Ruchika Goel, MD, is a professor and associate vice chair of research at SIU School of Medicine.

Defining the goal of platelet transfusion, prevention or treatment, may be as important as the platelet count itself.

in neonates and older children, reduced platelet increment (PI) did not lead to worse clinical outcomes, suggesting that this marker does not precipitate an increased, automatic need for transfusion.

, professor and associate vice chair of research at SIU School of Medicine, to glean key clinical takeaways from the findings.

How should clinicians and Transfusion services think about balancing transfusion safety measures with platelet effectiveness and pediatric care?

That’s an excellent question, and I want to emphasize it overall by rechecking: what is our goal? Number one, is it prevention of bleeding in an otherwise stable patient, or in an actively bleeding patient, or a high-risk patient? Is it hemostasis? Are you trying to do bleeding control?

So reassessing what our goal is and not mixing the two is very important. Looking for any other non–product-related causes of poor increment is also important. For example, any reason for high platelet consumption, infection, inflammation, DIC, splenic sequestration, medications, or any other reasons. For example, a patient being on ECMO—these are other reasons where you could have poor increment causes, and they need to be reassessed.

If there are further transfusions, consider looking at the overall clinical situation and assessing the requirement. That should be the decision-making criteria, not simply a suboptimal increment in a platelet count.

Do your findings suggest that we need to rethink what defines successful platelet transfusion in children beyond simply achieving a target platelet count?

Yes, I think this study emphasizes that, as well as the accompanying editorial by Dr Megan Delaney. They really push toward redefining what we call a successful platelet transfusion, really being beyond hitting a target platelet count, a more clinically meaningful definition of what we are trying to achieve.

For example, what I would say a successful platelet transfusion would be is something that achieves the intended clinical objective, whether it’s prophylaxis of bleeding or attempted bleeding control, while we have minimal transfusion burden. So optimizing our overall transfusion burden and making sure we have no or minimal transfusion-related harm while our overall clinical objective is met, and not using a specific number or an increment as our goal of a successful platelet transfusion.

So in practice, that would mean understanding our endpoints, such as hemostatic readiness, bleeding events, transfusion reactions and complications, and the overall transfusion burden. These are the factors that need to be taken into account as what we call a successful platelet transfusion.

What are the limitations to this study and how should clinicians interpret them?

The only thing I’d like to add is that readers should go in detail with the study limitations as well. This is a very large, very unique database, and there are very few such powerful databases that can assess the donor as well as the product and ultimately link it to clinical outcomes in the recipient. So there are several strengths, but there are also several limitations.

Ultimately, it is an electronic health record–driven result. We have highlighted several limitations of the study, and we do want to emphasize that there is a need for more prospective studies and perhaps a need for continued randomized controlled trials to study several clinical outcomes.

So while we address several strengths of the study, I want to redirect the reader to look at the limitations of the study and incorporate those into what they can do in their routine clinical practice, which should be evidence-based.

Goel R, Karam O, Warden DE, et al. Platelet Transfusion Practices and Outcomes in Neonates and Children. 

https://doi.org/10.1001/jamanetworkopen.2025.54531

Goel R. When Platelet Increments Matter, With Ruchika Goel, MD. Hcplive.com. Published February 13, 2026. Accessed February 14, 2026. 

https://www.hcplive.com/view/when-platelet-increments-matter-with-ruchika-goel-md

Articles

In pediatric platelet transfusions, what counts as success depends on the clinical context and treatment goals, not just the platelet count.

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Hematology

Rethinking What Defines Success in Pediatric Platelet Transfusion, With Ruchika Goel, MD

Dawn Eichenfield, MD, PhD, is a dermatologist at Rady Children's Hospital-San Diego and an assistant clinical professor of dermatology at UC San Diego School of Medicine.

, Dawn Eichenfield, MD, PhD, of Rady Children’s Hospital–San Diego and the University of California San Diego School of Medicine, spoke on-site with the 

 team regarding the rapidly shifting pediatric dermatology landscape, highlighting both therapeutic updates and various types of practical clinical considerations.

, Eichenfield emphasized the expanding set of potential systemic options now available for children, especially biologics for psoriasis.

