The panel concludes by discussing the long-term outcomes for patients treated for hypercortisolism.

Long-Term Outcomes for Patients Treated for Hypercortisolism

The panel concludes by discussing the long-term outcomes for patients treated for hypercortisolism, emphasizing the need for further studies and data, and sharing their final thoughts on the disease and its future management.

Honey East, MD

Chalmers discussed how brensocatib’s potential approval may transform the currently empty treatment landscape for bronchiectasis.

Investigating Brensocatib, Potential First Treatment for Bronchiectasis, with James Chalmers, MBChB, PhD

The FDA has accepted and granted priority review to Insmed’s New Drug Application (NDA) for brensocatib, a potential first treatment for patients with non-cystic fibrosis bronchiectasis. The therapy has a Prescription Drug User Fee Act (PDUFA) of August 12, 2025.

The NDA submission was based on data from the landmark Phase 3 ASPEN study, which met its primary endpoint of clinically and statistically significantly reducing the annualized rate of pulmonary exacerbations compared with placebo over 52 weeks in both its 10 and 25 mg dosages. The study assessed brensocatib 10 mg, brensocatib 25 mg, or placebo once daily for 52 weeks, followed by 4 weeks off treatment, in adults and adolescents.

® spoke with James Chalmers, MBChB, PhD, Professor, Asthma and Lung UK Chair of Respiratory Research, MBChB, PhD, FRCPE, FERS, Clinical Professor (Teaching and Research) of Respiratory Research, Respiratory Medicine and Gastroenterology, University of Dundee, United Kingdom, who served as a primary investigator on the ASPEN study, to learn more about bronchiectasis and its unmet needs in the field. He discussed brensocatib and how it may benefit patients with bronchiectasis as the potential first treatment to be approved for the condition.

“[Bronchiectasis has] been neglected, a bit of a Cinderella condition. In 2011 the European Respiratory Society… described bronchiectasis as the most neglected disease in respiratory medicine, because there's so many patients, but nobody was really doing research into it, and nobody, nobody was doing any clinical trials. And so, it's really exciting that that's now changing, because our patients really need it to change. And this [potential] first licensed therapy is a step towards addressing that major burden,” Chalmers told 

Chalmers shared his excitement for the potential FDA approval of brensocatib and also discussed some priorities in bronchiectasis research and care that will follow after getting a first treatment.

FDA Grants Priority Review to Insmed's Brensocatib for Treatment of Bronchiectasis with PDUFA Target Action Date Set for August 12, 2025. News release. Insmed. February 6, 2025. https://www.prnewswire.com/news-releases/fda-grants-priority-review-to-insmeds-brensocatib-for-treatment-of-bronchiectasis-with-pdufa-target-action-date-set-for-august-12-2025-302369466.html

James Chalmers, MBChB, PhD

Joseph, do you have any final thoughts about, or takeaways from, this discussion?

Education is important for everybody. Many health care providers, including myself, always had this feeling about FMT [fecal microbiota transplant]. Is it dangerous? Will patients wind up getting sick? Where does the stool come from? Is it a mix of donors or a single donor? How much will it cost? And the whole idea of a colonoscopy with bowel prep and propofol, which I mentioned before, seems like a lot. We need to change the way we think about the natural course of 

 infection [CDI]. Hopefully, people will and explore all treatment options that are available at this point.

As far as education with the practitioners, it is about educating the patients as well. If they happen to be frequent fliers for, let’s say, as you mentioned, UTIs [urinary tract infections]. Or they go to their physician, and they say, “I have a cold,” and they’re given an antibiotic. You have to explain to patients as you’re seeing them come into your practice that it’s not important to get an antibiotic every time you go to your physician’s office. And to educate them and not to try to doctor-shop and get an antibiotic if they don’t get the right answer from their primary care physician [PCP] or whomever. It’s important not only for practitioners, but for patients or potential patients for 

 Certainly, we’re going to continue to need patient education, and I appreciate that comment. Andrew?

