” Session participants are selected to give an oral or poster presentation of a quality improvement project they complete during fellowship. The QI project abstracts are peer reviewed and chosen by volunteers from the AGA Young Delegates. We asked several abstract reviewers from the 2019 session for advice on what makes an exceptional QI project and how to make an abstract stand out.

This session will be held again during DDW 2020. Interested participants should submit their abstract to the DDW descriptor GI Fellow-Directed QI Session via the DDW abstract submission site between Oct. 17 and Dec. 1, 2019.

What are the top 3 things that make an exceptional QI project?

Mohammad Bilal, MD, fellow, advanced endoscopy
Beth Israel Deaconess Medical Center Harvard Medical School in Boston

Quality improvement projects are essential to identify areas of improvement in a health care system/institution and to be able to improve them. The best kind of QI projects are those that lead to sustainable positive outcomes. This is not always easy and usually requires a multifocal intervention strategy. In my opinion, the top three things that make an exceptional QI project are:

1. A clear, focused, concise, realistic, and achievable “aim statement,” also known as a “SMART” (Specific, Measurable, Achievable, Relevant/Realistic, Time-Bound) aim statement.

2. A project that directly impacts patient-related outcomes.

3. A project that involves multiple members of the health care team in addition to physicians and patients, such as pharmacists, therapists, schedulers, or IT staff.

Chung Sang Tse, MD, gastroenterology fellow
Brown University Rhode Island Hospital in Providence, R.I.

Some of the most impressive QI abstracts that stood out to my fellow cojudges and I were those that employed novel yet widely applicable solutions to common problems, along with empiric data to assess their effects. For example, one of the most highly rated abstracts was one that used an electronic order set to standardize the acute care of inflammatory bowel disease flare in the emergency department and measured the impact on treatment outcomes. Another memorable abstract tested the use of meditation (via an instructional soundtrack) in the endoscopy suite to assess its effect on sedation use, procedural time, and patient comfort; although the results in the abstract did not reach statistical significance, the reviewers rated this abstract favorably for its attempt to improve the endoscopy experience in a low-cost and replicable manner. For this QI category, the reviewers weighed most heavily on a study’s impact, the rigor of methodology, and novelty of the problem/solution.

What advice would you give to prospective authors to make their abstracts stand out?

Michelle Hughes, MD, assistant professor of internal medicine
Section of digestive diseases, Yale School of Medicine in New Haven, Conn.

Turning your hard work from a QI project into a successful abstract can seem daunting. There are a few things to consider to help your findings reach your target audience. First, an abstract should be clear and concise. Limit background content and focus on highlighting your project’s methodology and results. Remember, readers look for a comprehensive overview so they can easily understand what you did and found without a lot of extra content obscuring the main points. Everything in your abstract should tie back to your stated aim. Does the background build a case for your project? Do the results and conclusion clearly answer the hypothesis and/or clinical question? If a reported result does not help answer your question, then it should probably be left out. Another tip is to always carefully follow the guidelines for abstract submission. Ensure you use correct formatting and follow all outlined rules. Ask a colleague to review and proofread your abstract draft prior to submission. Lastly, before you start writing, decide what take-away message or statement makes your project meaningful. Refer to this as you write and finalize your abstract so that you keep your message clear and remain focused. You did the hard work to execute your project – but without an effective abstract, the impact of your work may get lost.
 

Manol Jovani, MD, MPH, therapeutic endoscopy fellow
Division of gastroenterology and hepatology, Johns Hopkins Hospital in Baltimore

The abstract is the “elevator pitch” of your year(s)-long work, and as such it must be very carefully crafted. Dedicate specific time to the style and quality of your presentation long before the deadline. As for elevator pitches, first impressions are very important. For an abstract, it begins with the title. Select a declarative title that draws the interest of the reviewers/readers and tells them exactly what to expect, which also accurately represents the main findings. The introduction and aims should be very short and to the point. For example, it is counterproductive to start with “Ulcerative colitis (UC) and Crohn’s disease (CD) are the two major types of inflammatory bowel disease (IBD).” This does not add any information, states what is obvious to any GI reviewer, uses up 92 characters, and possibly gives a subconscious sense of unoriginality. Throughout the description of your methods/results, use clear language. A well-written abstract will explicitly and concisely state the populations included, statistical analyses performed, and specific outcomes. Make it easy for reviewers to follow along by presenting the results in a logical progression and by striving to use as few acronyms as possible. In addition, less is often more. The abstract should have only one main message, enunciated with the title, evident in the results, and interpreted with the conclusion. If you have additional space, use it to make compelling concluding remarks. As in an elevator pitch, the conclusion is as important as the introduction. Conclude by giving a clear message to the audience/reviewers on what the main findings are and suggest specific implications for future research. Do not end with “more studies are needed,” but rather suggest specifically how these results will influence our future understanding. Finally, know your audience and adapt your presentation to their needs, knowledge, and interests. This includes being mindful of the section to which you are submitting your abstract. An excellent abstract that could have been an oral presentation in one section may end up being a poster in another. Something important to remember about abstracts is that, while the content of the research is the most important part, the quality of its presentation serves to alert the reviewers and readers about it.
 

Ms. Mietzelfeld is a coordinator of data and training initiatives for the American Gastroenterological Association in Bethesda, Md.

The AGA Education & Training Committee sponsors a session during Digestive Disease Week^® (DDW) entitled “Advancing Clinical Practice: GI Fellow-Directed Quality Improvement (QI) Projects.” Session participants are selected to give an oral or poster presentation of a quality improvement project they complete during fellowship. The QI project abstracts are peer reviewed and chosen by volunteers from the AGA Young Delegates. We asked several abstract reviewers from the 2019 session for advice on what makes an exceptional QI project and how to make an abstract stand out.

This session will be held again during DDW 2020. Interested participants should submit their abstract to the DDW descriptor GI Fellow-Directed QI Session via the DDW abstract submission site between Oct. 17 and Dec. 1, 2019.

What are the top 3 things that make an exceptional QI project?

Mohammad Bilal, MD, fellow, advanced endoscopy
Beth Israel Deaconess Medical Center Harvard Medical School in Boston

Quality improvement projects are essential to identify areas of improvement in a health care system/institution and to be able to improve them. The best kind of QI projects are those that lead to sustainable positive outcomes. This is not always easy and usually requires a multifocal intervention strategy. In my opinion, the top three things that make an exceptional QI project are:

1. A clear, focused, concise, realistic, and achievable “aim statement,” also known as a “SMART” (Specific, Measurable, Achievable, Relevant/Realistic, Time-Bound) aim statement.

2. A project that directly impacts patient-related outcomes.

3. A project that involves multiple members of the health care team in addition to physicians and patients, such as pharmacists, therapists, schedulers, or IT staff.

Chung Sang Tse, MD, gastroenterology fellow
Brown University Rhode Island Hospital in Providence, R.I.

