Nonmyeloablative HLA-Matched Sibling Allogeneic Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Phenotype

• Autores: Matthew M. Hsieh, Courtney D. Fitzhugh, Patrick Weitzel, Mary E. Link
• Localización: JAMA: the journal of the American Medical Association, ISSN 0098-7484, Vol. 312, Nº. 1, 2014, págs. 48-56
• Idioma: inglés
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• Resumen

  • Importance Myeloablative allogeneic hematopoietic stem cell transplantation (HSCT) is curative for children with severe sickle cell disease, but toxicity may be prohibitive for adults. Nonmyeloablative transplantation has been attempted with degrees of preparative regimen intensity, but graft rejection and graft-vs-host disease remain significant.

    Objective To determine the efficacy, safety, and outcome on end-organ function with this low-intensity regimen for sickle cell phenotype with or without thalassemia.