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The Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Inherited Diseases Is Influenced by HLA Match, Year of Transplantation, and Immunized Female Donor
Karolinska Inst, Karolinska Univ Hosp, Translat Cell Therapy Res, Stockholm, Sweden;Karolinska Inst, Karolinska Univ Hosp, Dept Clin Sci Intervent & Technol, Stockholm, Sweden.
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Medical Sciences. Karolinska Inst, Karolinska Univ Hosp, Dept Oncol & Pathol, Stockholm, Sweden;Uppsala Univ, Dept Med Sci, Uppsala, Sweden;Uppsala Univ Hosp, KFUE, Uppsala, Sweden.
Karolinska Inst, Karolinska Univ Hosp, Dept Clin Sci Intervent & Technol, Stockholm, Sweden;Karolinska Inst, Karolinska Univ Hosp, Dept Pediat, Stockholm, Sweden.
Karolinska Inst, Karolinska Univ Hosp, Translat Cell Therapy Res, Stockholm, Sweden;Karolinska Inst, Karolinska Univ Hosp, Dept Clin Sci Intervent & Technol, Stockholm, Sweden.
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2019 (English)In: Transplantation, ISSN 0041-1337, E-ISSN 1534-6080, Vol. 103, no 6, p. 1247-1252Article in journal (Refereed) Published
Abstract [en]

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.

Place, publisher, year, edition, pages
LIPPINCOTT WILLIAMS & WILKINS , 2019. Vol. 103, no 6, p. 1247-1252
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Pediatrics
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URN: urn:nbn:se:uu:diva-393532DOI: 10.1097/TP.0000000000002481ISI: 000480682800036PubMedID: 30300286OAI: oai:DiVA.org:uu-393532DiVA, id: diva2:1354069
Funder
Swedish Research Council, K2014-64X-05971-34-4Available from: 2019-09-24 Created: 2019-09-24 Last updated: 2019-09-24Bibliographically approved

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