Ira Zackon, MD, is the senior medical director of Ontada Medical Office.

Higher Maintained Hemoglobin Associated With Improved Survival in MDS, With Ira Zackon, MD

Higher maintained hemoglobin levels have been strongly associated with improved overall survival in patients with 

, independent of Revised International Prognostic Scoring System (IPSS-R) risk, according to a recent retrospective cohort study.

67th American Society of Hematology (ASH) Annual Meeting and Exposition

 by Ira Zackon, MD, senior medical director of Ontada Medical Office, this study reevaluates the ideal goals of 

 management across a spectrum of hematologic diseases. Based on these data, Zackon and colleagues suggest that mild anemia may be an efficient target for medical and potentially transfusion therapies.

“Most transfusion guidelines say not to transfuse with hemoglobin unless it’s >8 or even 7,” Zackon told 

 in an exclusive interview. “We allow our patients to live with moderate or sometimes severe anemia if other therapies can’t quite get there. This study is affirming, perhaps, that we need to target a hemoglobin level >10 and maintain that for optimal outcomes.”

MDS is a heterogeneous group of disorders that can lead to bone marrow dysplasia, ineffective hematopoiesis, and the potential risk of transformation to acute myeloid leukemia. For lower-risk MDS cases, survival ranges from 2 to >10 years. Additionally, many patients with low-risk MDS exhibit anemia and transfusion dependence, which intrinsically reduces health-related quality of life and correlates with shorter survival.

Zackon and colleagues utilized electronic health record data from 2005 to 2025, including patients with MDS and moderate to severe anemia – defined as hemoglobin <10 – at baseline. Patients were then stratified into 3 hemoglobin groups based on their median value of the hemoglobin levels post index: <8, 8-10, and >10. Investigators also examined baseline characteristics, including age, sex, race, and standard categories of IPSS-R risk.

The study ultimately included 6533 patients with MDS; the median age at baseline was 78 years (interquartile range [IQR], 72-84), and 46% of patients were female. OF the 1133 participants with available IPSS-R data, 46% were classified as low/very low risk, 35.8% as intermediate risk, and 18.2% as high/very high risk.

During the study, median overall survival varied significantly by hemoglobin group. Patients with longitudinal hemoglobin >10 had a median overall survival of 54.1 months (95% CI, 51-57.9), compared to 25.2 (95% CI, 24.2-26.9) for patients with hemoglobin 8-10 and 12.7 (95% CI, 11.4-14) for those with hemoglobin <8. These differences remained across all time points up to 72 months, at which point survival was 38.2%, 16.3%, and 5.9%, respectively. After stratifying these by IPSS-R score alone, investigators found a median survival of 15.9 months (95% CI, 14-21.3) for high/very high risk patients, 25.9 (21.8-29) for intermediate risk patients, and 47.4 (41.1-53.2) for low/very low risk patients.

When stratified by serum ferritin levels, median overall survival was 32.9 months (95% CI, 31.1-34.5) for ferritin <1000 and 24.9 months (95% CI, 23.1-26.8) for ferritin ≥1000. For hemoglobin <8, median overall survival was 10.2 (95% CI, 8.8-12.2) for ferritin <1000 and 16.4 (95% CI, 14.6-19.1) for ferritin ≥1000. For hemoglobin 8-10, median overall survival was 27.7 (95% CI, 25.7-29.4) for ferritin <1000 and 28.4 (95% CI, 25.6-30.5) for ferritin ≥1000.

Ultimately, Zackon and colleagues determined that patients with MDS who exhibited maintained hemoglobin levels >10 g/dL also displayed improved overall survival, irrespective of IPSS-R risk stratification. Additionally, stratification by serum ferritin indicated that inferior overall survival in both moderate and severe maintained anemia was independent of possible iron overload, spotlighting the potential benefits of maintaining mild anemia as a goal of medical and transfusion therapies with optimal iron chelation.

“MDS is not rare in the general patient population, especially as we get older,” Zackon said. “We’ve had limited therapy successes over time, and it’s taken time to build that up. I think we’re looking forward to more impactful therapies on the anemia side. The more that we can improve anemia, which is a primary aspect of the disease, the more opportunity we have to improve not only the longevity, but the quality of our patients’ lives.”

