Methods: Patients 2-20.99 years of age and who meet SCURT eligibility without an 8/8 HLA matched unrelated donor will be enrolled. Patients will receive hydroxyurea (60mg/kg/day) and azathioprine (3mg/kg/day) from days-59 to-11. Fludarabine (30mg/m 2) will be given for 6 days (day-17 to-13), busulfan (4mg/kg/day) for 4 days (day-12 to-9) and cyclophosphamide for 4 days (day-7 to-4), thiotepa (10mg/kg, day-8), R-ATG (day-5 to-2), TLI (500cGy) followed by FHI T-cell depleted AlloSCT. GVHD prophylaxis will consist of tacrolimus. HPC, Apheresis-CD34-enriched cells will be collected by the CliniMACS (FDA IND 14359) with a target dose of 10.0 x 10 6 CD34+ cells/kg containing 2.0 x 10 5 CD3+ T cells/kg. Results: We have developed a FHI TCD Consortium of five clinical sites, four processing centers, and cores in donor chimerism, anti-HLA antibodies, immunology, cell processing, biostatistics, neuroimaging, neurocognition, quality of life, pulmonary function, pulmonary vascular, radiation, and a patient advocacy group (www.haploscdconsortium.org) (Table 1). Conclusion: We seek to develop a unique opportunity for high-risk SCD patients to be cured of this debilitating disease, potentially improving organ function, quality of life, and neurocognition while providing a universal allogeneic donor source for many more atrisk SCD patients.
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