2021 American Society of Hematology Annual Meeting*

Results from a phase I trial of mitapivat reporting evidence of increased hemoglobin levels, improved oxygen affinity, decreased markers of hemolysis, and decreased sickling rate.

Results from an open-label phase II trial showing that crizanlizumab is well tolerated in adolescent patients (aged 12 to <18 years) with SCD and was associated with a reduction in the rate of veno-occlusive crises.

Iptacopan monotherapy lowered LDH levels ≥60% within 12 weeks in all patients with PNH in this phase II study.

Amphiregulin was validated as a biomarker for monitoring patients with life-threatening acute GVHD based on data from 2 prospective phase II trials.

Urinary-derived human chorionic gonadotropin/epidermal growth factor appears safe, well-tolerated, and efficacious in addition to immunosuppressive therapy for patients with life-threatening acute graft-vs-host disease.

In patients with heavily pretreated chronic GVHD, axatilimab showed promising clinical activity with symptom improvement and acceptable safety.

Abatacept achieved an ORR of 49% and sustained reduction in prednisone dosing over time in patients with steroid-refractory chronic GVHD.

Monthly fitusiran prophylaxis significantly reduced the rate of bleeding events and improved quality of life for individuals with hemophilia A or B with inhibitors compared with on-demand bypassing agents.

Etavopivat improved sickle RBC health and markers of sickle cell disease and pathophysiology in this phase I clinical study.

In the ATLAS-A/B trial, prophylactic treatment with fitusiran resulted in significant reductions in annualized bleeding rates compared with on-demand factor replacement therapy in patients with hemophilia A or B and no inhibitors.