Overview

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

Status:
Recruiting
Trial end date:
2026-08-31
Target enrollment:
0
Participant gender:
All
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.
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Northside Hospital, Inc.
Treatments:
Cyclophosphamide
Fludarabine
Fludarabine phosphate
Thymoglobulin
Criteria
Inclusion Criteria:

- Availability of 3/6 - 5/6 matched (HLA-A, B, DR) related donor who must have negative
HLA cross-match in the host vs. graft direction

- Age <= 65 years for previously treated and <= 75 years for previously treated patients

- KPS >= 70%

- Aplastic Anemia that meets the following criteria:

Peripheral Blood (must fulfill 2 of 3):

- <500 PMN/mm3

- <20,000 platelets

- absolute reticulocyte count <40,000/microL

Bone Marrow (must be either):

- markedly hypocellular (<25% of normal cellularity)

- moderately hypocellular with 70% non-myeloid precursors and patient meets peripheral
blood criteria above

Exclusion Criteria:

- poor cardiac function (LVEF <40%)

- poor pulmonary function (FEV1 & FVC <50% predicted)

- poor liver function (bili >= 2mg/dL)

- poor renal function (creatinine >= 2.0mg/dL or creatinine clearance <40mL/min)

- prior allogeneic transplant