I am cautiously optimistic now, at this point, as to where we’re headed with CDI treatment, as we have the first FDA-approved FMT product at this point right now with seemingly more on the way. We’ve got the first one through the door, and it’s going to be a matter of seeing where things go from here. As you mentioned earlier, maybe hopefully in the next 10 years we’ll have narrowed it down to which key microorganisms are essential for restoring the microbiota. And if we see these improvements, then hopefully the treatments will continue to become more effective into the future as well.

Appreciate that. I have to say that I’ve considered myself in the past a drug pusher, if you will, in the good sense of the term. But I am excited the fact that there is now an FDA-approved fecal microbiota replacement and that’s a nice, good step forward with assurance that it’s screened and safe, for the most part. And that’s a major step for managing patients with recurrent 

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Experts provide practice pearls for educating patients on and using FMTs for the management of CDI.

Key Takeaways on FMTs in the Treatment of CDI

Joseph Reilly, BS, PharmD, BCGP

Celiac disease is a gluten-sensitive enteropathy, with a global pooled prevalence of 1.4%, which is increasing. Injury to the small bowel results in loss of absorptive surface area that translates into malabsorption of fat-soluble vitamins, iron, folic acid, and vitamin B12, which result in gastrointestinal and systemic symptoms. Here, we will review the updated recommendations of the American College of Gastroenterology (ACG) guidelines on the evaluation and management of celiac disease.

Patients with symptoms and signs of malabsorption, including chronic diarrhea with steatorrhea, weight loss, abdominal pain, and bloating, should be considered for celiac disease testing. The new guidelines suggest that those with a first-degree family member with confirmed diagnosed of celiac disease should also be considered for testing, regardless of whether they have symptoms. Screening in the general population is not recommended.

A combination of positive celiac serologies and small-bowel biopsies showing villous abnormalities are required to make the diagnosis of celiac disease. The sensitivity and specificity of tissue transglutaminase IgA (TTG-IgA) for untreated celiac disease is 95%, making it the serologic testing of choice. Because the prevalence of IgA deficiency in patients with celiac disease is as high as 2 - 3%, it is advisable to measure total IgA levels at the beginning of testing. If the levels of IgA are insufficient, an IgG-based test like deamidated gliadin peptide IgG or TTG-IgG would be the preferred serologic testing. For diagnosis, it is important to confirm that patients are incorporating gluten in their diet, as antibody-based testing is grounded on the intake of gluten.

For patients with positive serologies, EGD with duodenal biopsies is required to confirm the diagnosis.Those with a high clinical probability of celiac disease (>5%) and negative serology should undergo upper endoscopy with duodenal biopsies due to the possibility of seronegative celiac disease and for evaluation of other enteropathies. Obtaining 1 or 2 biopsies from the duodenal bulb and at least 4 biopsies from the post-bulbar duodenum is important for an adequate diagnosis as celiac disease can be patchy. The diagnosis is confirmed based on the presence of villous blunting, intraepithelial lymphocytosis, and crypt hyperplasia per the Marsh or simplified Corazza classification. Intraepithelial lymphocytosis (≥25/100 epithelial cells) alone is not specific for celiac disease, hence alternative diagnoses should be considered.

In cases of serology and histology discordance, testing for high-risk HLA haplotypes of celiac disease is helpful to clarify the diagnosis. If HLA-DQ2 and HLA-DQ8 are negative, the diagnosis of celiac disease can be safely ruled out. HLA-DQ2 and HLA-DQ8 also play a role when patients have already started a gluten-free diet on presentation. In those who are positive, a gluten challenge can be pursued.

In the case of children, a combination of elevated TTG IgA and a positive endomysial antibody (EMA) in a second blood sample is recommended to confirm the diagnosis. This approach is also reasonable for adult patients foregoing EGD, although the diagnosis would be deemed possible, instead of confirmed. For children younger than 2 years, the guidelines recommend a combination of TTG-IgA and deaminated gluten peptide (DGP)-IgG, as EMA IgA is less accurate in this population.

