Familial haploidentical stem cell transplant in children and adolescents with high-risk sickle cell disease: A phase 2 clinical trial
JAMA Pediatrics Feb 07, 2020
Cairo MS, Talano JA, Moore TB, et al. - Among children and adolescents with high-risk sickle cell disease (SCD), researchers sought to analyze familial haploidentical stem cell transplant. SCD is an autosomal recessive disorder linked to cerebral vasculopathy, stroke, acute chest syndrome, pulmonary hypertension and/or frequent vaso-occlusive crises, and a high risk of early death. Researches have recorded 90% to 100% event-free survival following a sibling allogeneic stem cell transplant matched to human leukocyte antigen. Reported outcomes have used unrelated allogeneic donor sources, however, a lack of unrelated matched donors and a higher than expected rate of graft failure and chronic graft-vs-host disease (GVHD) were notable disadvantages. The CD34+ enrichment and mononuclear cell addback (2 × 105 CD3 cells/kg of recipient body weight) were reported after matched unrelated donor transplants resulting in 100% engraftment and low cumulative incidence of acute GVHD and chronic GVHD.
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