Some of the most impressive QI abstracts that stood out to my fellow cojudges and I were those that employed novel yet widely applicable solutions to common problems, along with empiric data to assess their effects. For example, one of the most highly rated abstracts was one that used an electronic order set to standardize the acute care of inflammatory bowel disease flare in the emergency department and measured the impact on treatment outcomes. Another memorable abstract tested the use of meditation (via an instructional soundtrack) in the endoscopy suite to assess its effect on sedation use, procedural time, and patient comfort; although the results in the abstract did not reach statistical significance, the reviewers rated this abstract favorably for its attempt to improve the endoscopy experience in a low-cost and replicable manner. For this QI category, the reviewers weighed most heavily on a study’s impact, the rigor of methodology, and novelty of the problem/solution.

What advice would you give to prospective authors to make their abstracts stand out?

Michelle Hughes, MD, assistant professor of internal medicine
Section of digestive diseases, Yale School of Medicine in New Haven, Conn.

Turning your hard work from a QI project into a successful abstract can seem daunting. There are a few things to consider to help your findings reach your target audience. First, an abstract should be clear and concise. Limit background content and focus on highlighting your project’s methodology and results. Remember, readers look for a comprehensive overview so they can easily understand what you did and found without a lot of extra content obscuring the main points. Everything in your abstract should tie back to your stated aim. Does the background build a case for your project? Do the results and conclusion clearly answer the hypothesis and/or clinical question? If a reported result does not help answer your question, then it should probably be left out. Another tip is to always carefully follow the guidelines for abstract submission. Ensure you use correct formatting and follow all outlined rules. Ask a colleague to review and proofread your abstract draft prior to submission. Lastly, before you start writing, decide what take-away message or statement makes your project meaningful. Refer to this as you write and finalize your abstract so that you keep your message clear and remain focused. You did the hard work to execute your project – but without an effective abstract, the impact of your work may get lost.
 

Manol Jovani, MD, MPH, therapeutic endoscopy fellow
Division of gastroenterology and hepatology, Johns Hopkins Hospital in Baltimore

The abstract is the “elevator pitch” of your year(s)-long work, and as such it must be very carefully crafted. Dedicate specific time to the style and quality of your presentation long before the deadline. As for elevator pitches, first impressions are very important. For an abstract, it begins with the title. Select a declarative title that draws the interest of the reviewers/readers and tells them exactly what to expect, which also accurately represents the main findings. The introduction and aims should be very short and to the point. For example, it is counterproductive to start with “Ulcerative colitis (UC) and Crohn’s disease (CD) are the two major types of inflammatory bowel disease (IBD).” This does not add any information, states what is obvious to any GI reviewer, uses up 92 characters, and possibly gives a subconscious sense of unoriginality. Throughout the description of your methods/results, use clear language. A well-written abstract will explicitly and concisely state the populations included, statistical analyses performed, and specific outcomes. Make it easy for reviewers to follow along by presenting the results in a logical progression and by striving to use as few acronyms as possible. In addition, less is often more. The abstract should have only one main message, enunciated with the title, evident in the results, and interpreted with the conclusion. If you have additional space, use it to make compelling concluding remarks. As in an elevator pitch, the conclusion is as important as the introduction. Conclude by giving a clear message to the audience/reviewers on what the main findings are and suggest specific implications for future research. Do not end with “more studies are needed,” but rather suggest specifically how these results will influence our future understanding. Finally, know your audience and adapt your presentation to their needs, knowledge, and interests. This includes being mindful of the section to which you are submitting your abstract. An excellent abstract that could have been an oral presentation in one section may end up being a poster in another. Something important to remember about abstracts is that, while the content of the research is the most important part, the quality of its presentation serves to alert the reviewers and readers about it.
 

Ms. Mietzelfeld is a coordinator of data and training initiatives for the American Gastroenterological Association in Bethesda, Md.

The AGA Education & Training Committee sponsors a session during Digestive Disease Week^® (DDW) entitled “Advancing Clinical Practice: GI Fellow-Directed Quality Improvement (QI) Projects.” Session participants are selected to give an oral or poster presentation of a quality improvement project they complete during fellowship. The QI project abstracts are peer reviewed and chosen by volunteers from the AGA Young Delegates. We asked several abstract reviewers from the 2019 session for advice on what makes an exceptional QI project and how to make an abstract stand out.

This session will be held again during DDW 2020. Interested participants should submit their abstract to the DDW descriptor GI Fellow-Directed QI Session via the DDW abstract submission site between Oct. 17 and Dec. 1, 2019.

What are the top 3 things that make an exceptional QI project?

Mohammad Bilal, MD, fellow, advanced endoscopy
Beth Israel Deaconess Medical Center Harvard Medical School in Boston

Quality improvement projects are essential to identify areas of improvement in a health care system/institution and to be able to improve them. The best kind of QI projects are those that lead to sustainable positive outcomes. This is not always easy and usually requires a multifocal intervention strategy. In my opinion, the top three things that make an exceptional QI project are:

1. A clear, focused, concise, realistic, and achievable “aim statement,” also known as a “SMART” (Specific, Measurable, Achievable, Relevant/Realistic, Time-Bound) aim statement.

2. A project that directly impacts patient-related outcomes.

3. A project that involves multiple members of the health care team in addition to physicians and patients, such as pharmacists, therapists, schedulers, or IT staff.

Chung Sang Tse, MD, gastroenterology fellow
Brown University Rhode Island Hospital in Providence, R.I.

Some of the most impressive QI abstracts that stood out to my fellow cojudges and I were those that employed novel yet widely applicable solutions to common problems, along with empiric data to assess their effects. For example, one of the most highly rated abstracts was one that used an electronic order set to standardize the acute care of inflammatory bowel disease flare in the emergency department and measured the impact on treatment outcomes. Another memorable abstract tested the use of meditation (via an instructional soundtrack) in the endoscopy suite to assess its effect on sedation use, procedural time, and patient comfort; although the results in the abstract did not reach statistical significance, the reviewers rated this abstract favorably for its attempt to improve the endoscopy experience in a low-cost and replicable manner. For this QI category, the reviewers weighed most heavily on a study’s impact, the rigor of methodology, and novelty of the problem/solution.

What advice would you give to prospective authors to make their abstracts stand out?

Michelle Hughes, MD, assistant professor of internal medicine
Section of digestive diseases, Yale School of Medicine in New Haven, Conn.

Turning your hard work from a QI project into a successful abstract can seem daunting. There are a few things to consider to help your findings reach your target audience. First, an abstract should be clear and concise. Limit background content and focus on highlighting your project’s methodology and results. Remember, readers look for a comprehensive overview so they can easily understand what you did and found without a lot of extra content obscuring the main points. Everything in your abstract should tie back to your stated aim. Does the background build a case for your project? Do the results and conclusion clearly answer the hypothesis and/or clinical question? If a reported result does not help answer your question, then it should probably be left out. Another tip is to always carefully follow the guidelines for abstract submission. Ensure you use correct formatting and follow all outlined rules. Ask a colleague to review and proofread your abstract draft prior to submission. Lastly, before you start writing, decide what take-away message or statement makes your project meaningful. Refer to this as you write and finalize your abstract so that you keep your message clear and remain focused. You did the hard work to execute your project – but without an effective abstract, the impact of your work may get lost.
 