Editor’s Note: Zackon reports disclosures with Ontada and McKesson.

Su Z, Whitesell M, Zackon I, et al. Maintaining higher hemoglobin levels is associated with improved overall survival in myelodysplastic syndromes (MDS): A real-world retrospective analysis of electronic health records from US community settings over the past 20 years. Abstract presented at the 67th American Society of Hematology Annual Meeting. Orlando, FL. December 6-9, 2025.

Kim N, Pulte ED, Ehrlich LA, et al. US Food and Drug Administration Approval Summary: Imetelstat for Selected Patients With Low- to Intermediate-1 Risk Myelodysplastic Syndromes With Transfusion-Dependent Anemia. J Clin Oncol. 2025;43(35):3760-3768. 

Videos

Zackon discusses a retrospective 20-year analysis, which implies an easier-to-achieve hemoglobin target in myelodysplastic syndrome treatment.

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Anemia

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Chelsie Derman is an associate editor for 

who covers psychiatry, sleep medicine, geriatrics, migraine, and allergy. She joined MJH Life Sciences in September 2023 after graduating from The College of New Jersey with a bachelor’s degree in journalism and professional writing. In her free time, she enjoys creative writing and reading.

 had adequate knowledge of their condition, but effective self-management relied on stronger patient attitudes, especially in asymptomatic individuals.

In this cross-sectional study conducted between July and August 2023, investigators sought to assess patients’ knowledge, attitudes, and practices regarding hyperuricemia and to examine the mediating role of attitude in different patient subgroups. The study analyzed questionnaires from 406 respondents, with most male (90.39%; n = 367).

The study showed patients with hyperuricemia had a high commitment to following medical recommendations, including adhering to medications, modifying diet, avoiding high-purine/high-fat foods, avoiding alcohol, hydrating, regularly exercising, and monitoring acid levels. However, fewer patients underwent regular CT scans or participated in peer support activities.

“These findings suggest a strong willingness among patients to actively participate in their own healthcare, which is a positive aspect for clinical practice,” wrote study investigator Hong Shi, from Shanxi Bethune Hospital in China, and colleagues. “To further enhance patient care, healthcare providers can build upon this motivation by emphasizing the importance of regular medical imaging and encouraging participation in group-based support.”

Knowledge, attitude, and practice scores were 54.34 ± 8.68 (possible range: 13—65), 23.40 ± 2.36 (possible range: 6—30), and 40.31 ± 4.92 (possible range: 10—50), respectively. Structural equation modeling revealed a significant direct effect of knowledge on attitude (P =.009) and a direct effect of attitude on practice (P =.015). Additionally, the analysis showed that knowledge had a significant indirect effect on practice mediated through attitude (P =.003). The study did not find a significant direct relationship between knowledge and practice (P = 0.317), only between knowledge and attitude.

“This suggests that while knowledge serves as a foundation, attitude is a crucial intermediary shaping behavior,” investigators wrote.

The team added that, because hyperuricemia is a metabolic risk factor and not just a symptomatic disease, patients need to focus on preventive strategies, including lifestyle modification, dietary counseling, appropriate urate-lowering therapy, and regular biochemical monitoring. A study earlier this year using a new machine learning prediction model reported that physical activity and less sedentary time reduced the risk of gout in people with hypouricemia.

While Shi and colleagues’ study showed patients with hyperuricemia had a high commitment to following medical recommendations, the same was not always the case for asymptomatic patients.

 Many asymptomatic individuals underestimate the need for early prevention, whereas symptomatic patients view the disease as more serious and report higher attitude and practice scores.

Subgroup analyses showed that attitude played a stronger mediating role in patients without gout symptoms. In patients without gout, knowledge significantly influenced attitude (P = 0.009) and indirectly affected practice through attitude (P = 0.004); attitude also strongly impacted practice (P = 0.015). Conversely, in patients with gout symptoms, knowledge did not significantly influence attitude (P = 0.488) or practice (P = 0.509), though attitude still significantly affected practice (P = 0.025).

