title: The ethics of a proposed study of hematopoietic stem cell transplant for children with "less severe" sickle cell disease.
abstract: Hematopoietic stem cell transplant (HSCT) is the only cure for sickle cell disease (SCD). HSCT using an HLA-identical sibling donor is currently an acceptable treatment option for children with severe SCD, with expected HSCT survival >95% and event-free survival >85%. HSCT for children with less severe SCD (children who have not yet suffered overt disease complications or only had mild problems) is controversial. It is important to consider the ethical issues of a proposed study comparing HLA-identical sibling HSCT to best supportive care for children with less severe SCD. In evaluating the principles of nonmaleficence, respect for individual autonomy, and justice, we conclude that a study of HLA-identical sibling HSCT for all children with SCD, particularly hemoglobin SS and Sβ(0)-thalassemia disease, is ethically sound. Future work should explore the implementation of a large trial to help determine whether HSCT is a beneficial treatment of children with less severe SCD.
authors: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University, Atlanta, GA; and.
authors: Robert S
authors: http://orcid.org/0000-0003-4277-7523
authors: RS
authors: Nickel
authors: Laboratory Medicine, Yale University, New Haven, CT.
authors: Jeanne E
authors: JE
authors: Hendrickson
authors: Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University, Atlanta, GA; and.
authors: Ann E
authors: AE
authors: Haight
mesh_terms: D000755:Anemia, Sickle Cell / Q000150:complications / Q000401:mortality / Q000628:therapy*; D002648:Child; D002986:Clinical Trials as Topic / Q000941:ethics; D006086:Graft vs Host Disease / Q000209:etiology; D006680:HLA Antigens; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000941:ethics*; D006801:Humans; D019520:Living Donors / Q000941:ethics; D012307:Risk Factors; D035781:Siblings
chemical_list: D006680:HLA Antigens