Sickle Cell Anemia

Allogeneic Marrow Transplantation for Treatment of Sickle Cell Disease in Symptomatic Adults.

In collaboration with Fred Hutchinson Cancer Research Center

Objectives:

1. To define the role and risk/benefit of marrow transplantation in adults with sickle cell disease.

    

2. To evaluate the efficacy of the preparative regimen in ablating the underlying sickle cell disease.

    

Treatment:

Eligible patients will undergo treatment with the conditioning regimen of busulfan, cyclophosphamide, and antithymocyte globulin followed by conventional allogeneic bone marrow transplantation from a HLA-identical sibling with cyclosporine A and methotrexate graft-versus-host disease prophylaxis.

Eligibility:

1. Patients must have sickle cell disease (SS, SC, or Sb-thalassemia) and at least one of the following complications:

   a. Evidence of "silent" cerebral infarction by impaired neuropsychologic performance and abnormal MRI scan.

   b. Stage I-II sickle cell lung disease and/or a history of recurrent acute chest syndrome.

   c. Sickle cell nephropathy.

   d. Recurrent pain episodes and/or multiple joint osteonecrosis.

   e. Chronic RBC transfusion requirement with the development of RBC alloimmunization.

    

2. Patients with extensive cerebral vasculopathy, Stage III-IV sickle cell lung disease, severe renal impairment (GFR <30% predicted for age), or demonstrated lack of compliance with prior medical care are not eligible.

    

3. Patients must be between the ages of 16 and 40.

    

4. Patients must have a Karnofsky performance status of at least 70%.

    

5. Patients must be HIV negative.

Patients must not have active hepatitis or severe portal fibrosis/cirrhosis.