Patients with comorbidities, such as hypertension or other chronic conditions, were associated with lower knowledge and attitude scores, indicating that competing health priorities may impact patient engagement. However, patients’ existing awareness of hyperuricemia offers a strong starting point for tailored education by healthcare providers.

The study ultimately suggests that targeted educational interventions, such as dietary counseling, motivational interviewing, and support groups, may improve patients’ attitudes toward self-managing their hyperuricemia.

“…it is recommended to institute regular follow-up appointments for monitoring and reinforcing adherence, implement tailored behavioral interventions that strengthen attitudinal engagement, especially in asymptomatic patients, develop individualized care plans, and bolster multidisciplinary collaboration among healthcare professionals,” investigators concluded.

 “In particular, clinical practices should focus on correcting misconceptions about preventive measures before gout onset, strengthening patients’ motivation for lifestyle modification, and encouraging participation in health education and peer-support programs, which were areas identified as relatively weaker in our findings.”

Shi H, Zhu Y, Guo Q, et al. Patients' knowledge influences practice via attitudes toward hyperuricaemia: a mediation analysis. 

. Published online December 3, 2025. doi:10.1007/s10067-025-07813-1

Johnson V. Physical Activity and Sedentary Time Top Factors for Gout Risk in People With Hyperuricemia. HCPLive. April 30, 2025. Accessed December 10, 2025. 

https://join.hcplive.com/view/physical-activity-and-sedentary-time-top-factors-for-gout-risk-in-people-with-hyperuricemia

Articles

A recent study highlights the importance of patient attitudes in the self-management of hyperuricemia, especially among asymptomatic individuals. While patients generally adhere to medical recommendations, asymptomatic patients often underestimate the need for early prevention. The study found that knowledge influences practice indirectly through attitude, emphasizing the need for targeted educational interventions. Subgroup analyses revealed that attitude plays a stronger mediating role in patients without gout symptoms. The study suggests implementing tailored behavioral interventions and multidisciplinary collaboration to enhance patient engagement and correct misconceptions about preventive measures.

A study shows that knowledge of hyperuricemia alone doesn’t drive practice. Attitude significantly mediates self-management, particularly in asymptomatic individuals.

Gout

On the HCPLive gout page, resources on the topics of medical news and expert insight into gouty arthritis can be found. Content includes articles, interviews, videos, podcasts, and breaking news on gout, and more.

Rheumatology

Rheumatology condition center page is a comprehensive resource for clinical news and insights on rheumatologic disease. This page consists of interviews, articles, podcasts, and videos on the research, treatment and development of therapies for arthritis, gout, nr-AxSpA, and more.

Patient Attitudes Key to Effective Hyperuricemia Self-Management

Jun Ho Jang, MD, PhD, is a professor in the division of hematology-oncology at the Samsung Medical Center.

Combination Pozelimab and Cemdisiran Improves LHD Control in PNH, With Jun Ho Jang, MD, PhD

paroxysmal nocturnal hemoglobinuria (PNH)

 who received prior C5 inhibitor therapy exhibited robust control of lactose dehydrogenase (LHD) after transitioning to a combination of pozelimab and cemdisiran with a favorable safety profile compared to standard-of-care ravulizumab therapy, according to results from a phase 3 trial.

 by Jun Ho Jang, MD, PhD, a professor in the division of hematology-oncology at the Samsung Medical Center, this study highlights a new potential treatment pathway for PNH which may result in complete and durable inhibition of C5.

“This clinical trial shows the safety data is similar to the other clinical trials,” Jang told 

 in an exclusive interview. “But, from an efficacy view, dual inhibition is more effective because our data shows that the LDH level was maintained around 1, but other clinical trials have shown that LDH is maintained around 1.5.”

