Rilzabrutinib was well tolerated and associated with rapid, durable responses in heavily pretreated adults with immune thrombocytopenia.
In patients with cGVHD after alloSCT and 2-5 prior lines of systemic therapy, belumosudil was well tolerated with response rates > 70% and a median DoR of 50 weeks.
Early data suggest that the combination of itacitinib and calcineurin inhibitors as prophylaxis for GVHD was well tolerated in patients with hematologic malignancies undergoing stem cell transplantation.
Baricitinib appears to be well tolerated and effective for patients with therapy-refractory severe chronic GVHD, demonstrating rapid and durable responses.
HRQoL was sustained in the phase III BELIEVE study of luspatercept vs placebo in transfusion-dependent β-thalassemia.
Among the first 10 study participants with ≥ 3 months of follow-up, CTX001 infusion achieved cessation of pRBC transfusions for TDT patients, and eliminated veno-occlusive events for severe SCD patients.
Proof-of-concept phase I data for mitapivat, an oral, small molecule inhibitor of pyruvate kinase R, shows early evidence of efficacy and safety in patients with stable sickle cell disease.
Data from REACH3 shows ruxolitinib significantly improves responses and symptoms at Week 24 in patients with cGVHD, and inadequate steroid response, when compared with BAT.
Results from this phase III study of mycophenolate mofetil and a short course of corticosteroids vs corticosteroids alone as first-line treatment for patients with ITP shows the combination therapy to be safe and effective, reducing the rate of treatment failure by more than one half.
Initial results from HOPE-B, the largest phase III study of gene therapy for hemophilia B to date, show etranacogene dezaparvovec yields clinically meaningful levels of factor IX activity within 6 months.