The only effective therapy for celiac disease is gluten-free diet (GFD). Due to the importance of adherence to a gluten-free diet, consultation with a dietitian is recommended after diagnosis. About 80% of individuals with celiac disease who are on a GFD will have a negative serology after 6 to 12 months. Although clinical improvement and normalization of antibodies suggest adherence to GFD, intestinal mucosal healing should be one of the end-point goals of therapy. As healing can take time, particularly in adults, the guidelines suggest a follow-up endoscopy with biopsy after 2 years of following a GFD. Lack of mucosal healing correlates with lymphoproliferative malignancy, bone disease, and refractory celiac disease. In the case of asymptomatic children following a GFD, follow-up biopsies are not recommended.

Although multiple tools to detect gluten in food have come to the market recently, data is lacking about their impact on clinical outcomes and adherence to GFD. Hence, the guidelines suggest against gluten detection devices.

Although dysbiosis is present in patients with celiac disease, there is not enough data to support the use of probiotics. Although oat consumption is safe in most patients with celiac disease, some might react to the oat protein itself, and there is risk of contamination with wheat. Hence, the guidelines suggest consuming gluten-free oats.

Patients with celiac disease are at higher risk of pneumococcal infections compared with the general population, which is due to functional hyposplenism. Hence, the ACG guidelines recommend vaccination against pneumococcus after diagnosis as part of the preventative care of patients with celiac disease. For adults without previous vaccination, the ACG suggests 1 dose of PCV20 (pneumococcal conjugated vaccine 20) alone or 1 dose of PCV15, followed by 1 dose of PPSV23 (pneumococcal polysaccharide vaccine 23).

Measurement of bone density with dual-energy X-ray absorptiometry should also be considered.

The updated guidelines provide new recommendations about considering testing asymptomatic first-degree relatives of confirmed individuals with celiac disease, correlation between serologic and endoscopic diagnosis in children and adults, the role of mucosal healing, the lack of evidence of tools to detect gluten, and probiotics. It also highlights the importance of preferring gluten-free oats, and of preventative immunization and measurement of bone density. As research evolves in this field that affects an increasing number of individuals, more data will be available to facilitate their diagnosis, adherence to gluten-free diet, and preventative care.

Rubio-Tapia, Alberto MD1; Hill, Ivor D. MD2; Semrad, Carol MD3; Kelly, Ciarán P. MD4; Greer, Katarina B. MD, MS5; Limketkai, Berkeley N. MD, PhD, FACG6; Lebwohl, Benjamin MD, MS7. American College of Gastroenterology Guidelines Update: Diagnosis and Management of Celiac Disease. The American Journal of Gastroenterology 118(1):p 59-76, January 2023. | DOI: 10.14309/ajg.0000000000002075

An expert reviews the updated recommendations of the American College of Gastroenterology guidelines on the evaluation and management of celiac disease.

Celiac Disease ACG Guidelines Update

Lady Katherine Mejía Pérez, MD

Patient Case No. 2

Patient No. 2 has stage 3 CKD and uncontrolled hypertension. They have a moderate reduction in their estimated glomerular filtration rate, confirming stage 3 CKD. Their hypertension is likely contributing to CKD progression.

Difficult-to-treat hypertension is a condition where blood pressure remains uncontrolled despite the use of three or more antihypertensive medications, including a diuretic. This condition is distinct from resistant hypertension, which is more common and involves blood pressure that is not controlled with three medications but may not include a diuretic. Difficult-to-treat hypertension is less frequent, affecting a smaller subset of patients with high blood pressure. It often requires a more comprehensive approach, including lifestyle modifications and potential secondary causes evaluation, to achieve optimal management.

