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Biol Blood Marrow Transplant. 2016 Feb;22(2):207-211. doi: 10.1016/j.bbmt.2015.10.017. Epub 2015 Oct 21.

Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease.

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Blood and Marrow Transplant Program, University of California (San Francisco) Benioff Children's Hospital, Oakland, California. Electronic address:
Division of Hematology and Oncology, University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania.
Blood and Marrow Transplant Program, Duke University Medical Center, Durham, North Carolina.
Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Emory University, Atlanta, Georgia.
Center for Cellular Therapies and Research, AABB, Bethesda, Maryland.
Blood and Marrow Transplant Program, Ottawa Hospital Research Institute at University of Ottawa, Ottawa, Ontario, Canada.
Biostatistics and Computational Biology, Dana-Farber Cancer Institute, Boston, Massachusetts.
University of Colorado School of Medicine, Aurora, Colorado.
National Marrow Donor Program, Minneapolis, Minnesota.
Division of Pediatric Hematology/Oncology, University of Michigan, School of Medicine, Department of Pediatrics, Blood and Marrow Transplant Program, Ann Arbor, Michigan.
Fred Hutchinson Cancer Research Center and the University of Washington School of Medicine, Seattle, Washington.


Although a number of published trials exist of HLA-identical sibling hematopoietic cell transplantation (HCT) for sickle cell disease (SCD) that span 2 decades, when and for whom this therapy should be pursued is a subject of debate. Assessments of the risks of transplant-related complications that include infertility and debilitating graft-versus-host disease and long-term quality of life after successful HCT are difficult to perform without prospective trials in transplant and nontransplant cohorts. However, it is possible to assess the risk of mortality and to compare published rates of survival in individuals with SCD treated and not treated by HCT. In this brief review, projections about mortality risk based on recent published reports are reviewed and summarized. The published data show overall survival and event-free survival rates of 95% and 92%, respectively, in children treated by HLA-identical sibling HCT. The overall survival rates in the Center for International Blood and Marrow Transplant Research (N = 412) and European Blood and Marrow Transplant (N = 487) registries were 91% and 95%, respectively. These results provide broad support for the therapeutic value of HLA-identical sibling HCT for children with SCD and serve as the basis for a strong recommendation in favor of the option of HCT when a suitable donor is available. The experience of HLA-identical sibling HCT in adults with SCD is limited but appears to be similar to results in children. These preliminary observations, however, warrant further investigation.


Children; HLA-identical sibling; Hematopoietic cell transplant; Risks; Sickle cell anemia; Transplant-related complications

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