Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

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.

NCT Number	NCT02828592
Lead Sponsor	Northside Hospital, Inc.
Conditions	Severe Aplastic Anemia
Enrollment	20 participants
Start Date	2016-09-09
Primary Completion	2027-08-31 (estimated)
Study Completion	2028-08-31 (estimated)
Updated on ClinicalTrials.gov	2026-04-20