An American Red Cross discusses the importance of National Blood Donor Month and the challenges faced during the COVID-19 pandemic.

The National Blood Shortage: An Unprecedented Challenge

New ADA guidelines list more than 40 recommendations related to blood pressure targets, screening and diagnosis, and overall management strategies.

Cardiovascular Disease in Diabetes: How to Manage Risks

What you need to know about vaccinator and patient education, counseling, billing, and reimbursement.

ACP Policy Statement on COVID-19 Vaccine Distribution and Administration

Expert David W. Goodman, MD, explains why adults with ADHD may face unknown challenges during the pandemic.

Adult ADHD Challenges During COVID-19

Anne Dixon, MD, joins Lungcast to discuss obesity's effect on asthma, and the newest recommendations for managed care.

New Asthma Management Guidelines: A Podcast Primer

The US Food and Drug Administration (FDA) has accepted Merck’s New Drug Application (NDA) for gefapixant—an investigational, orally administered, selective P2X3 receptor antagonist, for the treatment of refractory chronic cough (RCC) or unexplained chronic cough (UCC) in adults.

“This submission underscores our commitment to help patients with refractory or unexplained chronic cough who currently have limited treatment options,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories in a 

“If approved by the FDA, gefapixant would be the first medicine approved specifically to help these patients, and we look forward to participating in the advisory committee meeting and working with the FDA as they review our application,” he continued.

Currently, no date for an advisory committee meeting has been scheduled, but the FDA has set a Prescription Drug User Fee Act (PDUFA), or target action date, of Dec. 21, 2021.

The NDA is based on results from the Phase 3 COUGH-1 and COUGH-2 clinical trials, which are the first dual studies to evaluate patients with RCC or UCC.

The multinational, randomized, double-blind, placebo-controlled studies assessed a combined total of 2044 participants (COUGH-1, n = 730; COUGH-2, n = 1314).

Patients were then randomly assigned to receive gefapixant 45 mg twice daily, gefapixant 15 mg twice daily, or placebo.

The primary efficacy outcome measures were 24-hour cough frequency at week 12 for COUGH-1 and 24-hour cough frequency at week 24 for COUGH-2. Frequency was measured using an ambulatory digital audio recording device.

 presented at the Virtual European Respiratory Society (ERS) International Congress 2020 showed that gefapixant 45 mg significantly reduced 24-hour cough frequency versus placebo at 12 weeks (-18.45% [95% CI, -32.92 to -0.86, 

This trend was also seen in COUGH-2. Gefapixant demonstrated a significant reduction at 24 weeks compared with placebo (-14.64% [95% CI, -26.07 to -1.43; P = 0.031]).

However, gexapixant 15 mg did not meant the primary efficacy endpoint in either Phase 3 study.

Articles

The application, submitted by Merck, is based on data from 2 clinical trials evaluating patients with refractory or unexplained chronic cough.

News

Pulmonology

Internal Medicine

FDA Accepts NDA for Gefapixant for Chronic Cough

American Academy of Allergy, Asthma & Immunology (AAAAI) 2021 Virtual

 Sessions this weekend, a late-breaking poster showed just more than half (57%) of pediatric patients with 

 adhered to their controller medication when monitored with a dose-administered feedback device.

, from a team at the Children’s Hospital Colorado, indicates even a high-risk population—who agreed to voluntary treatment monitoring, even—reports asthma controller adherence at a rate close to greater patient population averages.

In the second segment of an interview with 

, study author William Anderson, MD, discussed the concepts of remotely-monitored treatment adherence for asthma, the value of in-person versus telehealth check-ins during the COVID-19 pandemic, and overall thoughts on the virtual AAAAI 2021 sessions.

When doing assessments that involve patients knowingly playing a part of monitored treatment adherence, you would hope for better outcomes from them, in terms of them actually following prescriptions.

But, as you as you alluded to, that's indicative of its own outcome, and the need for better patient confidence in therapy. Would that mean improved education for patients?

 I think it's both education and communication. I think that, for a lot of providers, they don't know exactly what's happening in the home when it comes to taking these medications. And knowing what the rate of the patient's medication adherence is allows them to have that conversation with families to point out, 'Hey, this is the adherence that we're seeing for you. One, do you recognize that that's the only how frequent you're taking your medication? And if so, why is it that you're only taking your medication that often?'

That's where you can really start to analyze some of the barriers that might be present to good medication adherence. Once you've identified those, there's tailoring your treatment options or strategies to what you're learning from the patient.

This broader discussion about remote monitoring and better means of assuring patients are working with confidence of what a prescribing clinician would hope for their treatment. And all that speaks to our current state of isolation and very prioritized in-person care.

But do these findings say anything to value of in-person care—to the idea that it’s something that does help with treatment adherence?

 I can't necessarily say at this point in time whether having less in-person care made a difference one way or the other, because we didn't necessarily look at that in this study. But I will say that it just goes to show the importance of making sure you do continue the continuity of care with these patients. I think that those patients who are not getting routine feedback or are not having these conversations with providers—maybe they are who's more likely start to slip a little bit on taking their medications.

