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Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
Sponsor: Northside Hospital, Inc.
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.
Official title: A Study of T-Cell Replete, HLA-Mismatched Haploidentical Bone Marrow Transplantation With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Lacking HLA-Matched Related Donor
Key Details
Gender
All
Age Range
1 Year - 75 Years
Study Type
INTERVENTIONAL
Enrollment
20
Start Date
2016-09-09
Completion Date
2027-08-31
Last Updated
2025-10-30
Healthy Volunteers
No
Conditions
Interventions
Fludarabine
30 mg/m2 IV QD x 5 days (Days -6 to -2)
Cyclophosphamide
14.5 mg/kg/day IV x 2 doses (Days -6 \& -5)
Total Body Irradiation
300 cGy x1 dose (Day -1)
Rabbit ATG
1.5 mg/kg/day x 3 days (Days -3 to -1)
Cyclophosphamide
Post-transplant: 50 mg/kg IV QD (Day +3 to +4)
Locations (2)
Blood and Marrow Transplant Group of Georgia
Atlanta, Georgia, United States
Northside Hospital
Atlanta, Georgia, United States