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• W2897953397 abstract "Background. For many inborn errors of metabolism (IEM), allogeneic hematopoietic stem cell transplantation (HSCT) is the only cure. Methods. We report the outcome in 160 patients with inherited diseases, who were treated with HSCT in 3 decades. Median age was 3 years (range 0.1–63). Grafts were from matched related donors (MRDs, 56), matched unrelated donors (MUDs, 66), or HLA-mismatched donors (38). Results. Graft failure (GF) occurred in 26 patients (16%), severe acute graft-versus-host disease (GVHD) in 9 (6%), and chronic GVHD in 23 (12%). Ten-year survival was 64% before the year 2000 and 86% after that ( P = 0.01). Ten-year survival for MRD grafts was 90%, as opposed to 79% for MUD grafts and 56% for HLA-mismatched grafts ( P = 0.03). In multivariate analysis, GF was associated with having an HLA-mismatched donor ( P < 0.05) or MUD ( P = 0.015) and with reduced-intensity conditioning ( P < 0.01). Death was associated with year of transplant ( P = 0.015), having an HLA-mismatched donor ( P < 0.001), and being a male recipient from an immune female donor ( P = 0.05). Conclusions. The outcome after HSCT for IEM depends on HLA match, year and immune female donor." @default.
• W2897953397 created "2018-10-26" @default.
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• W2897953397 date "2019-06-01" @default.
• W2897953397 modified "2023-10-04" @default.
• W2897953397 title "The Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Inherited Diseases Is Influenced by HLA Match, Year of Transplantation, and Immunized Female Donor" @default.
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• W2897953397 doi "https://doi.org/10.1097/tp.0000000000002481" @default.
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