Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease
St. Jude Children's Research Hospital
Summary
The purpose of this study it to evaluate a reduced toxicity conditioning regimen for haploidentical donor HCT followed by a GVHD prophylaxis regimen comprising of post-transplant cyclophosphamide, sirolimus and abatacept with the goal to improve the GVHD-free rejection-free survival (GRFS) to greater than 90% after haploidentical donor HCT in children and young adults with SCD. Primary Objective: \- To assess the GVHD-free and rejection free survival (GRFS) after haploidentical donor HCT in children and young adults with SCD. Secondary Objectives: * Assess the overall survival (OS) and disease-free survival (DFS) after haploidentical donor HCT for SCD. * Estimate incidence and severity of acute and chronic GVHD after haploidentical donor HCT for SCD. * Assess the neutrophil and platelet engraftment kinetics after haploidentical donor HCT for SCD.
Eligibility
- Age range
- Up to 22 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: Transplant Recipient * Age less than or equal to 22 years. * Patients without a suitable HLA-matched sibling donor but with a suitable single haplotype matched (≥ 3 of 6) family member donor. Potential donors do not need to undergo eligibility determination prior to the recipients enrolling on the study. As long as a potential donor is identified and willing to donate hematopoietic progenitor cells, recipients can enroll on the study. * Patients with SCD (any genotype) who meet any ONE of the following criteria: * History of an abnormal transcranial Doppler measurement de…
Interventions
- DrugCyclophosphamide 50mg
IV
- DrugAbatacept
IV
- DrugAlemtuzumab
IV
- DrugHydroxyurea
IV
- DrugThiotepa
IV
- DrugFilgrastim
IV
- DrugSirolimus
IV
- DrugAzathioprine
Location
- St Jude Children's Research HospitalMemphis, Tennessee