Aplastic Anemia

A Trial Of Cyclophosphamide And Antithymocyte Globulin As Preparation For Allogeneic Bone Marrow Transplantation Of Patients With Severe Aplastic Anemia Who Have HLA-Identical Donors.

Objective:

The objective of this treatment protocol is the evaluation of the efficacy of high-dose cyclophosphamide and antithymocyte globulin as a means of obtaining durable engraftment and hematologic reconstitution in patients with severe aplastic anemia receiving allogeneic bone marrow transplants from
HLA-identical family donors.

Treatment:

Patients with severe aplastic anemia will be treated with cyclophosphamide and antithymocyte globulin, followed by conventional allogeneic bone marrow transplantation with tacrolimus and methotrexate graft-versus-host disease prophylaxis.

Eligibility:

1. Patients with severe aplastic anemia as defined by a markedly hypocellular bone marrow (<25% of normal) or moderately hypocellular bone marrow (25 - 50% of normal) with <30% of the remaining cells being hematopoietic.

    

2. Patients must have at least two of the following peripheral blood counts:

   a. Neutrophil count <500 mm3.

   b. Corrected reticulocyte count <1%.

   c. Platelet count <20,000 mm3.

    

3. Patients may not be older than 50 years of age.

    

4. Patients must have adequate renal, hepatic, cardiac and pulmonary function.