Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
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
Northside Hospital, Inc.
20 participants
Sep 9, 2016
INTERVENTIONAL
Conditions
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.
Eligibility
Inclusion Criteria11
- 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 Criteria5
- 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
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Interventions
30 mg/m2 IV QD x 5 days (Days -6 to -2)
14.5 mg/kg/day IV x 2 doses (Days -6 \& -5)
300 cGy x1 dose (Day -1)
1.5 mg/kg/day x 3 days (Days -3 to -1)
Post-transplant: 50 mg/kg IV QD (Day +3 to +4)
Locations(2)
View Full Details on ClinicalTrials.gov
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NCT02828592