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.
The overall response rate was 44.7% after a median follow-up of 15 months.
The tumor lysis syndrome risk decreased for 84.6% of patients in the ibrutinib/venetoclax group.
The median overall survival was not reached at 3 years.
The 7-year survival results were superior with MATRIX.
The 3-year overall survival rate was 95% with one regimen and 100% with the other.
Relapsed/refractory disease and older age were also associated with prolonged hospital stay and death.
The mortality rate was 0.13% in the overall chronic myeloid leukemia cohort.
Roughly a quarter of treatment cycles were administered solely as home-based treatment, with no hospital admissions required.
Six patients experienced a grade 5 treatment-related adverse event.
The 12-month overall survival rate was 88%.