Stem Cell Transplant for Hemoglobinopathy

Inclusion Criteria

• •Patients with Sickle Cell Disease/Thalassemia (SCD/THAL) 0-50 years of age with an acceptable stem cell donor and disease characteristic defined by the following:
• •* Stroke, central nervous system (CNS) hemorrhage or a neurologic event lasting longer than 24 hours, or abnormal cerebral magnetic resonance imaging (MRI) or cerebral arteriogram or MRI angiographic study and impaired neuropsychological testing
• •* Acute chest syndrome with a history of recurrent hospitalizations or exchange transfusions
• •* Recurrent vaso-occlusive pain 3 or more episodes per year for 3 years or more years or recurrent priapism,
• •* Impaired neuropsychological function and abnormal cerebral MRI scan
• •* Stage I or II sickle lung disease,
• •* Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate \[GFR\] 30-50% of the predicted normal value)
• •* Bilateral proliferative retinopathy and major visual impairment in at least one eye
• •* Osteonecrosis of multiple joints with documented destructive changes
• •* Requirement for chronic transfusions but with red blood cell (RBC) alloimmunization \>2 antibodies during long term transfusion therapy
• •Patients with transfusion dependent alpha- or beta-thalassemia 0-35 years of age with an acceptable stem cell donor as defined in the criteria in section above.
• •Patients with other non-malignant hematologic disorders that are transfusion-dependent or involve other potentially life-threatening cytopenias (including but not limited to Severe Congenital Neutropenia, Diamond-Blackfan Anemia and Shwachman-Diamond Syndrome) who are 0-35 years of age with an acceptable stem cell donor
• •Second Transplants
• •* Patients with sickle cell disease or thalassemia who have failed to engraft or have autologous recovery after a myeloablative SCT regimen or non-myeloablative regimen are eligible for this protocol.
• •* Regimen A2 will be utilized for patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or for any patient who has pre-existing organ dysfunction making them ineligible for a myeloablative preparative regimen.
• •* Regimen B will be utilized for patients with sickle cell disease or thalassemia who have an HLA-identical sibling donor.
• •* Patients must meet above criteria.
• •* If the patient has received prior radiation therapy, eligibility to receive additional radiation therapy must be determined by Dr. Dusenbery
• •* If first transplant was a non-myeloablative regimen, the second transplant can occur at any time
• •* If the first transplant was a myeloablative regimen, then the second transplant must be \> 6 months from the first transplant