Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
Status:
Recruiting
Trial end date:
2026-08-31
Target enrollment:
Participant gender:
Summary
Severe aplastic anemia is a rare and serious form of bone marrow failure related to an
immune-mediated mechanism that results in severe pancytopenia and high risk for infections
and bleeding. Patients with matched sibling donors for transplantation have a 80-90% chance
of survival; however, a response rate with just immunosuppression for those patients lacking
suitable HLA-matched related siblings is only 60%. With immunosuppression, only 1/3 of
patients are cured, 1/3 are dependent on long term immunosuppression, and the other 1/3
relapse or develop a clonal disorder. Recent studies have shown that using a haploidentical
donor for transplantation has good response rates and significantly lower rates of acute and
chronic GVHD.