 In this interview segment regarding additional sessions Eichenfield participated in, she pointed to the value of understanding a biologic’s mechanism of action, ongoing questions around disease modification in atopic dermatitis, and various practical pearls, including strategies to reduce injection-related anxiety and discomfort among children.

The following transcription summarizes Eichenfield’s on-site interview with 

 We would like to know more about some of the biggest takeaways from your Maui Derm workshop, the master class in pediatric dermatology.

Today, we did a workshop where we got to talk a lot more about taking care of kids. I actually talked about hair disorders, and based on looking at kids with telogen effluvium as well as alopecia areata. Actually, rarely, we sometimes get scarring disorders in pediatric dermatology as well. Then it was really nice, because we had a round table discussion with the other folks who were talking during the pediatric master class workshop. We got a chance to kind of see how other people approach really bad cases of androgenetic alopecia or alopecia areata, and how they dose their minoxidil. What do they do? First, topical minoxidil, then oral minoxidil. So it was a really, it was a really great, intriguing discussion that we had.

Would you go a bit more into depth on the dosing of minoxidil that you discussed there?

You look at the pediatric literature…there was actually a recent PD study where they looked at it, and it was an international study looking at androgenetic areata. It was really interesting because in that study, most people were on topical minoxidil. Nowadays, we're starting to use a lot more oral minoxidil. So I would say, if you looked at things like five, ten years ago, people were probably just doing topical minoxidil. But now, [we use oral minoxidil more], just because we feel like low-dose oral minoxidil is a lot safer and we have a lot more evidence for its use.

Also, we're definitely using it more in our pediatric and adolescent populations. So generally, for a girl, we probably would start with a quarter of a tablet. A tablet is like 2.5 milligrams. So a quarter is like 0.625. Then for males, we would start probably with 1.25, and of course, this is in kids who don't have other medical issues. No heart issues, no issues with their blood pressure, not on other concurrent medications that would interfere or have other side effects that are related to the minoxidil.

I think the big [takeaway] is really just the fact that we sometimes underdiagnose pediatric androgenetic alopecia, and sometimes we can misdiagnose some of our pediatric hair disorders. It's also very important to come to the correct diagnosis so that we can facilitate the correct management.

What are some of the key takeaways about HSV that you hope clinicians walk away with?

 I think the case that I presented is a very interesting case in that the clinical presentation is of an immunocompromised patient. It's always when you're looking at blisters, hemorrhagic blisters, hemorrhagic crusting, or ones that look punched out when you definitely want to consider HSV. Sometimes it could be something you're not even thinking of, and it can mimic some other diseases as well. So, that's definitely something to think about for the measles case. I think in our in this day and age, when some parents are not sure about vaccinations, and when it's kind of political and controversial, I think it's important for healthcare providers to know what measles looks like, and to really consider that in their differential where they're approaching pediatric patients with rashes.

Then, finally, we covered Leishmania. It's really interesting because I live in San Diego, and we definitely get a lot of patients who kind of come across the border. We've had a number of refugees who come through, who've actually had more kinds of tropical or exotic diseases. Leishmania is one of them, because they kind of came through the Darién Gap, while we were getting some refugees from that area.

Moving to a broader subject, what are you most excited or intrigued about in the field of dermatology in 2026 and beyond?

I think there's a lot that's in development, both in science and in clinical medicine, that we're going to be excited about for pediatric dermatology. Anything genetic-related is always exciting, because we always want to be able to better diagnose our patients before we start treatments. Then finally, as I had mentioned before, our patients don't like shots. So, some of the oral biologics coming out that will be very exciting to us, there are companies that are working on looking at biologics where you're going to be able to space out how often you get the shots. That, of course, will be very interesting to our patients, because no one likes shots. I think we have so much to look forward to this year and the coming years in dermatology.

The quotes used in this interview transcript were edited for clarity.

Eichenfield had no relevant disclosures of note for this discussion.

Eichenfield D, Frieden I, Treat J, et al. Fallon Friedlander S, Treat J, et al. Master Classes in Pediatric Dermatology. Presented at the 2026 Maui Derm Hawaii Conference, January 25-29.

