Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia

You may be eligible if

• Suitable UCB collection from an HLA-identical sibling
• Sickle cell anemia (Hb SS or S beta thalassemia) with significant disease manifestations as defined by at least one of the following criteria:
• 1. A history of painful events defined as three or more painful events in the 2 years prior to enrollment. Pain may occur in typical sites associated with vaso-occlusive painful events and cannot be explained by causes other than sickle cell disease. The pain must last at least 4 hours and require treatment with either parenteral narcotics, an equianalgesic dose of oral narcotics (if pain is treated in a local facility where parenteral narcotics are not routinely used to treat painful events), or parenteral nonsteroidal anti-inflammatory drugs. Painful events managed at home will be considered only if there is documentation of the event in a clinical record that may be reviewed by an investigator.
• 2. Acute chest syndrome (ACS) with two or more episodes of ACS with the development of a new infiltrate on chest radiograph and/or having a perfusion defect demonstrable on a lung radioisotope scan
• 3. Any combination of painful events and episodes of ACS that total three events in the 2 years before transplantation
• 4. Any clinically significant neurologic event (stroke or hemorrhage) or any neurologic defect lasting more than 24 hours
• 5. Abnormal cerebral MRI and abnormal cerebral MRA
• 6. An episode of dactylitis in the first year of life with significant anemia (Hbg less than 7 g/dL), or leukocytosis in the second year of life such that the risk of a severe adverse outcome before 18 years of age exceeds 54% (as defined by the cooperative study of sickle cell disease (CSSCD) infant cohort study)
• 7. History of positive trans-cranial Doppler studies (average greater than 200 cm/sec)
• Beta thalassemia major with significant disease manifestations as defined by the following criteria: Beta thalassemia genotype consistent with clinical diagnosis of beta thalassemia major (could include patients with E-beta thalassemia genotype) and requiring eight or more red blood cell (RBC) transfusions a year and iron chelation therapy. Younger patients who are at risk of transfusional iron overload but who have not yet initiated iron chelation therapy will be eligible.
• Adequate physical function as measured by the following criteria:
• 1. Cardiac: Asymptomatic or, if symptomatic, then left ventricular ejection fraction at rest must be greater than 40% and must improve with exercise, or shortening fraction greater than 26%
• 2. Hepatic: Less than 5 times the clinical baseline of AST and less than 2.5 times the clinical baseline mg/dL of total serum bilirubin (clinical baseline is determined from the mean of the four most recent test results)
• 3. Renal: Serum creatinine within normal range for age or if serum creatinine is outside normal range for age then renal function (creatinine clearance or GFR) greater than 50% of the lower limit of normal (LLN) for age
• 4. Pulmonary: Asymptomatic, or, if symptomatic, DLCO, FEV1, FEC (diffusion capacity) greater than 45% of predicted (corrected for hemoglobin); if unable to obtain PFT, oxygen saturation greater than 85% on room air