PNH is an extremely rare disorder, with an incidence of around 1 per million and a prevalence of around 8 per million in the US. The disease is caused by somatic mutations in the X-linked phosphatidylinositol glycan anchor biosynthesis class A gene, which triggers complete or partial deficiency of glycosylphosphatidil-anchored proteins (GPI-APs). This makes PNH erythrocytes sensitive to intravascular hemolysis and thrombosis. Additionally, PNH clones can be found in patients with 

This study was an open-label extension of a previous phase 3, open-label, active-controlled study, which indicated that the combo treatment led to robust control of LHD, with more patients achieving meaningful LDH control versus ravulizumab. The extension is a phase 3, open-label, 2-arm, multicenter study, consisting of a 108-week treatment period followed by a 52-week post-treatment safety follow-up for any patient who discontinued treatment. Patients who had received combo during the parent study continued to receive combo, while those who were assigned to ravulizumab were assigned to combo.

The primary efficacy endpoint for the extension was percent change in LDH from baseline to week 36. For patients given combo, baseline was defined as the last assessment prior to the first dose of treatment in the extension study. For those who had received ravulizumab in the parent study, baseline was the last assessment prior to the first dose of cemdisiran. Secondary endpoitns included change in CH50, transfusion avoidance, and breakthrough hemolysis events.

A total of 44 patients were included in the extension, of whom 19 patients on combo and 22 patients switched from ravulizumab received treatment until week 36. Mean baseline LDH was 22.1 U/L for patients staying on combo and 312.4 U/L for patients switched to ravulizumab. At week 36, the mean percent change from baseline was +6.4 for patients staying on combo and -19.7 for those switched to ravulizumab. After the first dose of combo, 21 of 22 patients in each arm maintained adequate control of LDH, defined as ≤1.5x the upper limit of normal. All patients who switched from ravulizumab achieved and maintained CH50 levels of 0 U/mL from baseline to week 24, with a mean change of -15 U/mL from baseline.

Ultimately, Jang and colleagues determined that combo therapy resulted in robust control of LDH. It was well-tolerated, with a favorable safety profile and no adverse events related to the development of large immune complexes or type III hypersensitivity reactions.

“Nowadays, we have lots of treatment options for PNH,” Jang said. “But, to select a specific treatment, we need to consider many factors, such as patient aging, morbidity, patient prevalence, and transfusion dependency.”

Editor's Note: Jang reports disclosures with AstraZeneca, Novartis, Samsung Bioepis, Sanofi, Regeneron, Kyowa Kirin, and others.

Jang JH, Patriquin C, Taneja D, et al. Efficacy and safety of pozelimab plus cemdisiran versus ravulizumab in patients with paroxysmal nocturnal hemoglobinuria who received prior C5 therapy. Abstract presented at the 67th American Society of Hematology Annual Meeting. Orlando, FL. December 6-9, 2025.

Bravo-Perez C, Guarnera L, Williams ND, Visconte V. Paroxysmal Nocturnal Hemoglobinuria: Biology and Treatment. Medicina (Kaunas). 2023;59(9):1612. Published 2023 Sep 6. 

Jang discusses the results of an open-label extension highlighting the comparative efficacy of this combination therapy versus standard-of-care ravulizumab.

Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria Condition Center page, visitors can find resources on the topics of medical news and expert insight into PNH. Content includes articles, interviews, videos, podcasts, and breaking news on PNH research, treatment, and drug development.

Professor and Laurence A. Boxer Research Professor of Pediatrics and Professor of Pathology, University of Michigan and CS Mott Children’s Hospital, Ann Arbor, Michigan

HOPE-B: 5-Year Etranacogene Dezaparvovec Maintains Durable Efficacy and Safety in Hemophilia B, With Steven Pipe, MD

Analysis from the HOPE-B trial shows that a single dose of etranacogene dezaparvovec maintains durable, endogenous factor IX (FIX) Padua expression and produces sustained reductions in bleeding rates among adults with severe or moderately severe 

 by Steven Pipe, MD, a professor of pediatrics and pathology at the University of Michigan, and provide the longest follow-up data to date from a phase 3 trial of gene therapy in hemophilia B, evaluating long-term durability of FIX expression, efficacy, and safety.