Eichenfield D. Spotlighting Pediatric Dermatology Updates for 2026, With Dawn Eichenfield, MD, PhD. HCPLive. January 29, 2026. Accessed February 13, 2026. 

https://www.hcplive.com/view/spotlighting-pediatric-dermatology-updates-2026-dawn-eichenfield-md-phd

Eichenfield speaks in this interview about several key takeaways drawn from her master class in pediatric dermatology at Maui Derm Hawaii.

Plaque Psoriasis

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Conference

 conference coverage page features articles, videos, and expert-led live coverage from major medical meetings throughout the year.

New Pediatric Atopic Dermatitis Updates for 2026, With Dawn Eichenfield, MD, PhD

Abigail Brooks is an assistant managing editor for 

She joined MJH Life Sciences in August 2023 shortly after graduating from Monmouth University where she earned her BA in Communication with a concentration in Public Relations/Journalism and later an MA in Interactive Digital Media. She enjoys traveling, running, and reading books. She can be reached at abrooks@mjhlifesciences.com.

Chronic kidney disease (CKD) affects an estimated 35 million people in the United States, yet it remains persistently underdiagnosed, underrecognized, and often addressed late in its course.

Despite a wave of therapeutic innovation over the past decade, including SGLT2 inhibitors, non-steroidal mineralocorticoid receptor antagonists, and GLP-1 receptor agonists, most patients continue to enter care at stage 3b or later, when significant and often irreversible kidney damage has already occurred. Against this backdrop, a recent multidisciplinary virtual forum from 

, led by Muthiah Vaduganathan, MD, MPH, codirector of the Center for Cardiometabolic Implementation Science at Brigham and Women’s Hospital, convened nephrologists from academic and private practice settings across the country to examine a central question: how do we move CKD detection and management upstream?

The discussion centered on operationalizing early identification of CKD using routine biomarkers such as estimated glomerular filtration rate (eGFR) and urine albumin-to-creatinine ratio (UACR), and on improving risk stratification before patients ever see a nephrologist. Participants highlighted national data showing persistent gaps in CKD awareness, particularly among younger individuals, women, and racial and ethnic minority populations.

Even among patients with documented CKD in the medical record, awareness remains low. Compounding the problem is a limited nephrology workforce, making universal early referral neither feasible nor necessary.

Instead, the conversation emphasized redefining early CKD as a cardiovascular risk condition and embedding screening within broader cardio-kidney-metabolic care. With cardiovascular disease remaining the leading cause of death in CKD, panelists argued that early kidney abnormalities, especially albuminuria, should be framed not simply as markers of future dialysis risk, but as powerful indicators of systemic vascular injury.

In an era of disease-modifying therapies capable of slowing progression and reducing cardiovascular events, the forum explored how clinicians, health systems, and payers might rethink detection strategies, referral pathways, and multidisciplinary collaboration.

Throughout the discussion, participants repeatedly returned to the reality that most referrals still occur when eGFR falls < 45 mL/min/1.73 m², while earlier-stage patients, particularly those with preserved eGFR but significant albuminuria, are often missed. UACR testing remains underutilized outside of diabetes care, and confusion persists among primary care clinicians regarding the interpretation of albuminuria versus total proteinuria.

Several panelists described educational initiatives aimed at improving primary care understanding, including reframing albuminuria as the “troponin of the kidney,” yet acknowledged that education alone may not be sufficient. Workforce shortages, time constraints in 20-minute visits, EHR fatigue, and limited health literacy all create structural barriers to earlier detection and intervention.

Ultimately, the forum highlighted a need for system-level change. Potential solutions discussed included risk-based referral models rather than rigid eGFR thresholds, automated EHR or AI-driven lab triggers, payer-aligned quality metrics tied to UACR screening, and community-based outreach in high-risk populations.

The broader shift toward a cardio-kidney-metabolic framework was seen as an opportunity to align specialties around shared therapies and shared risk reduction goals. However, panelists were candid: without stronger incentives, clearer risk stratification tools, and expanded workforce capacity, the promise of early CKD “remission” through modern therapies may remain unrealized.

