Kothari, AlokNgwube, AlexanderHayashi, RobertMurray, LisaDavis, JeffreyHaut, PaulLoechelt, Brett J.Shenoy, Shalini2015-12-212015-12-212015-07Kothari, A., Ngwube, A., Hayashi, R., Murray, L., Davis, J., Haut, P., … Shenoy, S. (2015). Hematopoietic Cell Transplantation Using Reduced-Intensity Conditioning Is Successful in Children with Hematologic Cytopenias of Genetic Origin. Biology of Blood and Marrow Transplantation, 21(7), 1321–1325. http://doi.org/10.1016/j.bbmt.2015.03.0191083-8791https://hdl.handle.net/1805/7787Genetically derived hematologic cytopenias are a rare heterogeneous group of disorders. Allogeneic hematopoietic cell transplantation (HCT) is curative but offset by organ toxicities from the preparative regimen, graft rejection, graft-versus-host disease (GVHD), or mortality. Because of these possibilities, consideration of HCT can be delayed, especially in the unrelated donor setting. We report a prospective multicenter trial of reduced-intensity conditioning (RIC) with alemtuzumab, fludarabine, and melphalan and HCT in 11 children with marrow failure of genetic origin (excluding Fanconi anemia) using the best available donor source (82% from unrelated donors). The median age at transplantation was 23 months (range, 2 months to 14 years). The median times to neutrophil (>500 × 106/L) and platelet (>50 × 109/L) engraftment were 13 (range, 12 to 24) and 30 (range, 7 to 55) days, respectively. The day +100 probability of grade II to IV acute GVHD and the 1-year probability of limited and extensive GVHD were 9% and 27%, respectively. The probability of 5-year overall and event-free survival was 82%; 9 patients were alive with normal blood counts at last follow-up and all were successfully off systemic immunosuppression. In patients with genetically derived severe hematologic cytopenias, allogeneic HCT with this RIC regimen was successful in achieving a cure. This experience supports consideration of HCT early in such patients even in the absence of suitable related donors.en-USPublisher PolicyCongenital hematologic cytopeniasReduced-intensity conditioningHematopoietic cell transplantationArticle