“For patients who want to be freed from regular regular factor IX infusions, this is really the only therapeutic strategy that can offer that,” Pipe explained in an interview with 

. “A single infusion giving years of stable expression and bleed protection is truly transformative. The earlier patients receive that benefit, the more we can change their long-term joint health trajectory.”

Etranacogene dezaparvovec (Hemegenix) is an adeno-associated virus vector-based one-time gene therapy. In 2022, the 

 approved etranacogene dezaparvovec as the first gene therapy for adults with hemophilia B who use factor IX prophylaxis therapy and have current or prior life-threatening bleeding or repeated spontaneous bleeding episodes.

“Integrating gene therapy into routine clinical practice requires substantial planning, building infrastructure, training staff, and refining workflows, it’s very different from administering factor replacement,” said Pipe. “But with the right systems in place, we can make the experience manageable for patients by leveraging local labs and minimizing disruptions to their daily lives.”

These findings were demonstrated by HOPE-B, a phase 3, open-label, single-arm clinical trial where patients received a single intravenous infusion of etranacogene dezaparvovec (2×10¹³ gc/kg) after a ≥6-month lead-in period of standard-of-care FIX prophylaxis to establish baseline bleeding rates. Efficacy endpoints included annualized bleeding rates (ABR), endogenous FIX activity, and exogenous FIX usage. Safety assessments included adverse events, liver function, and long-term risks such as oncogenicity.

The study population was comprised of adult males with severe or moderately severe hemophilia B (FIX ≤2%) with or without preexisting AAV5 neutralizing antibodies. Of 54 participants treated with etranacogene dezaparvovec, 50 completed 5 years of follow-up.

Investigators observed a significant reduction in ABR for all bleeds, from 4.16 during the lead-in period to 1.52 during months 7–60 post-gene therapy (rate ratio 0.37; 95% Confidence Interval [CI], 0.18–0.76; 

 = .0035). Annualized bleed rates at years 1, 2, 3, 4, and 5 were 1.33 (n = 54), 0.91 (n = 54), 0.83 (n = 53), 0.40 (n = 51), and 0.40 (n = 51), respectively. Spontaneous ABR decreased from 1.52 during the lead-in to 0.53 during months 7–60 (

 = .0133). Joint ABR decreased from 2.34 to 0.35, and traumatic ABR decreased from 2.01 to 0.34 (

During years 1-5, mean endogenous FIX activity remained stable at >36%, with similar outcomes in NAb-positive and -negative participants. Exogenous FIX use decreased by 96%, from 257,339 IU/year during the lead-in period to 10,924 IU/year during months 7–60 post-gene therapy (

 <.0001). Only 1 participant resumed FIX prophylaxis at 30 months following a decrease in FIX activity to 2-5% and the occurrence of spontaneous bleeding events.

Over the 5-year follow-up, investigators reported a total of 100 treatment-related adverse events (AE) in 39 participants. Most occurred within the first 4 months post-gene therapy, with only 5 occurring between years 4 and 5. The most frequent AE was alanine aminotransferase elevation (n = 10). According to investigators, only 9 participants received corticosteroids for a mean of 81.4 days (range, 51–130 days). They observed no long-term hepatotoxicity or AAV-related oncogenicity.

Consenting HOPE-B participants will continue long-term monitoring up to 15 years in the IX-TEND 3003 study.

Editor’s Note: Pipe reports relevant disclosures with Bayer, BioMarin, Chugai, and others.

Pipe S, Miesbach W, Recht M, et al. End-of-study analysis of the HOPE-B trial confirms the durable efficacy and safety of etranacogene dezaparvovec hemophilia b gene therapy over 5 years. Abstract presented at the 67th American Society of Hematology Annual Meeting. Orlando, FL. December 6-9, 2025.

Heo YA. Etranacogene Dezaparvovec: First Approval. 

. Published online February 21, 2023. doi:

https://doi.org/10.1007/s40265-023-01845-0

Iapoce C. Hemophilia B Gene Therapy Achieves Sustained Benefit Over 4 Years. 

