Researchers also measured in the study cohort a very high discontinuation rate for this agent.
Heme Today
Heme Today covers the latest news and analyses in bleeding, clotting, red blood cell, and iron disorders for hematologists and oncologists focused on bleeding disorders.
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In a cohort of adults and adolescents marstacimab produced bleed control superior to that from on-demand bypassing agent.
The agent notably in preclinical studies has accomplished increased HbF without any evident cytotoxicity.
The phase 3 VERONA trial, which compared this combination with placebo plus azacitidine, observed no new safety signals.
Dr. John Gansner and Dr. Martina H. Slingsby argue this new agent can provide benefit across all bleeding disorders.
Heme Today spoke with Ulrike Reiss, MD, and Andrew Davidoff, MD, of the trial's investigator team.
Gene therapy is reshaping sickle cell care, raising awareness and advancing treatments beyond the lab.
ASH warns NIH cuts would jeopardize blood disorder care and halt key progress in hematology research.
Reduced-intensity HSCT conditioning with lentiviral gene therapy decreased severe vaso-occlusive events by more than 80%.
An interim analysis of the COMPLETE study found that pegcetacoplan improved indicators of blood health.
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A phase II trial calculated a 4-year event-free survival rate of 96% and overall survival rate of 100% from the combination.
Differences were identified between patient racial groups and by urban versus rural populations.
A magnetic resonance imaging study has compared tissue iron levels and biventricular function between the two conditions.
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