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

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.

Description

Eligibility

Age range

1–75 years

Sex

All

Healthy volunteers

No

Interventions

• Drug
  Fludarabine

  30 mg/m2 IV QD x 5 days (Days -6 to -2)

• Drug
  Cyclophosphamide

  14.5 mg/kg/day IV x 2 doses (Days -6 \& -5)

• Radiation
  Total Body Irradiation

  300 cGy x1 dose (Day -1)

• Drug
  Rabbit ATG

  1.5 mg/kg/day x 3 days (Days -3 to -1)

• Drug
  Cyclophosphamide

  Post-transplant: 50 mg/kg IV QD (Day +3 to +4)

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