Cyclophosphamide and Antithymocyte Globulin, With or Without Total Body Irradiation, Followed By Allogeneic Hematopoietic stem Cell Transplantation For Severe Aplastic Anemia

Protocol Number: RMBMT-02

Major Objectives

• Cyclophosphamide 50mg/kg/day in the morning of days –5 through –2.
• ATG 30mg/kg in the evening of days –5 through –3.
• 12 hours between the am cyclophosphamide dose and the pm ATG dose.
• TBI 200cGY on Day 0 for unrelated donor transplant recipients.
• Transplant on Day 0.

Patient Selection

Inclusion Criteria:

• Bone marrow cellularity <20%.
• Two or more of the following abnormalities: ANC ≤ 500μl, platelets ≤ 20,000/μ/l, reticulocytes ≤ 50,000/μl.
• Total serum bilirubin < 2 times the upper limit of normal and calculated creatinine clearance ≥ 60ml/minute.
• Left ventricular ejection fraction measured by radionuclide ventriculography of ≥ 50%.
• An HLA-compatible donor, defined as a related or unrelated donor who is HLA-identical or a related donr mismatched at a single HLA-locus.

Exclusion Criteria:

• Uncontrolled infection or comorbidities that would preclude transplantation.
• Female patients who are pregnant and/or who do not practice adequate contraception.
• Patients who are HIV positive.
• Patients with clonal cytogenetic abnormalities or myelodysplasia diagnosed less than 3 months prior to the diagnosis of SAA.
• Patients with congenital or constitutional aplastic anemia or Fanconi anemia.