Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease

Prior treatment

• Karnofsky/Lansky (age appropriate) Performance Score \<50% (hemiplegia alone secondary to a previous stroke is not an exclusion)
• Previously received a HSCT

Other illness

• SCD Patients with documented uncontrolled infection

Other

• Females who are pregnant or breast-feeding
• SCD patients who have an unaffected HLA matched family donor willing to proceed to donation
• Demonstrated lack of compliance with medical care.
• Clinically significant fibrosis or cirrhosis of the liver

• Homozygous Hemoglobin S Disease, or Hemoglobin S Beta0/+ thalassemia
• Patients must demonstrate one or more of the following Sickle Cell Disease Complications
  • Clinically significant neurologic event (stroke) or any neurologic deficit lasting \>24 hours that is accompanied by an infarct on cerebral MRI
  • Minimum of two episodes of acute chest syndrome.
  • Recurrent painful events (at least 3 in the 2 years prior to enrollment).
  • Abnormal TCD study requiring starting on chronic transfusion therapy.
  • 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 patients.