Allogeneic hematopoietic stem cell transplantation for nonmalignant hematologic disorders using chemotherapy-only cytoreductive regimens and T-cell-depleted grafts from human leukocyte antigen-matched or - mismatched donors Journal Article

   

Authors:	Mussetti, A.; Kernan, N. A.; Prockop, S. E.; Scaradavou, A.; Lehrman, R.; Ruggiero, J. M.; Curran, K.; Kobos, R.; O'Reilly, R.; Boulad, F.
Article Title:	Allogeneic hematopoietic stem cell transplantation for nonmalignant hematologic disorders using chemotherapy-only cytoreductive regimens and T-cell-depleted grafts from human leukocyte antigen-matched or - mismatched donors
Abstract:	Nonmalignant hematologic disorders (NMHD) of childhood comprise a variety of disorders, including acquired severe aplastic anemia and inherited marrow failure syndromes. Patients with high-risk NMHD without matched related donors fare poorly with allogeneic hematopoietic alternative donor stem cell transplantation (allo-HSCT) and are at high risk for developing graft-versus-host disease following unmodified grafts. The authors retrospectively analyzed data on 18 patients affected by NMHD, lacking a human leukocyte antigen (HLA)-identical sibling donor, who underwent an alternative donor allo-HSCT at their institution between April 2005 and May 2013. Fifty percent of the patients had received prior immunosuppressive therapy, 72% had a history of infections, and 56% were transfusion dependent at the time of transplant. Cytoreduction included a combination of 3 of 5 agents: fludarabine, melphalan, thiotepa, busulfan, and cyclophosphamide. Grafts were T-cell depleted. All evaluable patients engrafted. Five died of transplant complications. The cumulative incidence of graft-versus-host disease was 6%. No patient had recurrence of disease. Five-year overall survival was 77%. Age at transplant <6 years was strongly associated with better survival. Based on these results, transplant with chemotherapy-only cytoreductive regimens and T-cell-depleted stem cell transplants could be recommended for patients with high-risk NMHD, especially at a younger age. © 2016 Taylor & Francis Group, LLC.
Keywords:	chemotherapy; bone marrow transplant; bone marrow failure; anemias
Journal Title:	Pediatric Hematology and Oncology
Volume:	33
Issue:	6
ISSN:	0888-0018
Publisher:	Taylor & Francis Group
Date Published:	2016-01-01
Start Page:	347
End Page:	358
Language:	English
DOI:	10.1080/08880018.2016.1204399
PROVIDER:	scopus
PUBMED:	27715384
PMCID:	PMC5175271
DOI/URL:	https://www.scopus.com/inward/record.uri?eid=2-s2.0-84990232858&partnerID=40&md5=cf69206ecc3b6801c52004253ce8b059
Notes:	Article -- Export Date: 6 December 2016 -- Source: Scopus

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