Hematopoietic Cell Transplantation for Mucopolysaccharidosis Patients Is Safe and Effective: Results after Implementation of International Guidelines
| العنوان: | Hematopoietic Cell Transplantation for Mucopolysaccharidosis Patients Is Safe and Effective: Results after Implementation of International Guidelines |
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| المؤلفون: | Jean Mercer, Mieke Aldenhoven, Ans T. van der Ploeg, Marc Bierings, Robert Wynn, Birgitta Versluys, Frits A. Wijburg, Peter M. van Hasselt, Simon Jones, Denise Bonney, Jaap Jan Boelens, Roisin E. Borrill, Mary Coussons |
| المساهمون: | Clinical Chemistry, Pediatrics, AGEM - Amsterdam Gastroenterology Endocrinology Metabolism, Paediatric Metabolic Diseases |
| المصدر: | Biology of Blood and Marrow Transplantation, 21(6), 1106-1109. Elsevier Inc. Biology of Blood and Marrow Transplantation, 21(6), 1106. Elsevier Inc. Biology of blood and marrow transplantation, 21(6), 1106-1109. Elsevier Inc. |
| بيانات النشر: | American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. |
| مصطلحات موضوعية: | Male, Oncology, Transplantation Conditioning, Mucopolysaccharidosis, Graft vs Host Disease, DONORS, ENZYME REPLACEMENT THERAPY, DISEASE, Recurrence, immune system diseases, hemic and lymphatic diseases, Child, Non-U.S. Gov't, Hurler syndrome, OUTCOMES, Hematopoietic cell transplantation, Research Support, Non-U.S. Gov't, Hematopoietic Stem Cell Transplantation, Neurodegenerative Diseases, Hematology, Enzyme replacement therapy, Prognosis, Fludarabine, Multicenter Study, surgical procedures, operative, Child, Preschool, Cord blood, Acute Disease, Practice Guidelines as Topic, Female, Cord Blood Stem Cell Transplantation, Unrelated Donors, Vidarabine, medicine.drug, medicine.medical_specialty, Cyclophosphamide, HURLERS-SYNDROME, RISK-FACTOR ANALYSIS, Research Support, Internal medicine, Journal Article, medicine, Humans, Transplantation, Homologous, Busulfan, Transplantation, business.industry, Siblings, Infant, Newborn, GRAFT, Infant, Mucopolysaccharidoses, Myeloablative Agonists, medicine.disease, Survival Analysis, Surgery, MARROW, Chronic Disease, business, Follow-Up Studies |
| الوصف: | Allogeneic hematopoietic cell transplantation, (HCT) is the only treatment able to prevent progressive neurodegenerative disease in a selected group of mucopolysaccharidosis (MPS) disorders. However, its use was historically limited by the high risk of graft failure and transplantation-related morbidity and mortality. Therefore; since 2005 new international HCT guidelines for MPS disorders were proposed. The survival and graft outcomes of MPS patients receiving HCT according to these guidelines in 2 European centers of expertise were,evaluated. Two consecutive conditioning regimens were used, busulfan/cyclophosphamide or, fludarabine/busulfan-based, both with exposure-targeted i.v. busulfan. A noncarrier matched sibling donor (MSD), matched unrelated cord blood (UCB), or matched unrelated donor (MUD) were considered to be preferred donors. If not available, a mismatched UCB donor was used. Participants were 62 MPS patients (56 MPS type I Hurler, 2 MPS type II, 2 MPS type III, and 2 MPS type VI) receiving HCT at median age 13.5 months (range, 3 to 44). Forty-one patients received a UCB donor, 17 MSD, and 4 MUD. High overall survival (95.2%) and event-free survival (90.3%) were achieved with only low toxicity: 13.3% acute graft-versus-host disease aGVHD) grades II to IV and 14.8% chronic GVHD (1.9% extensive). A mismatched donor predicted for lower event-free survival (P = .04). A higher age at HCT was a predictor for both aGVHD (P = .001) and chronic GVHD (P = .01). The use of a mismatched donor was a predictor for aGVHD (P = .01). Higher rates of full-donor chimerism were achieved in successfully transplanted UCB recipients compared with MSD/MUD (P = .002). If complying with the international HCT guidelines, HCT in MPS patients results in high safety and efficacy. This allows extension of HCT to more attenuated MPS types. Because a younger age at HCT is associated with reduction of HCT-related toxicity, newborn screening may further increase safety. (C) 2015 American Society for Blood and Marrow Transplantation. |
| وصف الملف: | image/pdf |
| اللغة: | English |
| تدمد: | 1083-8791 |
| DOI: | 10.1016/j.bbmt.2015.02.011 |
| الوصول الحر: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6b1f827b2a3d7846ceef730bf40c6f11Test |
| حقوق: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....6b1f827b2a3d7846ceef730bf40c6f11 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 10838791 |
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| DOI: | 10.1016/j.bbmt.2015.02.011 |