Manol Jovani, MD, MPH, therapeutic endoscopy fellow
Division of gastroenterology and hepatology, Johns Hopkins Hospital in Baltimore

The abstract is the “elevator pitch” of your year(s)-long work, and as such it must be very carefully crafted. Dedicate specific time to the style and quality of your presentation long before the deadline. As for elevator pitches, first impressions are very important. For an abstract, it begins with the title. Select a declarative title that draws the interest of the reviewers/readers and tells them exactly what to expect, which also accurately represents the main findings. The introduction and aims should be very short and to the point. For example, it is counterproductive to start with “Ulcerative colitis (UC) and Crohn’s disease (CD) are the two major types of inflammatory bowel disease (IBD).” This does not add any information, states what is obvious to any GI reviewer, uses up 92 characters, and possibly gives a subconscious sense of unoriginality. Throughout the description of your methods/results, use clear language. A well-written abstract will explicitly and concisely state the populations included, statistical analyses performed, and specific outcomes. Make it easy for reviewers to follow along by presenting the results in a logical progression and by striving to use as few acronyms as possible. In addition, less is often more. The abstract should have only one main message, enunciated with the title, evident in the results, and interpreted with the conclusion. If you have additional space, use it to make compelling concluding remarks. As in an elevator pitch, the conclusion is as important as the introduction. Conclude by giving a clear message to the audience/reviewers on what the main findings are and suggest specific implications for future research. Do not end with “more studies are needed,” but rather suggest specifically how these results will influence our future understanding. Finally, know your audience and adapt your presentation to their needs, knowledge, and interests. This includes being mindful of the section to which you are submitting your abstract. An excellent abstract that could have been an oral presentation in one section may end up being a poster in another. Something important to remember about abstracts is that, while the content of the research is the most important part, the quality of its presentation serves to alert the reviewers and readers about it.
 

Ms. Mietzelfeld is a coordinator of data and training initiatives for the American Gastroenterological Association in Bethesda, Md.

Noninvasive vagus nerve stimulation may reduce the intensity of vertigo and headache in patients with acute vestibular migraine attacks, according to a small, open-label study published online Sept. 25 in Neurology.

The retrospective, single-center study included data from 18 patients who had vestibular migraine and received acute treatment with a handheld stimulation device. The device used in the study, GammaCore, is approved for the treatment of migraine. There are no approved treatments for vestibular migraine.

“There’s a huge need for effective treatments for vestibular migraine attacks,” first author Shin C. Beh, MD, said in a news release. “People with vestibular migraine do not always have headaches, and when they do, they are often less severe than in typical migraine, so the pain-relieving drugs used for typical migraine often are not effective. People can take drugs that suppress the vertigo or the nausea, but those drugs cause drowsiness and make it hard for people to go about their usual activities.” Dr. Beh is an assistant professor of neurology and neurotherapeutics at University of Texas Southwestern Medical Center in Dallas.

The patients had an average age of about 46 years, and 16 were women. A total of 14 patients received treatment during an acute vestibular migraine attack, and 4 received treatment while they had bothersome interictal dizziness consistent with persistent perceptual postural dizziness.

After stimulation, vertigo improved in 13 of the 14 people with vestibular migraine attacks. In two of these patients, vertigo resolved completely, and five had at least a 50% improvement in vertigo symptoms. On a 0-10 scale, patients’ mean vertigo rating decreased from 5.2 before stimulation to 3.1 after stimulation.

Of five patients with vestibular migraine attacks who had headache, all experienced a reduction in headache pain. Mean headache severity decreased from 6 before stimulation to 2.4 after stimulation.

None of the four patients with interictal dizziness experienced a reduction in dizziness after stimulation.

Patients place the device against the neck to receive electrical stimulation, and patients received stimulation for 2 minutes on each side of the neck.

Participants reported a mild pulling sensation of the neck muscles during the stimulation but did not report pain or other side effects.

“Vestibular migraine is the most common neurologic cause of vertigo and can greatly interfere with a person’s daily life,” Dr. Beh said. “If these results can be confirmed with larger studies, not only could there finally be a treatment for vestibular migraine, such a treatment would also be easy to use.”

A randomized, double-blind, sham-controlled study is needed to further assess the use of noninvasive vagus nerve stimulation in treating vestibular migraine, the authors said.

The study had no targeted funding, and Dr. Beh had no relevant disclosures. Coauthor Deborah I. Friedman, MD, professor of neurology and neurotherapeutics and ophthalmology at UT Southwestern Medical Center, disclosed serving on advisory boards or speaking for pharmaceutical and medical device companies. Dr. Friedman is on the editorial advisory board of Neurology Reviews.

[email protected]

SOURCE: Beh SC et al. Neurology. 2019 Sep 25. doi: 10.1212/WNL.0000000000008388.

Noninvasive vagus nerve stimulation may reduce the intensity of vertigo and headache in patients with acute vestibular migraine attacks, according to a small, open-label study published online Sept. 25 in Neurology.

The retrospective, single-center study included data from 18 patients who had vestibular migraine and received acute treatment with a handheld stimulation device. The device used in the study, GammaCore, is approved for the treatment of migraine. There are no approved treatments for vestibular migraine.

“There’s a huge need for effective treatments for vestibular migraine attacks,” first author Shin C. Beh, MD, said in a news release. “People with vestibular migraine do not always have headaches, and when they do, they are often less severe than in typical migraine, so the pain-relieving drugs used for typical migraine often are not effective. People can take drugs that suppress the vertigo or the nausea, but those drugs cause drowsiness and make it hard for people to go about their usual activities.” Dr. Beh is an assistant professor of neurology and neurotherapeutics at University of Texas Southwestern Medical Center in Dallas.

The patients had an average age of about 46 years, and 16 were women. A total of 14 patients received treatment during an acute vestibular migraine attack, and 4 received treatment while they had bothersome interictal dizziness consistent with persistent perceptual postural dizziness.

After stimulation, vertigo improved in 13 of the 14 people with vestibular migraine attacks. In two of these patients, vertigo resolved completely, and five had at least a 50% improvement in vertigo symptoms. On a 0-10 scale, patients’ mean vertigo rating decreased from 5.2 before stimulation to 3.1 after stimulation.

Of five patients with vestibular migraine attacks who had headache, all experienced a reduction in headache pain. Mean headache severity decreased from 6 before stimulation to 2.4 after stimulation.

None of the four patients with interictal dizziness experienced a reduction in dizziness after stimulation.

Patients place the device against the neck to receive electrical stimulation, and patients received stimulation for 2 minutes on each side of the neck.

Participants reported a mild pulling sensation of the neck muscles during the stimulation but did not report pain or other side effects.

“Vestibular migraine is the most common neurologic cause of vertigo and can greatly interfere with a person’s daily life,” Dr. Beh said. “If these results can be confirmed with larger studies, not only could there finally be a treatment for vestibular migraine, such a treatment would also be easy to use.”

A randomized, double-blind, sham-controlled study is needed to further assess the use of noninvasive vagus nerve stimulation in treating vestibular migraine, the authors said.

The study had no targeted funding, and Dr. Beh had no relevant disclosures. Coauthor Deborah I. Friedman, MD, professor of neurology and neurotherapeutics and ophthalmology at UT Southwestern Medical Center, disclosed serving on advisory boards or speaking for pharmaceutical and medical device companies. Dr. Friedman is on the editorial advisory board of Neurology Reviews.

