When there is no match,the game is not over: Alternative donor options for hematopoietic stem cell transplantation in sickle cell disease |
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| Affiliation: | 1. Sickle Cell Branch, National Heart, Lung, and Blood Institute, Bethesda, MD;2. Department of Hematology, Washington Hospital Center/Georgetown University, Washington, DC;3. Division of Blood and Marrow Transplantation, Children’s National Health System, Washington, DC;1. Hematología-Oncología Pediátricas, Hospital General Universitario Gregorio Marañón, Madrid, Spain;2. Radiología Infantil, Hospital General Universitario Gregorio Marañón, Madrid, Spain;3. Servicios de Hematología y Hemoterapia, Hospital General Universitario Gregorio Marañón, Madrid, Spain;1. Department of Pediatrics, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH;2. Department of Medicine, University of Cincinnati, Cincinnati, OH;3. Global Health Center, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH;4. Department of Epidemiology and Public Health, University of Texas Health Sciences Center, Houston, TX;5. University of Cincinnati College of Medicine, Cincinnati, OH;1. Division of Hematology/Oncology, Boston Children’s Hospital, Boston, MA;2. Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA;3. Department of Pediatrics, Harvard Medical School, Boston, MA;1. Sidney Kimmel Cancer Center at Johns Hopkins, Johns Hopkins University, Baltimore, MD; and;2. Vanderbilt-Meharry Sickle Cell Disease Center of Excellence, Vanderbilt University Medical Center, Nashville, TN |
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| Abstract: | Many patients with sickle cell disease experience severe morbidity and early mortality. The only curative option remains hematopoietic stem cell transplantation. Although HLA-matched sibling transplantation has been very successful for adults and children, the vast majority of patients with sickle cell disease do not have an HLA-matched sibling. Alternative donor options include haploidentical, unrelated umbilical cord blood, and matched unrelated donor transplantation. This report summarizes major alternative donor transplantation studies reported to date and ongoing and upcoming clinical trials. We conclude that when there is no HLA-match, all these approaches should be systematically considered before ruling out the option of hematopoietic stem cell transplantation. |
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| Keywords: | Alternative donor transplant Haploidentical Umbilical cord blood Matched unrelated donor Sickle cell disease |
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