Panelists discuss how chronic kidney disease (CKD) and uncontrolled hypertension create a damaging cycle, in which hypertension accelerates kidney damage while declining kidney function makes blood pressure harder to control.

Manesh R. Patel, MD

Niels Jongs, PhD, is a statistician at the University of Groningen.

Niels Jongs, PhD

Troum discussed the AGILE study and how DECT scanning helped visualize improvements in uncontrolled gout.

Orrin Troum, MD: Accurately Imaging Gout With DECT Scanning

 of pegloticase (Krystexxa; Amgen) may reduce the treatment burden for patients with uncontrolled gout in new research which found 1-hour infusions to be well-tolerated.

Orrin Troum, MD, clinical professor of medicine, Keck School of Medicine, University of Southern California, and director, clinical rheumatology research, Providence St John's Hospital, Santa Monica, California, presented data from the phase 4 AGILE study evaluating shortened infusions at last month’s 

can College of Rheumatology (ACR) Convergence 2024

 spoke with Troum to learn more about gout management and how shortened infusion times can help reduce patients’ treatment burdens. He also discussed autoimmune investigations he is involved in at Providence St John's, including indications such as lupus, Sjogren disease, and psoriatic arthritis.

Troum emphasized the value of dual-energy computed tomography (DECT) scans in managing gout and the importance of expanding the access of use of DECT scans, which are not available for rheumatologists in all areas.

“By DECT scanning, you can get a very clear picture with over 90% accuracy with being specific and sensitive to identifying urate crystals, and that has been a big help to me, because it helps us in targeting patients, specifically with tophaceous gout that we can measure… and by the end of Krystexxa therapy, they don't have anymore. So that's really a very good outcome for me that I've used… Not everybody has access to it, but [it’s been a] very useful tool for me,” Troum said.

Relevant disclosures for Troum include AbbVie/Abbott, Amgen, Bristol-Myers Squibb, Eli Lilly, Genentech, GlaxoSmithKlein, Novartis, Roche, and UCB.

Troum O, Botson J, Fang F, et al. Safety, Tolerability and Efficacy of Pegloticase Administered with a Shorter Infusion Duration in Subjects with Uncontrolled Gout Receiving Methotrexate: Primary Findings of the AGILE Open-label Trial. Presented at: Presented at: ACR Convergence 2024; November 14-19; Washington, DC. Abstract 2012.

Orrin M. Troum, MD

 What else is out there? We have hydroxyurea. We have voxelotor, crizanlizumab, Endari, transfusions. What’s next? There’s a plethora of treatment options to treat the variety of patients who have who have sickle cell disease. We’re stuck with 4 or 5 options. What else is in the pipeline to treat sickle cell disease?

 If we had this conversation 5 years ago, it wouldn’t be much of a conversation. It’s a good time to be in sickle cell, and the future is bright. Gene therapy, despite the issues, is there. There are many ways to do that. As someone had mentioned, science will prevail with time. The curative options are there, but it’s important to recognize that they’re not going to be there tomorrow, and they’re not going to be there next year for the masses.

There are a number of other therapies out there. Fetal hemoglobin is out there. Fetal hemoglobin is a good target. We know about BCL11A. There are groups trying to make fetal hemoglobin higher using a medication that is in development, and I’m sure there are many ways that’s being tried. There’s a class of medications, called PKR activators, that were designed for pyruvate kinase deficiency. It’s is a very small population, but they can be repurposed to reduce hemolysis in sickle cell disease in ways not too unsimilar to voxelotor. Crizanlizumab is medicine that targets P-selectin.

Several other medications in that class are in development or are being repurposed to see if they can address pain. Because even though we’re trying to address the root cause, when a patient comes with pain, what can you do to abort that pain? We have nothing, except for opiates, which are not the solution. It’s a tough battle, but there are efforts trying to do that with P-selectin and all selectins, and many ways with inflammation. But there are many classes. This is a bigger conversation than it could have been even a couple of years ago. It’s an exciting time, and we’ll get to a point where sickle cell disease treatment is a cocktail approach. You take a couple of things, and as long as we can work on it, if we can address things from different angles, then we’ll make our patients have better lives.

