6 United States sites
2 to 20 Years
– Homozygous Hemoglobin S Disease, or Hemoglobin S Beta0/+ thalassemia
– Patients must demonstrate one or more of the following Sickle Cell Disease
1. Clinically significant neurologic event (stroke) or any neurologic deficit
lasting >24 hours that is accompanied by an infarct on cerebral MRI
2. Minimum of two episodes of acute chest syndrome.
3. Recurrent painful events (at least 3 in the 2 years prior to enrollment).
4. Abnormal TCD study requiring starting on chronic transfusion therapy.
5. At least one silent infarct lesion on a MRI scan of the head.
– A familial haploidentical donor without homozygous sickle cell disease
– Adequate organ function (renal, liver, cardiac and pulmonary function)
– Karnofsky or Lansky (age appropriate) Performance Score ≥50%
– Liver biopsy is optional to assess for iron overload in chronically transfused
– Females who are pregnant or breast-feeding
– SCD Patients with documented uncontrolled infection
– SCD patients who have an unaffected HLA matched family donor willing to proceed to
– Karnofsky/Lansky (age appropriate) Performance Score <50% (hemiplegia alone secondary to a previous stroke is not an exclusion) - Demonstrated lack of compliance with medical care. - Clinically significant fibrosis or cirrhosis of the liver - Previously received a HSCT