Sickle cell disease (SCD) is an autosomal recessive disorder associated with cerebral vasculopathy, stroke, acute chest syndrome, pulmonary hypertension and/or frequent vaso-occlusive crises, and a high risk of early mortality.1,2 Studies have reported 90% to 100% event-free survival (EFS) following human leukocyte antigen matched sibling allogeneic stem cell transplant.3,4 Reported results have used unrelated allogeneic donor sources; however, a paucity of unrelated matched donors and a higher than expected rate of graft failure and chronic graft-vs-host disease (GVHD) were notable disadvantages.5 The CD34+ enrichment and mononuclear cell (MNC) addback (2 × 105 CD3 cells/kg of recipient body weight) have been reported following matched unrelated donor transplant that resulted in 100% engraftment and a low cumulative incidence of acute GVHD and chronic GVHD.6
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Cairo MS, Talano J, Moore TB, et al. Familial Haploidentical Stem Cell Transplant in Children and Adolescents With High-Risk Sickle Cell Disease: A Phase 2 Clinical Trial. JAMA Pediatr. 2020;174(2):195–197. doi:10.1001/jamapediatrics.2019.4715
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