Absolutely. Well, thank you so much. This has been an amazing conversation. I want to thank Abdullah, Wally, and Pat. Before we close, tell the audience your 1 big dream, or 1 final thought, that you want everybody who sees or hears or takes care of patients with sickle cell disease to know as a takeaway message from this conversation. I’m going to start with Abdullah. Your 1 final word that you want them to know about?

As you said, this has been a really exciting conversation. Wally, you, and Pat contributed greatly. Considering this discussion, there are 2 things. It’s important, scientifically, to pursue curative options like gene therapy. I’ll repeat that science will prevail. We will have to overcome the risks, understand them better, and remedy them. That’s 1 thing. 

Coming back to Wally’s point, we need more disease-modifying agents that we can use in Africa or India. The typical approach would be—I agree with Pat—new F-inducing agents or a combination of F-inducing agents. Maybe a combination of F-induction with some downstream modifications like anti-inflammatory or antiadhesion therapies. These are going to be important, and I believe these have to be pursued in sickle cell, along with these high-tech, higher-source modalities. This includes swallowing a pill, which can easily be conveyed to Africa or low-resource countries.

Thank you so much, Abdullah. I’m going to follow up with Wally. Wally, what are your 1 or 2 big-picture, take-home thoughts?

 I’d like to put my public health hat on. I want our audience to know about a report that came out in November from the National Academies of Sciences, Engineering, and Medicine. Ify, you and your colleagues did wonderful preparing 512 pages about all that is wrong in sickle cell care in the United States and what needs to be done to make sickle cell care better. There were 7 pillars that you pointed out, and they had to do with education and social policy. They had to do with patient empowerment and disparities. You declared sickle cell to be a disparities disease, as Pat said earlier. They had to do with therapy as well. But it wasn’t just therapy that you focused on. You focused on social determinates of health, you focused on policy, racism, and changes in the fabric of the health care system, especially for adults with sickle cell. We don’t even have an adult sickle cell disease health care system. We have a shack instead of a home for adults with sickle cell disease. That’s the 1 thing I want to take away. None of this discussion we just had will matter is there’s nobody to care for the patients. And in many parts of the country, there’s nobody—doctor, nurse, or other—to care for these patients.

Very well said. Thank you so much, Wally. 

I’m going to add to that because it’s so important. The names of the medicines don’t matter. Whether you live in Cincinnati, or Angola, or India, if we have good therapies, they should be available. We need to leverage that equity to global treatment of sickle cell disease. Hydroxyurea was developed in the 1980s and ’90s and is only now becoming available in Africa. Not really, even. I don’t want to give a talk when I’m retiring in a few decades telling patients about all these new therapies that we’re using in the United States and how sickle cell disease is history, except in Africa, where 90% of kids are still dying before they’re adults. That can’t happen. As these therapies are developed, by Bill Gates or whomever, we’re going to need a lot of people. We need to be sure that these are truly equitable in a timely manner, or we’re doing a huge disservice to the global sickle cell community.

Thank you so much. I’m going to give my thoughts. The first thing I want the audience to recognize is that sickle cell disease is a lifetime genetic disorder. It doesn’t go away when you become a teenager or when you leave pediatrics. It doesn’t disappear when you get married. It’s there for the rest of your life. It does shorten your life span. 

The second thing is pain with sickle cell disease. It’s important, but it’s not the only important thing. Organ damage, that chronic, slow slump of devastation to the organs is a reality, and no patient will die because of pain. They will die from strokes, kidney disease, pulmonary hypertension, infection, heart problems, lung problems. It’s important to look at them as treating not just pain but the entire person. That entire person will also involve their quality of life. Ask them how they’re doing, how are they functioning. Do they get up and go to work? If not, why not? When you’re giving treatment, look at the impact of treatment. Not just on their pain, as we talked about, but on their overall quality of life. 