[email protected]

SOURCE: Beh SC et al. Neurology. 2019 Sep 25. doi: 10.1212/WNL.0000000000008388.

Noninvasive vagus nerve stimulation may reduce the intensity of vertigo and headache in patients with acute vestibular migraine attacks, according to a small, open-label study published online Sept. 25 in Neurology.

The retrospective, single-center study included data from 18 patients who had vestibular migraine and received acute treatment with a handheld stimulation device. The device used in the study, GammaCore, is approved for the treatment of migraine. There are no approved treatments for vestibular migraine.

“There’s a huge need for effective treatments for vestibular migraine attacks,” first author Shin C. Beh, MD, said in a news release. “People with vestibular migraine do not always have headaches, and when they do, they are often less severe than in typical migraine, so the pain-relieving drugs used for typical migraine often are not effective. People can take drugs that suppress the vertigo or the nausea, but those drugs cause drowsiness and make it hard for people to go about their usual activities.” Dr. Beh is an assistant professor of neurology and neurotherapeutics at University of Texas Southwestern Medical Center in Dallas.

The patients had an average age of about 46 years, and 16 were women. A total of 14 patients received treatment during an acute vestibular migraine attack, and 4 received treatment while they had bothersome interictal dizziness consistent with persistent perceptual postural dizziness.

After stimulation, vertigo improved in 13 of the 14 people with vestibular migraine attacks. In two of these patients, vertigo resolved completely, and five had at least a 50% improvement in vertigo symptoms. On a 0-10 scale, patients’ mean vertigo rating decreased from 5.2 before stimulation to 3.1 after stimulation.

Of five patients with vestibular migraine attacks who had headache, all experienced a reduction in headache pain. Mean headache severity decreased from 6 before stimulation to 2.4 after stimulation.

None of the four patients with interictal dizziness experienced a reduction in dizziness after stimulation.

Patients place the device against the neck to receive electrical stimulation, and patients received stimulation for 2 minutes on each side of the neck.

Participants reported a mild pulling sensation of the neck muscles during the stimulation but did not report pain or other side effects.

“Vestibular migraine is the most common neurologic cause of vertigo and can greatly interfere with a person’s daily life,” Dr. Beh said. “If these results can be confirmed with larger studies, not only could there finally be a treatment for vestibular migraine, such a treatment would also be easy to use.”

A randomized, double-blind, sham-controlled study is needed to further assess the use of noninvasive vagus nerve stimulation in treating vestibular migraine, the authors said.

The study had no targeted funding, and Dr. Beh had no relevant disclosures. Coauthor Deborah I. Friedman, MD, professor of neurology and neurotherapeutics and ophthalmology at UT Southwestern Medical Center, disclosed serving on advisory boards or speaking for pharmaceutical and medical device companies. Dr. Friedman is on the editorial advisory board of Neurology Reviews.

[email protected]

SOURCE: Beh SC et al. Neurology. 2019 Sep 25. doi: 10.1212/WNL.0000000000008388.

Norman E. Sharpless, MD, acting commissioner of the Food and Drug Administration, has issued remarks that were prepared for a testimony before a U.S. House Energy and Commerce subcommittee on FDA regulation of electronic nicotine delivery systems and investigation of vaping illnesses.

Dr. Sharpless’s statement focuses on two priorities: the continuing investigation into the cause of lung injury associated with the use of vaping products and the FDA’s ongoing efforts to address an epidemic of youth use of electronic nicotine delivery systems (ENDS). In regards to lung injuries associated with vaping, Dr. Sharpless said that the FDA and Centers for Disease Control and Prevention, in conjunction with state partners, have been investigating the outbreak and noted that, while most cases have involved tetrahydrocannabinol, there is as of yet no common cause or product linked to all cases.

The FDA is not currently pursuing any legal action against personal usage of vaping products, but “if we determine that someone is manufacturing or distributing illicit, adulterated vaping products that caused illness and death for personal profit, we would consider that to be a criminal act,” Dr. Sharpless said.

Research from the 2018 and 2019 National Youth Tobacco Surveys has indicated that ENDS usage among youth has risen dramatically in recent years, Dr. Sharpless continued in the statement. The FDA has pursued several courses of action, including the issue of a warning letter to Juul for marketing unauthorized modified-risk tobacco products to children.

However, he noted, youth e-cigarette use continues to rise, which is the reason for the FDA’s intention to finalize a compliance policy related to flavored ENDS, a policy supported by President TRump.

“FDA is not ‘banning’ flavors, as has been described in some news reports. Rather, FDA intends to enforce existing law that limits the marketing of such products,” Dr. Sharpless said. “This policy would not mean that flavored e-cigarettes could never be marketed. If a company can show through an application to FDA that a specific product meets the standard set forth by Congress, then the FDA would authorize that ENDS product for sale.”

Find the full statement on the FDA website.

[email protected]

Norman E. Sharpless, MD, acting commissioner of the Food and Drug Administration, has issued remarks that were prepared for a testimony before a U.S. House Energy and Commerce subcommittee on FDA regulation of electronic nicotine delivery systems and investigation of vaping illnesses.

Dr. Sharpless’s statement focuses on two priorities: the continuing investigation into the cause of lung injury associated with the use of vaping products and the FDA’s ongoing efforts to address an epidemic of youth use of electronic nicotine delivery systems (ENDS). In regards to lung injuries associated with vaping, Dr. Sharpless said that the FDA and Centers for Disease Control and Prevention, in conjunction with state partners, have been investigating the outbreak and noted that, while most cases have involved tetrahydrocannabinol, there is as of yet no common cause or product linked to all cases.

The FDA is not currently pursuing any legal action against personal usage of vaping products, but “if we determine that someone is manufacturing or distributing illicit, adulterated vaping products that caused illness and death for personal profit, we would consider that to be a criminal act,” Dr. Sharpless said.

Research from the 2018 and 2019 National Youth Tobacco Surveys has indicated that ENDS usage among youth has risen dramatically in recent years, Dr. Sharpless continued in the statement. The FDA has pursued several courses of action, including the issue of a warning letter to Juul for marketing unauthorized modified-risk tobacco products to children.

However, he noted, youth e-cigarette use continues to rise, which is the reason for the FDA’s intention to finalize a compliance policy related to flavored ENDS, a policy supported by President TRump.

“FDA is not ‘banning’ flavors, as has been described in some news reports. Rather, FDA intends to enforce existing law that limits the marketing of such products,” Dr. Sharpless said. “This policy would not mean that flavored e-cigarettes could never be marketed. If a company can show through an application to FDA that a specific product meets the standard set forth by Congress, then the FDA would authorize that ENDS product for sale.”

Find the full statement on the FDA website.

[email protected]

Norman E. Sharpless, MD, acting commissioner of the Food and Drug Administration, has issued remarks that were prepared for a testimony before a U.S. House Energy and Commerce subcommittee on FDA regulation of electronic nicotine delivery systems and investigation of vaping illnesses.

