Avatrombopag increases cumulative platelet response rate vs placebo in refractory chronic ITP.
This ad hoc analysis found durable responses and multiple episodes of clinically meaningful reductions in transfusion dependence among patients with β-thalassemia receiving luspatercept.
Interim results from the phase III Northstar-3 study showed that most patients with transfusion-dependent β-thalassemia who received betibeglogene autotemcel gene therapy produced sufficient HbAT87Q to stop RBC transfusions for ≥ 3 months.
Early-stage data suggest rapid, durable activity of the novel Bruton tyrosine kinase inhibitor PRN1008 in heavily pretreated patients with relapsed/refractory ITP.
Preliminary data showed that sutimlimab resulted in rapid and durable increase in platelet count in patients with multirefractory chronic ITP.
First-in-class inhibitor of classical complement pathway demonstrates rapid and durable efficacy in cold agglutinin disease.
Preliminary results from pilot feasibility study suggest that a gene therapy construct targeting BCL11A is safe, effectively induces HbF expression, and improves sickling signs and symptoms when delivered to patients with severe SCD.