NYMC Faculty Publications
Familial Haploidentical Stem Cell Transplant in Children and Adolescents With High-Risk Sickle Cell Disease: A Phase 2 Clinical Trial
DOI
10.1001/jamapediatrics.2019.4715
Journal Title
JAMA Pediatrics
First Page
195
Last Page
197
Document Type
Article
Publication Date
2-2020
Department
Pediatrics
Second Department
Health Behavior and Community Health
Third Department
Biostatistics
Abstract
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
Recommended Citation
Cairo, M. S., Talano, J., Moore, T. B., Shi, Q., Weinberg, R. S., Grossman, B., & Shenoy, S. (2020). Familial Haploidentical Stem Cell Transplant in Children and Adolescents With High-Risk Sickle Cell Disease: A Phase 2 Clinical Trial. JAMA Pediatrics, 174 (2), 195-197. https://doi.org/10.1001/jamapediatrics.2019.4715