Dr. Sharpless’s statement focuses on two priorities: the continuing investigation into the cause of lung injury associated with the use of vaping products and the FDA’s ongoing efforts to address an epidemic of youth use of electronic nicotine delivery systems (ENDS). In regards to lung injuries associated with vaping, Dr. Sharpless said that the FDA and Centers for Disease Control and Prevention, in conjunction with state partners, have been investigating the outbreak and noted that, while most cases have involved tetrahydrocannabinol, there is as of yet no common cause or product linked to all cases.

The FDA is not currently pursuing any legal action against personal usage of vaping products, but “if we determine that someone is manufacturing or distributing illicit, adulterated vaping products that caused illness and death for personal profit, we would consider that to be a criminal act,” Dr. Sharpless said.

Research from the 2018 and 2019 National Youth Tobacco Surveys has indicated that ENDS usage among youth has risen dramatically in recent years, Dr. Sharpless continued in the statement. The FDA has pursued several courses of action, including the issue of a warning letter to Juul for marketing unauthorized modified-risk tobacco products to children.

However, he noted, youth e-cigarette use continues to rise, which is the reason for the FDA’s intention to finalize a compliance policy related to flavored ENDS, a policy supported by President TRump.

“FDA is not ‘banning’ flavors, as has been described in some news reports. Rather, FDA intends to enforce existing law that limits the marketing of such products,” Dr. Sharpless said. “This policy would not mean that flavored e-cigarettes could never be marketed. If a company can show through an application to FDA that a specific product meets the standard set forth by Congress, then the FDA would authorize that ENDS product for sale.”

Find the full statement on the FDA website.

[email protected]

Key clinical point: A small cohort of patients with CIS meeting MS diagnostic criteria stopped DMT after 5 years of no disease activity, and just under half had MS recurrence.

Major finding: Of the cohort of 46 patients, 23 had disease recurrence, with recurrence more likely among younger patients.

Study details: Cohort study of 46 patients with initial clinical episode of MS, meeting Barkhof criteria for MS on magnetic resonance imaging.

Disclosures: The authors reported no conflicts of interest. No external sources of funding were reported.

Citation: Monschein T et al. ECTRIMS 2019. Abstract P654.

Key clinical point: A small cohort of patients with CIS meeting MS diagnostic criteria stopped DMT after 5 years of no disease activity, and just under half had MS recurrence.

Major finding: Of the cohort of 46 patients, 23 had disease recurrence, with recurrence more likely among younger patients.

Study details: Cohort study of 46 patients with initial clinical episode of MS, meeting Barkhof criteria for MS on magnetic resonance imaging.

Disclosures: The authors reported no conflicts of interest. No external sources of funding were reported.

Citation: Monschein T et al. ECTRIMS 2019. Abstract P654.

Key clinical point: A small cohort of patients with CIS meeting MS diagnostic criteria stopped DMT after 5 years of no disease activity, and just under half had MS recurrence.

Major finding: Of the cohort of 46 patients, 23 had disease recurrence, with recurrence more likely among younger patients.

Study details: Cohort study of 46 patients with initial clinical episode of MS, meeting Barkhof criteria for MS on magnetic resonance imaging.

Disclosures: The authors reported no conflicts of interest. No external sources of funding were reported.

Citation: Monschein T et al. ECTRIMS 2019. Abstract P654.

Key clinical point: Newer oral and intravenous drugs for multiple schlerosis provide significantly better disease activity control than the older injectables as initial disease-modifying therapy in children.

Major finding: The number needed to treat with a newer disease-modifying drug rather than an injectable agent to prevent one relapse was 3.7.

Study details: A prospective, multicenter, observational cohort study including 741 pediatric patients with MS followed while on their first-time disease-modifying therapy.

Disclosures: The study was sponsored by the Multiple Sclerosis Society. Dr. Kysko reported having no financial conflicts in regard to the study.

Citation: Krysko KM. ECTRIMS 2019, abstract 249.

Key clinical point: Newer oral and intravenous drugs for multiple schlerosis provide significantly better disease activity control than the older injectables as initial disease-modifying therapy in children.

Major finding: The number needed to treat with a newer disease-modifying drug rather than an injectable agent to prevent one relapse was 3.7.

Study details: A prospective, multicenter, observational cohort study including 741 pediatric patients with MS followed while on their first-time disease-modifying therapy.

Disclosures: The study was sponsored by the Multiple Sclerosis Society. Dr. Kysko reported having no financial conflicts in regard to the study.

Citation: Krysko KM. ECTRIMS 2019, abstract 249.

Key clinical point: Newer oral and intravenous drugs for multiple schlerosis provide significantly better disease activity control than the older injectables as initial disease-modifying therapy in children.

Major finding: The number needed to treat with a newer disease-modifying drug rather than an injectable agent to prevent one relapse was 3.7.

Study details: A prospective, multicenter, observational cohort study including 741 pediatric patients with MS followed while on their first-time disease-modifying therapy.

Disclosures: The study was sponsored by the Multiple Sclerosis Society. Dr. Kysko reported having no financial conflicts in regard to the study.

Citation: Krysko KM. ECTRIMS 2019, abstract 249.

Key clinical point: Treatment with DMT for more than 10 years reduces the risk of confirmed disability progression in MS.

Major finding: Continuous treatment with DMT reduced the risk of 24-month confirmed disability progression by 35%.

Study details: A retrospective analysis of data for 15,602 patients with relapsing-remitting MS.

Disclosures: Two of the researchers are employees of Biogen International, which supported the research. Several investigators received compensation or funding from various pharmaceutical companies.

Citation: Laffaldano P et al. ECTRIMS 2019. Abstract 94.

Key clinical point: Treatment with DMT for more than 10 years reduces the risk of confirmed disability progression in MS.

Major finding: Continuous treatment with DMT reduced the risk of 24-month confirmed disability progression by 35%.

Study details: A retrospective analysis of data for 15,602 patients with relapsing-remitting MS.

Disclosures: Two of the researchers are employees of Biogen International, which supported the research. Several investigators received compensation or funding from various pharmaceutical companies.

Citation: Laffaldano P et al. ECTRIMS 2019. Abstract 94.

Key clinical point: Treatment with DMT for more than 10 years reduces the risk of confirmed disability progression in MS.

Major finding: Continuous treatment with DMT reduced the risk of 24-month confirmed disability progression by 35%.

Study details: A retrospective analysis of data for 15,602 patients with relapsing-remitting MS.

Disclosures: Two of the researchers are employees of Biogen International, which supported the research. Several investigators received compensation or funding from various pharmaceutical companies.

Citation: Laffaldano P et al. ECTRIMS 2019. Abstract 94.

The Food and Drug Administration has alerted health care professionals and patients about a voluntary recall of some prescription ranitidine (Zantac) because of detected N-nitrosodimethylamine (NDMA) levels, according to a news release from the agency.

The recall applies to 14 lots in which NDMA, a probable human carcinogen and nitrosamine impurity formed as a byproduct of several industrial and natural processes, has been detected at levels above those set by the FDA, according to a company announcement on Sept. 23 from Sandoz. According to the announcement, which also specifies the affected lots, the company has not received any reports of adverse events related to use of the products in the recall.

