Independent Conference Highlights of the 2022 ASH Annual Meeting*

Weekly efanesoctocog alfa prophylaxis yields meaningful improvement in reduction of pain across multiple endpoints for patients with hemophilia A when switching from standard-of-care prophylaxis.

Vemircopan monotherapy demonstrated control of intravascular hemolysis, prevention of clinically significant extravascular hemolysis, and improvement in fatigue in treatment-naive patients with PNH.

In patients with primary/chronic ITP, efgartigimod was associated with clinically and statistically significant improvements in platelet counts vs placebo.

In patients with CAD without a recent transfusion history, the classical complement pathway inhibitor sutimlimab was associated with improvements in fatigue and quality of life that were maintained for more than 1 year.

In this current analysis of the phase III ATLAS-PPX switch trial, monthly fitusiran prophylaxis improved HRQoL more than factor/BPA prophylaxis in adult and adolescent patients with hemophilia A or B with or without inhibitors.

Ruxolitinib plus corticosteroids demonstrated high ORR and durable responses in pediatric patients with untreated or steroid-refractory acute GVHD, with a safety profile consistent with previous experience.

In patients with paroxysmal nocturnal hemoglobinuria and residual anemia on stable anti‒complement 5 standard-of-care therapy, iptacopan monotherapy provided significant hematologic improvement vs continued standard-of-care treatment.

Phase III BMT CTN 1703 trial of novel GVHD prophylaxis triplet PTCy + TAC + MMF vs TAC + MTX following reduced-intensity conditioning allogeneic stem cell transplant demonstrated improved GVHD-free relapse-free survival vs TAC + MTX.