US Centers for Disease Control and Prevention. Chronic Kidney Disease in the United States, 2023. May 15, 2024. Accessed February 11, 2026.

Experts discuss shifting CKD detection upstream, improving albuminuria screening, and enabling earlier, risk-based intervention.

Nephrology

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Rethinking Early Detection of CKD: A Shift Toward Upstream Care

Alex Hillenbrand is an assistant editor for 

. She earned her BA in English from Lafayette College in 2023 and went on to write for pop culture, entertainment, and sports media outlets before joining MJH Life Sciences in 2025. In her free time, Alex enjoys cooking, going on walks, and meeting new people. Contact her at mjhlifesciences.com.

 issued a Complete Response Letter for the New Drug Application (NDA) of Disc Medicine’s bitopertin as a treatment for patients with 

The Agency acknowledges sufficient evidence of reduced whole blood metal-free protoporphyrin IX (PPIX) from the AURORA and BEACON trials, but stated a need to see results of the ongoing phase 3 APOLLO study before bitopertin receives approval.

EPP is a rare, inherited metabolic disorder marked by severe, immediate, and painful photosensitivity, often beginning in childhood. In this patient population, a deficiency in the ferrochelatase enzyme results in protoporphyrin buildup, which is indicated by intense burning, itching, and swelling upon exposure to sunlight.

Bitopertin is an investigational, clinical-stage, orally administered glycine transporter 1 (GlyT1) inhibitor, designed to modulate heme biosynthesis. GlyT1 is a membrane transporter required to supply sufficient glycine for heme biosynthesis and support erythropoiesis. If approved for EPP, bitopertrin has the potential to be the first disease-modifying therapy.

“We are committed to delivering bitopertin to patients, knowing how critical this potentially disease-modifying therapy is to the EPP community. While our efforts at utilizing expedited pathways to get bitopertin to patients quickly have not come to fruition, we are continuing to pursue all avenues in support of FDA approval,” said John Quisel, JD, PhD, President and Chief Executive Officer of Disc Medicine, in a statement. “The CRL will delay the potential approval of bitopertin, but we have confidence in the ongoing APOLLO trial, for which we are seeing incredible enthusiasm from the EPP community. Confidence in our product and program guides our approach, and we will continue working closely with the FDA to support their review.”

Bitopertis has been under review for accelerated approval and as part of the Commissioner’s National Priority Voucher pilot program, with accelerated approval contingent on the proposed surrogate endpoint, percent change in whole blood metal-free PPIX, and whether this was reasonably likely to predict a clinical benefit.

While the FDA agreed that phase 2 double-blind, placebo-controlled AURORA and phase 2 open-label BEACON met the proposed surrogate endpoint, neither showed evidence of an association between percent change in PPIX and sunlight exposure-based endpoints. In turn, the Agency indicated the phase 3 APOLLO results would be needed as evidence for traditional approval.

APOLLO is a randomized, double-blind, placebo-controlled study evaluating the efficacy, safety, and tolerability of bitopertin in participants with EPP or X-Linked protoporphyria (XLP). Currently, the estimated enrollment size includes 150 participants ≥ 12 years of age.

Specifically, APOLLO investigators are assessing whether bitopertin increases pain-free sunlight exposure after 6 months of treatment and how PPIX concentration levels change from baseline.

The trial began enrollment in March 2025, and has an estimated completion timeline of October 2026.

Disc Medicine Inc. Disc Medicine Receives Complete Response Letter from FDA for Bitopertin for the Treatment of EPP. GlobeNewswire News Room. Published February 13, 2026. Accessed February 13, 2026. 

https://www.globenewswire.com/news-release/2026/02/13/3238299/0/en/Disc-Medicine-Receives-Complete-Response-Letter-from-FDA-for-Bitopertin-for-the-Treatment-of-EPP.html

Clinicaltrials.gov. Published 2026. Accessed February 13, 2026. 

https://clinicaltrials.gov/study/NCT06910358

The FDA denied accelerated approval for bitopertin, citing a need for evidence on clinical benefit from the ongoing phase 3 APOLLO trial. 