So, if you're able to have those conversations in person, via telehealth, whatever it might be, I think it's just important to make sure that to continue this one way or the other. We don't want to see our patients in a time like COVID be afraid to come into the office, and then we lose them to follow-up, and then their asthma actually gets worse during that time.

So, if you can reach out in terms of an electronic intervention, telehealth, whatever it might be, we certainly want to maintain those on that connection with patients and that care, even during a pandemic.

Is there anything else you would want to add, relative to AAAAI 2021?

 I'm really excited. I'm looking forward to the virtual format. Obviously, it's great that we're able to have the meeting this year. Unfortunately, last year was canceled because of COVID. So, it's hard to believe that it's been a year at this point in time. But it should be a good meeting. Virtual is going to be fantastic, but we're looking forward to the day where we can meet up with our colleagues in-person again, hopefully very soon.

From better education, to remote monitoring and consistent check-ins, clinicians may need to initiate better adherence in their younger, severely ill patients.

Asthma

Conference

Pediatrics

Family Medicine

What Is Needed to Improve Pediatric Asthma Controller Therapy Adherence

Dupilumab (Dupixent) has showed sustained benefit in health-related quality of life (HRQoL) and symptom burden in patients with 

American Academy of Allergy, Asthma & Immunology (AAAAI) 2021 Virtual Sessions

 this week gives hope to possibly nearing the first biologic treatment indicated for the rare disease in the US.

, study author Evan Dellon, MD, MPH, of the Center for Gastrointestinal Biology and Disease, UNC School of Medicine, discussed the burdensome chronic effects of EoE on its varied patient population.

Symptoms can evolve from dysphagia in the young patient population, to esophageal scarring and inflammation which burdens patient swallowing at a younger age.

Though pediatric patients are a particular concern for Dellon and colleagues due to their burdens of pain, regurgitation, food processing failure, and their inability to distinguish between vomiting and EoE symptoms, it’s important for frontline clinicians to remember they’re not the lone at-risk group.

“I think that’s an important point to make—not only for patients that are having dysphagia, but also for providers,” Dellon explained. “Just because you’re seeing a 60-70 year old person who doesn’t fit the young profile, it could still be there.”

Dellon also discussed the very diverse EoE care team, which include caregivers from any of allergy, gastroenterology, pediatrics, primary care, nutrition, diet, and pathology. In terms of prescribing a therapy such as dupilumab for EoE, most practices will have the luxury of choice.

“I think allergists are familiar with this medication for other atopic medications, but GI doctors are quite familiar with administering biologics from the inflammatory bowel disease space,” Dellon said.

Videos

How dupilumab is observed to benefit EoE patients—and who may administer the biologic therapy.

Gastroenterology

Allergy

Diverse Eosinophilic Esophagitis Symptoms and Care Teams

Tezepelumab, an investigative monoclonal antibody therapy that inhibits the thymic stromal lymphopoietin (TSLP), was associated with clinically meaningful exacerbation reductions among patients with 

 at varied eosinophil counts in new phase 3 data.

Results from the NAVIGATOR trial, presented at the 

, show tezepelumab also reduced the risk of hospitalization or emergency department visits over 52 weeks in treated patients versus placebo.

The newest findings for the subcutaneous novel pathway asthma treatment further evidence its benefit for patients suffering from severe, uncontrolled disease.

, study author Andrew Menzies-Gow, BSc, MBBS, PhD, deputy medical director at the Royal Brompton & Harefield Hospitals, discussed the NAVIGATOR findings, and their preceded research of tezepelumab in this key patient population.

Menzies-Gow also discussed the characteristics of severe, uncontrolled asthma, and how a TSLP inhibitor therapy may significantly influence the disease course.

“The excitement around tezepelumab is really around its unique mechanism of action,” he explained. “With tezepelumab, we’re targeting TSLP—so we’re going upstream, we’re looking at one of the epithelial-derived cytokines, which really drives all the downstream inflammation.”

Menzies-Gow also discussed the pursuit of optimal biologic treatment duration for severe asthma, and how this last year’s greater reliance on monoclonal antibodies in the face of COVID-19 may effect the allergic and inflammatory specialty.

“Looking into the future, the next advance might be moving away from injectable biologics to inhaled biologics,” Menzies-Gow said. “That might be the way forward, to let more people have a biologic.”

The study, “Efficacy and safety of tezepelumab in adults and adolescents with severe, uncontrolled asthma: results from the phase 3 NAVIGATOR study,” was presented at AAAAI 2021.

An interview on the TSLP inhibitor's effect on exacerbations, hospitalizations, and disease progress over 52-week findings.

Hospital Medicine

Tezepelumab Benefits Severe, Uncontrolled Asthma in Phase 3 NAVIGATOR Findings

New research shows a recently revised diagnostic criteria is sufficient for emergency departments to identify and treat 

The World Allergy Organization (WAO) proposed in 2019 a revision of the anaphylaxis diagnostic criteria proposed in 2006 by the National Institute of Allergy and Infectious Disease/ Food Allergy and Anaphylaxis Network (NIAD/FAAN).

