Hematology Month in Review: July 2024

July’s hematology month in review underlines the field’s forward stride in improving care, with key updates to the hematological pipeline and study data on benefiting patient outcomes. Recent trials have demonstrated notable advances in treatments for hematological conditions. AFFINE showed giroctocogene fitelparvovec significantly reduces bleeding rates in hemophilia A patients compared with standard prophylaxis, while XTEND-Kids confirmed the benefit of once weekly efanesoctocog alfa for children with the condition. REACH5 showed ruxolitinib's effectiveness and tolerability in pediatric chronic graft-versus-host disease

Other trials focused on the risk-benefit of mRNA COVID-19 vaccines for sickle cell disease, ultimately releasing data in favor of the SARS-CoV-2 vaccination. Online survey data noted the need for more consistency in iron deficiency screening in heart failure and a recent case-control study found long-term exposure to prescription medication could influence iron deficiency anemia development.

Pipeline News

Hemophilia A Gene Therapy Displays Superiority in Phase 3 AFFINE Trial

Topline Phase 3 results showed giroctocogene fitelparvovec met the primary non-inferiority and key superiority objectives of total annualized bleeding rate in a 15-month follow-up period, compared with standard-of-care FVIII prophylaxis, in patients with moderately severe to severe hemophilia A.

“I’m excited by the strength of these positive results from the AFFINE trial that show giroctocogene fitelparvovec was generally well-tolerated, and demonstrate the transformative potential of this gene therapy candidate to provide superior bleed protection compared with routine FVIII prophylaxis, while helping relieve the treatment burden for people living with hemophilia A,” said Andrew Leavitt, MD, director of the Adult Hemophilia Treatment Center, University of California, San Francisco.

XTEND-Kids Trial Reinforces Safety, Efficacy of Efanesoctocog Alfa in Children

Results from the Phase 3 XTEND-Kids trial reinforced the safety and efficacy of once-weekly efanesoctocog alfa (ALTUVIIIO) prophylaxis for children with severe hemophilia A. The treatment provided high sustained factor VIII activity in the normal to near-normal range, leading to effective bleeding prevention, and was associated with mainly nonserious adverse events.

“By providing high-sustained factor VIII activity levels consistently early in life with once-weekly dosing, [ALTUVIIIO] has the potential to improve reported outcomes for children living with hemophilia,” said principal investigator Lynn Malec, MD, associate professor of medicine and pediatrics at The Medical College of Wisconsin.

REACH5: Ruxolitinib Active, Well-Tolerated for Chronic GVHD in Children

Interim analysis of the Phase 2 REACH5 study showed the efficacy and tolerability of ruxolitinib for treatment-naive or corticosteroid-refractory pediatric patients with chronic graft-versus-host disease (GVHD).

In these data, ruxolitinib treatment led to an overall response rate of 40% at Cycle 7 Day 1 of treatment administration, in 28-day cycles, for approximately 3 years.

“Pending final analysis, this study suggests that ruxolitinib is active and well-tolerated in both treatment-naive and corticosteroid-refractory patients aged 2 years to younger than 18 years with chronic GVHD, thereby supporting its use in this patient population,” wrote investigators.

Study Updates

COVID-19 Vaccination Shows Favorable Risk-Benefit in Sickle Cell

An American Society of Hematology (ASH) network analysis revealed a positive risk-benefit ratio of mRNA vaccination for COVID-19 among individuals with sickle cell disease (SCD).

Those with SCD achieved antibodies against SARS-CoV-2 at similar rates to the general population, with decreases in circulating anti-receptor binding domain antibodies comparable to immune decay in vaccine recipients.

“It is our hope that this information will help inform providers and future research studies as well as offer some assurance to those in the SCD community that future mRNA vaccines can offer more benefit than risk to this patient population,” wrote investigators.

Survey Reports Heterogeneity in Iron Deficiency Management in Heart Failure

Online survey data from the IRON-HF international survey underlined the need for consistency in iron deficiency screening and treatment strategies for patients with heart failure (HF). Most physicians confirmed routine screening for iron deficiency in HF patients, but the data revealed an underutilization of iron supplementation strategies.

“Commitment by scientific societies to the diffusion of standardized screening and treatment protocols, as well as the development of comprehensive HF education programs, are pivotal to bridge these gaps in care,” wrote investigators.

Prescription Medication May Influence Iron Deficiency Anemia Development

A case-control study found long-term exposure to proton pump inhibitors and oral anticoagulants was independently associated with the risk of iron deficiency anemia (IDA) development. On the other hand, longstanding treatment with antiplatelet therapy, antidepressants, or non-steroidal anti-inflammatories did not affect the development of IDA.

“While the association does not prove causation, the findings could potentially influence prescribing practice, particularly of PPIs in those with unexplained recurrent IDA,” wrote investigators.

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