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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.

Authors
  • Mussetti, Alberto1
  • Kernan, Nancy A2
  • Prockop, Susan E2
  • Scaradavou, Andromachi2
  • Lehrman, Rachel2
  • Ruggiero, Julianne M2
  • Curran, Kevin2
  • Kobos, Rachel2
  • O'Reilly, Richard2
  • Boulad, Farid2, 3
  • 1 a Dipartimento di Ematologia e Onco-Ematologia Pediatrica , Fondazione IRCCS Istituto Nazionale dei Tumori , Milan , Italy. , (Italy)
  • 2 b Bone Marrow Transplant Service, Department of Pediatrics , Memorial Sloan Kettering Cancer Center , New York , New York , USA.
  • 3 c Division of Pediatric Hematology/Oncology , New York Presbyterian Hospital, Weill Cornell Medical College , New York , New York , USA.
Type
Published Article
Journal
Pediatric Hematology and Oncology
Publisher
Informa UK (Taylor & Francis)
Publication Date
September 2016
Volume
33
Issue
6
Pages
347–358
Identifiers
PMID: 27715384
Source
Medline
Keywords
License
Unknown

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.

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