RDR Alert®

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FDA News

FDA Issues Complete Response Letter to Bitopertin For Erythropoietic Protoporphyria

Diana Isaacs, PharmD, is an endocrine clinical pharmacist, director of Education and Training in Diabetes Technology, and codirector of Endocrine Disorders in Pregnancy at the Cleveland Clinic.

Diabetes Dialogue: Technology, Therapeutics, & Real-World Perspectives!

Diabetes Dialogue: Oral Ozempic and the Risks of Compounding Formulas

In this episode, cohosts Diana Isaacs, PharmD, and Natalie Bellini, DNP, explore major updates in the evolving GLP-1 receptor agonist landscape, with a particular focus on oral semaglutide formulations, branding changes, and regulatory concerns surrounding compounded alternatives.

00:05:33 The importance of administration

00:06:56 Compounding drugs - the Hims and Hers drama

00:07:56 The danger of unregulated compound drugs

The discussion begins with the FDA approval of a higher-dose oral semaglutide formulation now branded as Ozempic for type 2 diabetes. Previously marketed as Rybelsus, the formulation itself remains bioequivalent, with no changes in efficacy, safety, or pharmacokinetics. The rebranding aligns oral and injectable Ozempic, simplifying clinical conversations and reducing prior authorization and coverage confusion. Similarly, oral Wegovy is now available for obesity management, reinforcing a unified naming convention: Ozempic for type 2 diabetes and Wegovy for obesity, regardless of route of administration.

The hosts note that while oral semaglutide has been available for several years, uptake of Rybelsus lagged behind expectations, in part due to limited brand recognition and confusion about its equivalence to injectable semaglutide. Early anecdotal experience suggests greater patient interest in “oral Ozempic,” highlighting the impact of naming clarity on real-world adoption. The simplified dosing structure of the newer formulations may further facilitate transitions during the upcoming rollout period.

Importantly, Isaacs and Bellini review key pharmacologic considerations. Oral semaglutide depends on the absorption enhancer SNAC (sodium N-[8-(2-hydroxybenzoyl) amino] caprylate) to protect the peptide from gastric degradation and promote systemic uptake. Administration remains strict: taken on an empty stomach with no more than 4 ounces of water, followed by a 30-minute interval before food, beverages, or other medications. Despite daily dosing, the drug retains a long half-life of approximately one week, meaning that temporary discontinuation—for example, prior to planned pregnancy—requires appropriate lead time. They emphasize that daily administration reflects absorption variability rather than a shorter pharmacologic duration.

The episode then pivots to safety concerns surrounding compounded oral semaglutide products. With GLP-1 shortages largely resolved, some telehealth companies—including Hims & Hers—have begun marketing lower-cost compounded oral versions. This has prompted legal action from Novo Nordisk, given that the proprietary SNAC technology is patented and critical to the drug’s validated bioavailability. The hosts caution that compounded oral formulations may lack regulatory oversight, standardized absorption data, and robust phase 1–4 clinical trial evidence. Without validated pharmacokinetics, patients risk receiving ineffective therapy—or potentially unsafe products—despite appealing lower price points.

The episode concludes with cautious optimism. While branding simplification and cost reductions represent meaningful progress in access and patient engagement, the hosts underscore the necessity of evidence-based prescribing and clinician-led patient education. As the GLP-1 therapeutic landscape continues to expand, maintaining safety, regulatory integrity, and pharmacologic rigor remains paramount.

Editor’s Note: Isaacs reports disclosures with Dexcom, Abbott, Lilly, Novo Nordisk, Medtronic, Insulet, and others. Bellini reports disclosures with Abbott Diabetes Care, MannKind, Povention Bio, and others.

Podcasts

Diana Isaacs, PharmD, and Natalie Bellini, DNP, discuss Ozempic’s approval in a higher-dose oral form and lingering concerns over compounded drugs.

Endocrinology

The HCPLive endocrinology page is a comprehensive resource for clinical news and insights on endocrine system conditions. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for diabetes, hormonal disease, and more.

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Diabetes Dialogue: Technology, Therapeutics, & Real-World Perspectives is a monthly podcast hosted by Diana Isaacs, PharmD, and Natalie Bellini, DNP, aimed at providing a regular roundup of the latest clinically applicable insights across diabetes and metabolic diseases, with a focus on leveraging technology to improve care.

podcasts page features a feed of medical news and interview podcast series, including DocTalk, Lungcast, Derm Discussions, and Heart Trials.