A team, led by Justine M. Ade, MD, Mayo Clinic, assessed the accuracy of the revised clinical criteria in 2019 compared to the original diagnostic criteria in 2006 in data presented during the 

In the cohort study, the researchers identified a stratified sample of 817 patients presenting to an academic Emergency Department between January 2013 and September 2017. The patients included were identified and sampled based on the presence of ICD-9 or ICD-10 codes associated with an emergency department visit that indicated either anaphylaxis, symptoms potentially secondary to anaphylaxis, or an allergic reaction.

The investigators assessed the diagnostic accuracy of the 2 criteria’s by comparison to expert review by an allergist-immunologist or fellow and calculated test characteristics.

Overall, the 2 criteria’s were very similar, with the addition of severe gastrointestinal symptoms especially with non-food allergens and respiratory compromise with bronchospasm or laryngeal involvement added to the 2016 version of the guidelines.

When properly weighed, the sampling represented a cohort of 2191 patients with a median patient age of 42 years old.

The weighted sensitivity, specificity, positive predictive value (PPV), and negative predictive value (NPV) of the 2006 criteria were 92.6%, 76.9%, 65.3%, and 95.7%, respectively, compared to 93.4%, 72.7%, 61.5%, and 95.9%, respectively for the revised version.

“Despite a select number of cases that may be missed by one criteria or both, the revised NIAID/FAAN clinical criteria have test characteristics very similar to the original NIAID/FAAN criteria and are therefore likely to be useful for the diagnosis of anaphylaxis in the ED,” the authors wrote.

Advocating for better treatment of anaphylaxis and food allergies has been a staple of past AAAAI meetings.

Last year, Dennis K. Ledford, MD, University of South Florida College of Medicine and Division of Allergy and Immunology, explained in an interview with 

Ledford was set to present during a session on anaphylaxis during surgery when the conference was cancelled because of COVID-19, making it 1 of the first medical conferences to be impacted by the pandemic.

“It was to increase awareness of the problem of anaphylaxis around anesthesia or surgery because it's not terribly common, but surgery is a common process,” Ledford said. “And it can be misinterpreted where low blood pressure, for example, is due to the anesthesia and not an anaphylactic reaction.”

Validation of the revised National Institute of Allergy and Infectious Disease / Food Allergy and Anaphylaxis Network Diagnostic Criteria in Emergency Department Patients

In data presented during AAAAI 21, researchers compared the 2006 and 216 versions of the diagnostic criteria proposed by the NIAD/FAAN. 

Anaphylaxis

Pain

Revised Anaphylaxis Diagnostic Guidelines Seen as an Improvement

Perspective on how the interleukin-targeting biologic has unique benefit for the rare, under-resourced disease.

Rare Disease Report®

Dupilumab for Eosinophilic Esophagitis

Oxygen saturation levels similar for both asthma patients and non-asthma patients following mask use. 

Coronavirus

Infectious Disease

Masks Deemed Safe for Asthma Patients

Podcasts

In this first episode, Kim Smith-Whitley, MD, discusses the history, presentations, and research status of sickle cell disease.

Sickle Cell

The Rare Disease Report Podcast: Sickle Cell Disease

MYMD-1 targets the root cause of inflammation for patients with IPF.

Researchers Ready New Treatment for IPF

Individuals with PH are often undiagnosed or misdiagnosed; therefore, the exact prevalence of the disorder is unclear. 

Nephrology

American Kidney Fund Launches Awareness Campaign for Primary Hyperolaxuria 

Lisa Sarfaty, director of strategic planning for the National Organization for Rare Disorders, speaks on the role of the organization and the importance of the Rare Disease Day campaign.

Welcome to Rare Disease Day: An Interview with a NORD Representative

Late-breaking data show allegic children as young as 1-4 years old may have sustained benefit from daily peanut protein exposure.

Sublingual Immunotherapy May Provide Peanut Allergy Desensitization in Toddlers

An interview with a Children's Hospital Colorado investigator on new remote-monitoring research into children's adherence trends.

Study: Pediatric Adherence to Drugs Treating Severe Asthma is About 57%

Trials testing hydroxychloroquine against COVID-19 have produced mixed results.

Rheumatology

Early Phase Trial Results Positive for HCQ Against COVID-19

The adjusted allergy prevalence in recent years was 2.6% in infants who have been introduced to peanuts at 12 months.

Latest Clinical News

FDA Accepts NDA for Gefapixant for Chronic Cough

What Is Needed to Improve Pediatric Asthma Controller Therapy Adherence

Diverse Eosinophilic Esophagitis Symptoms and Care Teams

Tezepelumab Benefits Severe, Uncontrolled Asthma in Phase 3 NAVIGATOR Findings

Revised Anaphylaxis Diagnostic Guidelines Seen as an Improvement