We talked about screening for sickle cell disease at birth, screening for complications following the guidelines, and early intervention. For example, retinopathy picked up early can prevent blindness, treating hyposthenuria early prevents ESRD [end-stage renal disease], and picking up pulmonary hypertension can prolong life by putting the right treatment in place. But there’s also supportive care. And let’s not forget the role of the primary care provider. The last time I checked, having sickle cell disease does not protect you from getting breast cancer, colon cancer, high blood pressure, diabetes, or diverticulitis, or any other thing that anybody else gets. My patients have all this, and they deserve whole care—not sickle cell care but all-around holistic medical care.

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Experts look into what is next for sickle cell disease and provide key thoughts on the space. 

What’s Next and Final Thoughts in SCD 

Patrick McGann, MD, MS, Cincinnati Children’s Hospital  

Previewing Revolutionizing Atopic Dermatitis 2025, with Raj Chovatiya, MD

With dermatology evolving faster than ever, hyperfocused meetings such as the 

Revolutionizing Atopic Dermatitis (RAD) Annual Meeting

 remain a vital hub for education, collaboration, and advancing patient care.

In recent years, atopic dermatitis has gained major attention, with rapid growth in treatment options. But turning these options into practical solutions for real patients has made provider education more critical than ever.

"Every year we get really great abstracts at RAD that are debuting for the first time for late breaking researchers that relate to atopic dermatitis," said meeting cochair Raj Chovatiya, MD, PhD, a clinical associate professor of medicine at Rosalind Franklin University Chicago Medical School and founder and director of the Center for Medical Dermatology and Immunology Research in Chicago, in an interview. "This year will be no exception, and there's going to be a lot of really cool stuff being unveiled."

At the 2024 RAD meeting, the community explored findings from the LEVEL UP data, the Measure Up program, and the DELTA studies. Along with trial updates and guideline discussions, the meeting also puts focus on real-life clinical topics—like treating skin of color, managing comorbidities, and more.

In this interview, Chovatiya—who is serving as the cochair for this year's RAD annual meeting, happening June 06–07, 2025, in Nashville, TN—shares why RAD has become a must-attend for anyone managing atopic dermatitis. He explains how the sessions, speakers, and discussions stand out for their focus on day-to-day challenges, patient impact, and improving care delivery.

Campbell P. MJH Life Sciences Celebrates Acquisition of Revolutionizing Atopic Dermatitis (RAD) Annual Meeting. HCP Live. Published November 11, 2024. Accessed April 9, 2025. 

https://www.hcplive.com/view/mjh-life-sciences-celebrates-acquisition-of-revolutionizing-atopic-dermatitis-rad-annual-meeting

Revolutionizing Atopic Dermatitis 2025 Annual Meeting cochair Raj Chovatiya, MD, PhD, previews the upcoming meeting. 

Previewing Revolutionizing Atopic Dermatitis 2025, with Raj Chovatiya, MD, PhD

Raj Chovatiya, MD, PhD

Final Insights

Video content above is prompted by the following:

Are there any final insights you’d like to share regarding LDL-C and Lp(a) screening and management of high-risk cardiovascular patients?

Pediatric lipid screening is crucial for early detection and management of dyslipidemia, which can prevent cardiovascular diseases later in life. Current guidelines recommend universal lipid screening for children between ages 9-11 and 17-21, with selective screening for those with risk factors. However, implementation remains inconsistent due to barriers such as lack of awareness and time constraints in clinical practice. To improve uptake, strategies include integrating screening into routine pediatric visits, utilizing electronic health records for reminders, and educating healthcare providers and families about the importance and benefits of early lipid screening.

Panelists discuss how effective cardiovascular risk management requires comprehensive screening for both LDL-C and Lp(a), along with aggressive treatment strategies for high-risk patients.

Authors