According to the FDA release, so far, only the specified lots of ranitidine are known to be contaminated, and patients can continue taking this stomach acid–reducing histamine₂ blocker from lots that are not affected by the recall.

“When we identify lapses in the quality of drugs that pose potential risks for patients, the FDA makes all efforts to understand the issue and provide our best recommendation to the public as quickly and accurately as possible,” said acting FDA Commissioner Norman E. Sharpless, MD.

As part of this ongoing investigation, the FDA recently posted a testing protocol for detecting NDMA in ranitidine; the agency hopes regulators and industry will use this protocol to begin their own laboratory testing as well and send samples to the FDA for further testing.

More information about the recall, as well as instructions for patients and health care professionals, can be found in the full news release on the FDA website. The agency also encourages any adverse reactions be reported to its MedWatch program.

[email protected]

The Food and Drug Administration has alerted health care professionals and patients about a voluntary recall of some prescription ranitidine (Zantac) because of detected N-nitrosodimethylamine (NDMA) levels, according to a news release from the agency.

The recall applies to 14 lots in which NDMA, a probable human carcinogen and nitrosamine impurity formed as a byproduct of several industrial and natural processes, has been detected at levels above those set by the FDA, according to a company announcement on Sept. 23 from Sandoz. According to the announcement, which also specifies the affected lots, the company has not received any reports of adverse events related to use of the products in the recall.

According to the FDA release, so far, only the specified lots of ranitidine are known to be contaminated, and patients can continue taking this stomach acid–reducing histamine₂ blocker from lots that are not affected by the recall.

“When we identify lapses in the quality of drugs that pose potential risks for patients, the FDA makes all efforts to understand the issue and provide our best recommendation to the public as quickly and accurately as possible,” said acting FDA Commissioner Norman E. Sharpless, MD.

As part of this ongoing investigation, the FDA recently posted a testing protocol for detecting NDMA in ranitidine; the agency hopes regulators and industry will use this protocol to begin their own laboratory testing as well and send samples to the FDA for further testing.

More information about the recall, as well as instructions for patients and health care professionals, can be found in the full news release on the FDA website. The agency also encourages any adverse reactions be reported to its MedWatch program.

[email protected]

The Food and Drug Administration has alerted health care professionals and patients about a voluntary recall of some prescription ranitidine (Zantac) because of detected N-nitrosodimethylamine (NDMA) levels, according to a news release from the agency.

The recall applies to 14 lots in which NDMA, a probable human carcinogen and nitrosamine impurity formed as a byproduct of several industrial and natural processes, has been detected at levels above those set by the FDA, according to a company announcement on Sept. 23 from Sandoz. According to the announcement, which also specifies the affected lots, the company has not received any reports of adverse events related to use of the products in the recall.

According to the FDA release, so far, only the specified lots of ranitidine are known to be contaminated, and patients can continue taking this stomach acid–reducing histamine₂ blocker from lots that are not affected by the recall.

“When we identify lapses in the quality of drugs that pose potential risks for patients, the FDA makes all efforts to understand the issue and provide our best recommendation to the public as quickly and accurately as possible,” said acting FDA Commissioner Norman E. Sharpless, MD.

As part of this ongoing investigation, the FDA recently posted a testing protocol for detecting NDMA in ranitidine; the agency hopes regulators and industry will use this protocol to begin their own laboratory testing as well and send samples to the FDA for further testing.

More information about the recall, as well as instructions for patients and health care professionals, can be found in the full news release on the FDA website. The agency also encourages any adverse reactions be reported to its MedWatch program.

[email protected]

COPENHAGEN – Lumateperone, a novel investigational drug for schizophrenia with a unique triple mechanism of action, showed impressive safety and tolerability while achieving a continuous decline in schizophrenia symptoms over the course of a year in a long-term, open-label study, Suresh Durgam, MD, said at the annual congress of the European College of Neuropsychopharmacology.

Bruce Jancin/MDedge News

Indeed, patients on lumateperone at the 1-year mark showed significant reductions in LDL cholesterol, total cholesterol, serum prolactin, and body weight, compared with baseline values recorded when participants were on various standard-of-care antipsychotics prior to switching. Other cardiometabolic parameters, including fasting blood glucose, insulin, triglycerides, and HDL cholesterol, showed only negligible change over the course of study, according to Dr. Durgam, a psychiatrist and senior vice president for late-stage clinical development and medical affairs at Intra-Cellular Therapies, the New York–based company developing lumateperone as its lead product.

This favorable cardiometabolic profile contrasts sharply with those of currently available antipsychotic agents, many of which worsen cardiometabolic risk factors. That would seem to be a major advantage for lumateperone and is likely to be a factor in the Food and Drug Administration’s ongoing deliberation over the company’s new drug application. The agency has promised a decision on the application by the end of December, Dr. Durgam said.

Intra-Cellular Therapies’ stock price took a hit in July 2019, when the FDA abruptly canceled an advisory committee meeting scheduled to consider lumateperone. The agency sought additional information on animal toxicology studies. Having received it from the company, the FDA now no longer plans to schedule an advisory committee meeting before issuing its marketing approval decision.

Lumateperone is an oral once-daily drug that doesn’t require titration. Its high degree of tolerability is thought to be attributable to the drug’s mechanism of action, which involves simultaneous modulation of three different neurotransmitter pathways: serotonin, dopamine, and glutamate. The drug is a potent serotonin 5-HT_2a antagonist and serotonin reuptake inhibitor, a dopamine D2 presynaptic partial agonist and postsynaptic antagonist, and it also modulates glutamate via activation of the D1 receptor.

Three phase 3, double-blind, placebo-controlled randomized clinical trials of 4-6 weeks duration have been completed in a total of 1,481 patients with acute exacerbation of schizophrenia. Two trials were positive, with lumateperone achieving significantly greater mean reductions in the Positive and Negative Syndrome Scale (PANSS) total score than placebo, while the third was negative, with no significant between-group difference. Of note, the safety profile of lumateperone was indistinguishable from placebo with the sole exception of somnolence, where the 20% incidence was twice that of placebo-treated controls. However, in the open-label program, dosing was switched from morning to evening, with a resultant drop in somnolence to the placebo level, Dr. Durgam said.

The open-label program has two parts. Part 1 was conducted in 302 patients with stable, generally mild schizophrenia symptoms while on risperidone, olanzapine, or various other antipsychotics commonly prescribed in the United States. They were switched to lumateperone at 42 mg once daily for 6 weeks, at which point they demonstrated significant reductions in body weight, serum prolactin, insulin, total cholesterol, and LDL cholesterol. They then were switched back to their former medications, with a resultant worsening of those parameters to prelumateperone levels, providing evidence of a cause-and-effect relationship with cardiometabolic risk factors.

Part 2 of the open-label program is the long-term study, in which 603 patients with stable symptoms on standard-of-care antipsychotics were switched to lumateperone at 42 mg/day, to be followed for 1 year or more. Dr. Durgam presented an interim analysis focused on the first 107 patients to achieve the 1-year treatment milestone. Most were obese at baseline: the group’s mean body mass index was 31.3 kg/m2. They experienced progressive weight loss, with a mean reduction of 1.82 kg on day 175 and 3.16 kg on day 350. About 24% of subjects had a 7% or greater reduction in body weight, while 8% had at least a 7% weight gain. Waist circumference decreased by an average of 5.2 cm from a baseline of 103.2 cm in men and by 1.9 cm in women.

