Hematopoietic Stem Cell Transplantation for Severe Combined Immunodeficiency (SCID)
نویسندگان
چکیده
منابع مشابه
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Thalassemia is an autosomal recessive disorder associated with defective synthesis of the α- or β-chain of hemoglobin. For β-thalassemia major patients, therapeutic options are either monthly red cell transfusions and chelation therapy or allogeneic stem cell transplant. Stem cell transplant is the only curative approach and success is inversely correlated with the degree of iron overload and h...
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Deficiency of the purine salvage enzyme adenosine deaminase leads to SCID (ADA-SCID). Hematopoietic cell transplantation (HCT) can lead to a permanent cure of SCID; however, little data are available on outcome of HCT for ADA-SCID in particular. In this multicenter retrospective study, we analyzed outcome of HCT in 106 patients with ADA-SCID who received a total of 119 transplants. HCT from mat...
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ژورنال
عنوان ژورنال: Frontiers in Pediatrics
سال: 2019
ISSN: 2296-2360
DOI: 10.3389/fped.2019.00481