title: First report of successful stem cell transplantation in a patient with sickle cell hemoglobin D disease.
abstract: Sickle cell hemoglobin D disease is a rare variant of sickle cell disease. Affected patients suffer from episodes of acute exacerbation of clinical course with a wide range of manifestations such as acute chest syndrome, stroke, painful vaso-occlusive crises, acute sequestration crises, joint necrosis, organ failure, infections, and temporary aplastic crises, collectively called sickling crises. Conventional treatments for patients with sickle cell disease include hydroxyurea therapy and prophylactic red blood cell transfusion. However, morbidity and mortality rates remain high with these remedies. In this article, we report hematopoietic stem cell transplantation as an alternative treatment in children with high-risk factors. According to our knowledge and an extensive review of the literature, stem cell transplantation in sickle cell hemoglobin D disease previously has not been reported in any published study and our patient is the first case.
authors.affiliation: Hematology-Oncology and Stem Cell Transplantation Research Center, Tehran University of Medical Sciences, Tehran, Iran.
authors.forename: Amir Ali
authors.initials: AA
authors.lastname: Hamidieh
authors.forename: Mahdi
authors.initials: M
authors.lastname: Jalili
authors.forename: Omid
authors.initials: O
authors.lastname: Khojasteh
authors.forename: Ardeshir
authors.initials: A
authors.lastname: Ghavamzadeh
mesh_terms: D000755:Anemia, Sickle Cell / Q000628:therapy*; D002648:Child; D004247:DNA / Q000235:genetics; D006451:Hemoglobin, Sickle / Q000235:genetics; D006455:Hemoglobins, Abnormal / Q000235:genetics; D006801:Humans; D008297:Male; D009154:Mutation / Q000235:genetics; D033581:Stem Cell Transplantation; D016896:Treatment Outcome
chemical_list: D006451:Hemoglobin, Sickle; D006455:Hemoglobins, Abnormal; C032001:hemoglobin D; D004247:DNA