The primary focus of the ongoing long-term study is safety. The most common treatment-emergent adverse events during a full year of therapy were dry mouth, headache, and diarrhea, each occurring in about 7% of patients. Only 0.8% of patients developed extrapyramidal symptoms.

At 150 days of treatment in 340 patients, 30% had achieved a PANSS response, defined as at least a 20% improvement in PANSS total score, compared with baseline. At 300 days in the smaller group who had reached that milestone at the time of the interim analysis, the PANSS response rate had grown to 41%.

Among patients with schizophrenia and comorbid depression as defined by a Calgary Depression Scale for Schizophrenia (CDSS) score of 6 or more at baseline, lumateperone at 42 mg/day improved depressive symptoms, such that 60% of those patients achieved a CDSS response – that is, at least a 50% reduction in the score – by day 75. This finding supports data from earlier short-term studies, and suggests that lumateperone’s multiple mechanisms of action and high tolerability make it a promising candidate for treatment of depression and other symptom domains of schizophrenia that are currently inadequately treated, according to Dr. Durgam.

Dr. Durgam also presented an update on the lumateperone program for bipolar depression, which consists of three phase 3, double-blind, placebo-controlled, 6-week-long clinical trials totaling 1,455 patients. Two have been completed: one positive and the other negative with an unusually high placebo response rate. The ongoing third trial will be the tiebreaker. Safety and tolerability have been as noted in other lumateperone studies.

In the positive trial, the primary efficacy endpoint was change in Montgomery-Åsberg Depression Rating Scale, which improved in lumateperone-treated patients by an average of 16.7 points from a baseline score of just over 30, a significantly better result than the 12.1-point reduction in placebo-treated controls. The treatment benefit was similar in bipolar I and bipolar II patients.

The phase 3 trial* for treatment of agitation in patients with Alzheimer’s disease and other dementias was stopped early for lack of efficacy in an interim analysis. And lumateperone is in ongoing phase 2 trials for sleep disturbances associated with neuropsychiatric disorders. The phase 2 study* in major depressive disorder has been completed.

[email protected]

*This article was updated 9/28/2019.

COPENHAGEN – Lumateperone, a novel investigational drug for schizophrenia with a unique triple mechanism of action, showed impressive safety and tolerability while achieving a continuous decline in schizophrenia symptoms over the course of a year in a long-term, open-label study, Suresh Durgam, MD, said at the annual congress of the European College of Neuropsychopharmacology.

Bruce Jancin/MDedge News

Indeed, patients on lumateperone at the 1-year mark showed significant reductions in LDL cholesterol, total cholesterol, serum prolactin, and body weight, compared with baseline values recorded when participants were on various standard-of-care antipsychotics prior to switching. Other cardiometabolic parameters, including fasting blood glucose, insulin, triglycerides, and HDL cholesterol, showed only negligible change over the course of study, according to Dr. Durgam, a psychiatrist and senior vice president for late-stage clinical development and medical affairs at Intra-Cellular Therapies, the New York–based company developing lumateperone as its lead product.

This favorable cardiometabolic profile contrasts sharply with those of currently available antipsychotic agents, many of which worsen cardiometabolic risk factors. That would seem to be a major advantage for lumateperone and is likely to be a factor in the Food and Drug Administration’s ongoing deliberation over the company’s new drug application. The agency has promised a decision on the application by the end of December, Dr. Durgam said.

Intra-Cellular Therapies’ stock price took a hit in July 2019, when the FDA abruptly canceled an advisory committee meeting scheduled to consider lumateperone. The agency sought additional information on animal toxicology studies. Having received it from the company, the FDA now no longer plans to schedule an advisory committee meeting before issuing its marketing approval decision.

Lumateperone is an oral once-daily drug that doesn’t require titration. Its high degree of tolerability is thought to be attributable to the drug’s mechanism of action, which involves simultaneous modulation of three different neurotransmitter pathways: serotonin, dopamine, and glutamate. The drug is a potent serotonin 5-HT_2a antagonist and serotonin reuptake inhibitor, a dopamine D2 presynaptic partial agonist and postsynaptic antagonist, and it also modulates glutamate via activation of the D1 receptor.

Three phase 3, double-blind, placebo-controlled randomized clinical trials of 4-6 weeks duration have been completed in a total of 1,481 patients with acute exacerbation of schizophrenia. Two trials were positive, with lumateperone achieving significantly greater mean reductions in the Positive and Negative Syndrome Scale (PANSS) total score than placebo, while the third was negative, with no significant between-group difference. Of note, the safety profile of lumateperone was indistinguishable from placebo with the sole exception of somnolence, where the 20% incidence was twice that of placebo-treated controls. However, in the open-label program, dosing was switched from morning to evening, with a resultant drop in somnolence to the placebo level, Dr. Durgam said.

The open-label program has two parts. Part 1 was conducted in 302 patients with stable, generally mild schizophrenia symptoms while on risperidone, olanzapine, or various other antipsychotics commonly prescribed in the United States. They were switched to lumateperone at 42 mg once daily for 6 weeks, at which point they demonstrated significant reductions in body weight, serum prolactin, insulin, total cholesterol, and LDL cholesterol. They then were switched back to their former medications, with a resultant worsening of those parameters to prelumateperone levels, providing evidence of a cause-and-effect relationship with cardiometabolic risk factors.

Part 2 of the open-label program is the long-term study, in which 603 patients with stable symptoms on standard-of-care antipsychotics were switched to lumateperone at 42 mg/day, to be followed for 1 year or more. Dr. Durgam presented an interim analysis focused on the first 107 patients to achieve the 1-year treatment milestone. Most were obese at baseline: the group’s mean body mass index was 31.3 kg/m2. They experienced progressive weight loss, with a mean reduction of 1.82 kg on day 175 and 3.16 kg on day 350. About 24% of subjects had a 7% or greater reduction in body weight, while 8% had at least a 7% weight gain. Waist circumference decreased by an average of 5.2 cm from a baseline of 103.2 cm in men and by 1.9 cm in women.

The primary focus of the ongoing long-term study is safety. The most common treatment-emergent adverse events during a full year of therapy were dry mouth, headache, and diarrhea, each occurring in about 7% of patients. Only 0.8% of patients developed extrapyramidal symptoms.

At 150 days of treatment in 340 patients, 30% had achieved a PANSS response, defined as at least a 20% improvement in PANSS total score, compared with baseline. At 300 days in the smaller group who had reached that milestone at the time of the interim analysis, the PANSS response rate had grown to 41%.

Among patients with schizophrenia and comorbid depression as defined by a Calgary Depression Scale for Schizophrenia (CDSS) score of 6 or more at baseline, lumateperone at 42 mg/day improved depressive symptoms, such that 60% of those patients achieved a CDSS response – that is, at least a 50% reduction in the score – by day 75. This finding supports data from earlier short-term studies, and suggests that lumateperone’s multiple mechanisms of action and high tolerability make it a promising candidate for treatment of depression and other symptom domains of schizophrenia that are currently inadequately treated, according to Dr. Durgam.

