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Research Letter
December 9, 2019

Familial Haploidentical Stem Cell Transplant in Children and Adolescents With High-Risk Sickle Cell Disease: A Phase 2 Clinical Trial

Author Affiliations
  • 1Department of Pediatrics, New York Medical College, Valhalla
  • 2Department of Medicine, New York Medical College, Valhalla
  • 3Department of Pathology, New York Medical College, Valhalla
  • 4Department of Microbiology & Immunology, New York Medical College, Valhalla
  • 5Department of Cell Biology & Anatomy, New York Medical College, Valhalla
  • 6Department of Pediatrics, Medical College of Wisconsin, Milwaukee
  • 7Department of Pediatrics, University of California, Los Angeles, Los Angeles
  • 8Department of Epidemiology & Community Health, New York Medical College, Valhalla
  • 9The New York Blood Center, New York
  • 10Department of Pathology & Immunology, Washington University in St Louis, St Louis, Missouri
  • 11Department of Pediatrics, Washington University in St Louis, St Louis, Missouri
JAMA Pediatr. 2020;174(2):195-197. doi:10.1001/jamapediatrics.2019.4715

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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