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Strategic Alliance Partnership

Sophie Stocker, BsC, PhD, is a senior hospital scientist at St Vincent’s Hospital Sydney, and a senior lecturer at The University of Sydney

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Stocker discusses findings from a pilot phase trial with clinical insight into a wearable patch for continuous drug measurement of vancomycin. 


Wearable Device Delivers 5-Minute Data on Vancomycin Levels in Pilot Trial, With Sophie Stocker, MD

Muthiah Vaduganathan, MD, MPH, is a cardiologist specializing in cardiorenal metabolism at Brigham and Women's Hospital in Boston, MA. Vaduganathan is a member of Practical Cardiology's advisory board and co-host of Don't Miss a Beat, a podcast presented by Inside Cardiology.

This Heart Failure Awareness Week, revisit recent advancements and scope out the future of heart failure care with HCPLive.

Heart Failure

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Smoothing the Road: Improving Heart Failure Awareness Among Clinicians and Patients

vice president of programs, public affairs, and research at Lung Health Foundation.

Developers emphasize plain language, explicit next steps, and clear limits to ensure the PLCOm2012-based tool supports, rather than replaces, formal lung cancer screening.


Pulmonology

Infectious Disease condition center page is a comprehensive resource for clinical news and insights on respiratory health. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for asthma, COPD, interstitial lung disease, COVID-19, and more.

Lung Cancer

Safeguards in New Lung Cancer Risk Calculator, With Jessica Moffatt, PhD

Mark Kaufmann, MD, is a clinical professor in the Department of Dermatology at the Icahn School of Medicine at Mount Sinai in New York City.

This segment of an interview from Maui Derm Hawaii, featuring Mark Kaufmann, MD, highlights key advice on AI scribe use and billing effectiveness in dermatology practices.

Discussing AI Scribes and Improving Billing Practices in Dermatology, With Mark Kaufmann, MD

Zobair Younossi, MD, MPH, is chairman of the Global NASH/MASH Council and professor and chairman of Beatty Liver and Obesity Research at Inova Health System.

Younossi emphasizes the need for NIT-based risk stratification for MASLD, citing his research supporting their widespread adoption across clinical settings.

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From Validation to Implementation: Scaling NITs in MASLD Care, With Zobair Younossi, MD, MPH

Rudolf Valenta, of the Medical University of Vienna

Valenta discusses how seasonal allergen exposure selectively boosts IgE production through plasmablast recall, without a coordinated IgG response.


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Seasonal Allergen Exposure Recalls IgE Plasmablasts in Birch Allergy, With Rudolf Valenta

Low post-transfusion platelet increments did not predict mortality or length of stay in pediatric patients.

When Platelet Increments Matter, With Ruchika Goel, MD

Jeffrey Downey, PhD, is a postdoctoral student at the Swirski laboratory at Icahn School of Medicine at Mount Sinai.

Downey discusses the recent discovery of influenza’s interaction with the interferon 1 pathway, as well as a hypothetical method for preventing it.

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Interferon 1 Responsible for Causing Cardiac Events During Influenza Infection, With Jeffrey Downey, PhD

The free, user-friendly calculator provides a 6-year lung cancer risk estimate to help patients and clinicians assess screening eligibility.


Tool Predicts a 6-Year-Risk Lung Cancer Risk in Minutes, With Jessica Moffatt, PhD

Survey findings highlight abdominal and bowel symptom control, affordability, and formulary or insurance coverage as factors driving perceptions and prescribing patterns of PERT.

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Latest News

Rethinking What Defines Success in Pediatric Platelet Transfusion, With Ruchika Goel, MD

New Pediatric Atopic Dermatitis Updates for 2026, With Dawn Eichenfield, MD, PhD

Rethinking Early Detection of CKD: A Shift Toward Upstream Care

FDA Issues Complete Response Letter to Bitopertin For Erythropoietic Protoporphyria

Diabetes Dialogue: Oral Ozempic and the Risks of Compounding Formulas