Non-Myeloablative Bone Marrow Transplant for Patients With Sickle Cell Anemia and Other Blood Disorders

Inclusion Criteria

• •DISEASE CHARACTERISTICS:
• •Diagnosis of 1 of the following sickle cell anemias (Hb SS):
• •* Hb S/β° thalassemia
• •* Hb S/β+ thalassemia
• •* Hb SC disease
• •* Hb SE disease
• •* Hb SD disease
• •* Hemoglobin SO-Arab disease
• •* Hb S/hereditary persistence of fetal hemoglobin
• •Meets 1 of the following criteria:
• •* History of invasive pneumococcal disease
• •* Stroke or CNS event lasting \> 24 hours
• •* MRI changes indicative of brain parenchymal damage
• •* Evidence of cerebrovascular disease by magnetic resonance angiography
• •* Acute chest syndrome requiring exchange transfusion or hospitalization
• •* Recurrent vaso-occlusive pain crisis (\> 2 per year for the last 2 years)
• •* Stage I or II sickle lung disease
• •* Sickle retinopathy
• •* Osteonecrosis
• •* Red cell alloimmunization (\> 2 antibodies) during long-term transfusion
• •* Constellation of dactylitis in the first year of life AND a baseline hemoglobin \< 7 g/dL and leukocytosis (WBC \> 13.4/mm\^3) in the absence of infection during the second year of life
• •* Pitted RBC count \> 3.5% during the first year of life
• •Ineligible for or refused bone marrow transplantation from an HLA-matched sibling donor
• •Partially mismatched (at least haploidentical) first-degree relative donor available
• •* No minor (donor anti-recipient) ABO incompatibility if an ABO compatible donor is available
• •PATIENT CHARACTERISTICS:
• •ECOG performance status (PS) 0-1 OR Karnofsky or Lansky PS 70-100%
• •LVEF ≥ 35%
• •FEV\_1 and forced vital capacity ≥ 40% predicted
• •Direct bilirubin \< 3.1 mg/dL
• •No moderate to severe pulmonary hypertension by ECHO
• •No debilitating medical or psychiatric illness that would preclude study participation
• •No HIV positivity
• •Not pregnant or nursing
• •Negative pregnancy test
• •Fertile patients must use effective contraception
• •PRIOR CONCURRENT THERAPY:
• •No prior transfusions from donor
• •No immunosuppressive agents, including steroids as antiemetics, within 24 hours after the last dose of post-transplantation cyclophosphamide