According to research published in Lancet Haematology, a novel approach to haploidentical bone marrow transplantation results in excellent overall survival (OS) with minimal graft vs host disease (GVHD) in patients with relapsed or refractory severe aplastic anemia and may now be considered a standard salvage treatment for the disorder.
The researchers reported the outcomes of BMT CTN 1502, a single-arm, multicenter, phase 2 clinical trial that evaluated OS in patients (≤75 years) with relapsed or refractory severe aplastic anemia after haploidentical bone marrow transplantation with use of post-transplantation cyclophosphamide-based GVHD prophylaxis (ClinicalTrials.gov Identifier: NCT02918292). The study’s primary endpoint was 1-year post-transplantation OS.
A total of 31 patients (median age, 24.9 years; interquartile range [IQR], 10.4-51.3; male, 61%; female, 39%) underwent bone marrow transplantation. The median follow-up duration was 24.3 months (IQR, 12.1-29.2). Patients who were site-reported as non-White comprised 42% of the patient population, and most (61%) were from under-represented racial and ethnic groups.
At 1-year posttransplant, 77% of patients were alive with engraftment, and 3% of patients were alive with autologous recovery. The 1-year OS was 81% (95% CI, 62-91).
The most common grade 3-5 adverse events (AEs) included abnormal liver tests (23%), cardiovascular changes (including sinus tachycardia, heart failure, pericarditis; 48%), gastrointestinal issues (32%), nutritional disorders (23%), and respiratory disorders (26%). Death due to disease and unsuccessful bone marrow transplantation was reported for 19% of patients.
“This study provides strong evidence for use of post-transplantation cyclophosphamide-based GVHD prophylaxis for patients with severe aplastic anemia when a haploidentical donor is used,” the researchers concluded.
DeZern AE, Eapen M, Wu J, et al. Haploidentical bone marrow transplantation in patients with relapsed or refractory severe aplastic anaemia in the USA (BMT CTN 1502): a multicentre, single-arm, phase 2 trial. Lancet Haematol. Published online September 9, 2022. doi:10.1016/S2352-3026(22)00206-X