Dr. Durgam also presented an update on the lumateperone program for bipolar depression, which consists of three phase 3, double-blind, placebo-controlled, 6-week-long clinical trials totaling 1,455 patients. Two have been completed: one positive and the other negative with an unusually high placebo response rate. The ongoing third trial will be the tiebreaker. Safety and tolerability have been as noted in other lumateperone studies.

In the positive trial, the primary efficacy endpoint was change in Montgomery-Åsberg Depression Rating Scale, which improved in lumateperone-treated patients by an average of 16.7 points from a baseline score of just over 30, a significantly better result than the 12.1-point reduction in placebo-treated controls. The treatment benefit was similar in bipolar I and bipolar II patients.

The phase 3 trial* for treatment of agitation in patients with Alzheimer’s disease and other dementias was stopped early for lack of efficacy in an interim analysis. And lumateperone is in ongoing phase 2 trials for sleep disturbances associated with neuropsychiatric disorders. The phase 2 study* in major depressive disorder has been completed.

[email protected]

*This article was updated 9/28/2019.

COPENHAGEN – Lumateperone, a novel investigational drug for schizophrenia with a unique triple mechanism of action, showed impressive safety and tolerability while achieving a continuous decline in schizophrenia symptoms over the course of a year in a long-term, open-label study, Suresh Durgam, MD, said at the annual congress of the European College of Neuropsychopharmacology.

Bruce Jancin/MDedge News

Indeed, patients on lumateperone at the 1-year mark showed significant reductions in LDL cholesterol, total cholesterol, serum prolactin, and body weight, compared with baseline values recorded when participants were on various standard-of-care antipsychotics prior to switching. Other cardiometabolic parameters, including fasting blood glucose, insulin, triglycerides, and HDL cholesterol, showed only negligible change over the course of study, according to Dr. Durgam, a psychiatrist and senior vice president for late-stage clinical development and medical affairs at Intra-Cellular Therapies, the New York–based company developing lumateperone as its lead product.

This favorable cardiometabolic profile contrasts sharply with those of currently available antipsychotic agents, many of which worsen cardiometabolic risk factors. That would seem to be a major advantage for lumateperone and is likely to be a factor in the Food and Drug Administration’s ongoing deliberation over the company’s new drug application. The agency has promised a decision on the application by the end of December, Dr. Durgam said.

Intra-Cellular Therapies’ stock price took a hit in July 2019, when the FDA abruptly canceled an advisory committee meeting scheduled to consider lumateperone. The agency sought additional information on animal toxicology studies. Having received it from the company, the FDA now no longer plans to schedule an advisory committee meeting before issuing its marketing approval decision.

Lumateperone is an oral once-daily drug that doesn’t require titration. Its high degree of tolerability is thought to be attributable to the drug’s mechanism of action, which involves simultaneous modulation of three different neurotransmitter pathways: serotonin, dopamine, and glutamate. The drug is a potent serotonin 5-HT_2a antagonist and serotonin reuptake inhibitor, a dopamine D2 presynaptic partial agonist and postsynaptic antagonist, and it also modulates glutamate via activation of the D1 receptor.

Three phase 3, double-blind, placebo-controlled randomized clinical trials of 4-6 weeks duration have been completed in a total of 1,481 patients with acute exacerbation of schizophrenia. Two trials were positive, with lumateperone achieving significantly greater mean reductions in the Positive and Negative Syndrome Scale (PANSS) total score than placebo, while the third was negative, with no significant between-group difference. Of note, the safety profile of lumateperone was indistinguishable from placebo with the sole exception of somnolence, where the 20% incidence was twice that of placebo-treated controls. However, in the open-label program, dosing was switched from morning to evening, with a resultant drop in somnolence to the placebo level, Dr. Durgam said.

The open-label program has two parts. Part 1 was conducted in 302 patients with stable, generally mild schizophrenia symptoms while on risperidone, olanzapine, or various other antipsychotics commonly prescribed in the United States. They were switched to lumateperone at 42 mg once daily for 6 weeks, at which point they demonstrated significant reductions in body weight, serum prolactin, insulin, total cholesterol, and LDL cholesterol. They then were switched back to their former medications, with a resultant worsening of those parameters to prelumateperone levels, providing evidence of a cause-and-effect relationship with cardiometabolic risk factors.

Part 2 of the open-label program is the long-term study, in which 603 patients with stable symptoms on standard-of-care antipsychotics were switched to lumateperone at 42 mg/day, to be followed for 1 year or more. Dr. Durgam presented an interim analysis focused on the first 107 patients to achieve the 1-year treatment milestone. Most were obese at baseline: the group’s mean body mass index was 31.3 kg/m2. They experienced progressive weight loss, with a mean reduction of 1.82 kg on day 175 and 3.16 kg on day 350. About 24% of subjects had a 7% or greater reduction in body weight, while 8% had at least a 7% weight gain. Waist circumference decreased by an average of 5.2 cm from a baseline of 103.2 cm in men and by 1.9 cm in women.

The primary focus of the ongoing long-term study is safety. The most common treatment-emergent adverse events during a full year of therapy were dry mouth, headache, and diarrhea, each occurring in about 7% of patients. Only 0.8% of patients developed extrapyramidal symptoms.

At 150 days of treatment in 340 patients, 30% had achieved a PANSS response, defined as at least a 20% improvement in PANSS total score, compared with baseline. At 300 days in the smaller group who had reached that milestone at the time of the interim analysis, the PANSS response rate had grown to 41%.

Among patients with schizophrenia and comorbid depression as defined by a Calgary Depression Scale for Schizophrenia (CDSS) score of 6 or more at baseline, lumateperone at 42 mg/day improved depressive symptoms, such that 60% of those patients achieved a CDSS response – that is, at least a 50% reduction in the score – by day 75. This finding supports data from earlier short-term studies, and suggests that lumateperone’s multiple mechanisms of action and high tolerability make it a promising candidate for treatment of depression and other symptom domains of schizophrenia that are currently inadequately treated, according to Dr. Durgam.

Dr. Durgam also presented an update on the lumateperone program for bipolar depression, which consists of three phase 3, double-blind, placebo-controlled, 6-week-long clinical trials totaling 1,455 patients. Two have been completed: one positive and the other negative with an unusually high placebo response rate. The ongoing third trial will be the tiebreaker. Safety and tolerability have been as noted in other lumateperone studies.

In the positive trial, the primary efficacy endpoint was change in Montgomery-Åsberg Depression Rating Scale, which improved in lumateperone-treated patients by an average of 16.7 points from a baseline score of just over 30, a significantly better result than the 12.1-point reduction in placebo-treated controls. The treatment benefit was similar in bipolar I and bipolar II patients.

The phase 3 trial* for treatment of agitation in patients with Alzheimer’s disease and other dementias was stopped early for lack of efficacy in an interim analysis. And lumateperone is in ongoing phase 2 trials for sleep disturbances associated with neuropsychiatric disorders. The phase 2 study* in major depressive disorder has been completed.

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*This article was updated 9/28/2019.