 February 7, 2025. Accessed December 10, 2025. https://www.hcplive.com/view/hemophilia-b-gene-therapy-achieves-sustained-benefit-over-4-years

End-of-study analysis from the HOPE-B trial shows durable endogenous FIX expression, reduced annualized bleeding rates, and a favorable safety profile.


Dr. Sambhara is the founder and Medical Director of Research for the Eye Clinic of Wisconsin’s clinical trial program.

Advice on Maintaining Flexibility with Second-Generation Anti-VEGF Agents in Retinal Vascular Diseases

In this closing segment, Dr. Harit Bhatt and Dr. Deepak Sambhara reflect on the importance of collaboration, mentorship, and lifelong learning in the evolving field of retina. Dr. Sambhara shares heartfelt advice for new retina specialists, emphasizing that “availability is the best ability.” He encourages early-career physicians to stay present and engaged—whether by attending meetings, collaborating with colleagues and industry partners, or studying emerging clinical data to remain on the leading edge of innovation. He highlights the value of building relationships with mentors and peers, noting how the retina community’s openness and collegiality have been vital to his own growth. Dr. Bhatt echoes these sentiments, reinforcing the importance of curiosity and continuous learning as the field rapidly advances. The discussion concludes on an inspiring note, celebrating the shared mission of improving patient outcomes through teamwork, innovation, and dedication to lifelong education.

Dr. Sambara emphasizes the importance of collaboration and continuous learning for new retina specialists to thrive in their practice.

Video Series

Experts discuss the future of retinal disease treatments, highlighting the promise of new therapies and the need for personalized patient care.

 Integrating Gene Therapy and TKIs with Current Second-Generation Anti-VEGF Agents in Clinical Practice

Experts discuss evolving strategies in treat-and-extend protocols for retinal diseases, emphasizing OCT's role in optimizing patient care and outcomes.

Treat-and-Extend Strategies with Second-Generation Anti-VEGF in Patients with Retinal Vascular Diseases

Experts discuss the safety of retinal treatments, addressing concerns about rare side effects and emphasizing the overall efficacy of these medications.

Reinforcing Safety and Patient Confidence with Second-Generation Anti-VEGF Therapies

Experts discuss switching from 2 mg to 8 mg treatments for AMD and DME, emphasizing loading strategies and reduced steroid use in practice.

Optimizing Treatment Transitions and Reducing Steroid Use with Second-Generation Anti-VEGF Therapies in Retinal Vascular Diseases

Healthcare professionals discuss the integration of second-generation medications into practice, emphasizing patient-specific responses and treatment challenges.

Integrating Second-Generation Anti-VEGF Therapies into Clinical Practice 

Experts discuss promising results from the Quasar trial for Eylea 8 mg, highlighting its potential impact on retinal vein occlusion treatment strategies.

Aflibercept 8 mg in Patients with RVO 

Explore the latest findings from the Pulsar and Photon trials, highlighting innovative treatment extensions for AMD and DME patients.

Long-Term Insights with the High-Dose Anti-VEGF Agent Aflibercept 8 mg In AMD and DME Management 

Physicians discuss the promising durability and safety of Flibrice 8 mg, highlighting its potential to improve patient care in challenging cases.

The Role of Faricimab in Extending Durability and Treating Retinal Vascular Diseases

Experts discuss advancements in retinal vascular disease treatment, focusing on the impact of next-generation anti-VEGF therapies and their safety.

Enhancing Treatment Durability with Second-Generation High-Dose Anti-VEGF Agents in Retinal Vascular Diseases 

Discover insights on evolving retinal care standards with next-generation anti-VEGF therapies from leading experts in the field.

Latest News

Higher Maintained Hemoglobin Associated With Improved Survival in MDS, With Ira Zackon, MD

Patient Attitudes Key to Effective Hyperuricemia Self-Management

Combination Pozelimab and Cemdisiran Improves LHD Control in PNH, With Jun Ho Jang, MD, PhD

HOPE-B: 5-Year Etranacogene Dezaparvovec Maintains Durable Efficacy and Safety in Hemophilia B, With Steven Pipe, MD

Advice on Maintaining Flexibility with Second-Generation Anti-VEGF Agents in Retinal Vascular Diseases