Positive Results Seen With Voxelotor in Pediatric Patients With Sickle Cell Disease
Researchers sought to determine whether the use of voxelotor in a younger population of patients with sickle cell disease would be safe and effective.
Researchers sought to determine whether the use of voxelotor in a younger population of patients with sickle cell disease would be safe and effective.
Researchers sough to determine whether belumosudil would help treat patients with chronic GVHD.
Researchers sought to determine whether mitapivat would improve clinical outcomes in patients with thalassemias who are not transfusion-dependent.
Researchers sought to determine whether BT200, a new anti-von Willebrand factor, would have efficacy in patients with von Willebrand disease.
Researchers sought to determine whether iptacopan would have efficacy in first line treatment of patients with PNH.
Researchers sought to determine whether pevonedistat plus azacitidine would result in a lower residual mutation load than seen with azacitidine alone in patients with MDS.
The median duration of response was 35.7 weeks with iberdomide, bortezomib, and dexamethasone compared with not reached in patients who received iberdomide, daratumumab, and dexamethasone or iberdomide, bortezomib, and carfilzomib
The median progression-free survival was not reached in the D-Rd group compared with 34.4 months in the Rd group.
Adding isatuximab to pomalidomide and dexamethasone improved progression-free survival (PFS) and PFS2.
In patients who had received at least 2 prior therapy lines, the overall response rates were 49.9% in SCHOLAR-5 vs 94.2% in ZUMA-5.