Report from the 57th American Society of Hematology Annual Meeting and Exposition — Benign Hematology

Editors from NEJM Journal Watch Oncology and Hematology attended the recent meeting of the American Society of Hematology (ASH). Here, Dr. David Green describes the latest findings in benign hematology.

Anti-Sickling Drug for Sickle Cell Disease

GBT440 reversibly binds to the alpha chain of hemoglobin, stabilizing the oxy-Hb conformation and inhibiting HbS polymerization. Polymerization of HbS induces red cell sickling, hemolysis, and vaso-occlusion in patients with sickle cell disease (SCD).

To examine the safety and efficacy of GBT440, investigators conducted a prospective, randomized, placebo-controlled, double-blind phase I/II study in 64 healthy volunteers and 16 SCD patients (abstract 542).

GBT440 had a terminal half-life of 1.5–3 days, p…