{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,7,30]],"date-time":"2025-07-30T12:32:56Z","timestamp":1753878776410,"version":"3.41.2"},"reference-count":21,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2017,6,14]],"date-time":"2017-06-14T00:00:00Z","timestamp":1497398400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Transplantation"],"published-print":{"date-parts":[[2017,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Maroteaux\u2010Lamy syndrome is a rare inherited lysosomal storage disorder with a progressive course. <jats:styled-content style=\"fixed-case\">HSCT<\/jats:styled-content> is a curable option for treatment in these patients. The following report describes our experience in <jats:styled-content style=\"fixed-case\">HSCT<\/jats:styled-content> for three patients with Maroteaux\u2010Lamy syndrome using non\u2010sibling donors. All of the patients received the same myeloablative regimen consisting of intravenous busulfan, cyclophosphamide, and rabbit antithymocyte globulin. Patients underwent <jats:styled-content style=\"fixed-case\">HSCT<\/jats:styled-content> from haploidentical other\u2010related (n=1), full\u2010matched other\u2010related (n=1), and one\u2010locus\u2010mismatched unrelated donor. Stem cell sources included bone marrow (n=1), peripheral blood (n=1), and cord blood (n=1). Currently, two patients who received transplant from other\u2010related donors showed full engraftment and regression of the symptoms of the disease, while for the patient with unrelated cord blood donor, graft failure resulted in progression of the disease and death. The result of our study showed beneficial effects of <jats:styled-content style=\"fixed-case\">HSCT<\/jats:styled-content> even from heterozygote donor. Due to rarity of the disease, future multicenter studies are recommended to find the best treatment approaches based on the patients\u2019 status.<\/jats:p>","DOI":"10.1111\/petr.12981","type":"journal-article","created":{"date-parts":[[2017,6,15]],"date-time":"2017-06-15T11:19:03Z","timestamp":1497525543000},"source":"Crossref","is-referenced-by-count":10,"title":["Non\u2010sibling hematopoietic stem cell transplantation using myeloablative conditioning regimen in children with Maroteaux\u2010Lamy syndrome: A brief report"],"prefix":"10.1111","volume":"21","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9473-3632","authenticated-orcid":false,"given":"Maryam","family":"Behfar","sequence":"first","affiliation":[{"name":"Hematology\u2010Oncology and Stem Cell Transplantation Research Center Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"S. Sharareh","family":"Dehghani","sequence":"additional","affiliation":[{"name":"Hematology\u2010Oncology and Stem Cell Transplantation Research Center Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tahereh","family":"Rostami","sequence":"additional","affiliation":[{"name":"Hematology\u2010Oncology and Stem Cell Transplantation Research Center Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ardeshir","family":"Ghavamzadeh","sequence":"additional","affiliation":[{"name":"Hematology\u2010Oncology and Stem Cell Transplantation Research Center Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Amir Ali","family":"Hamidieh","sequence":"additional","affiliation":[{"name":"Hematology\u2010Oncology and Stem Cell Transplantation Research Center Tehran University of Medical Sciences  Tehran Iran"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2017,6,14]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-5-5"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/BF00288275"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2006-2184"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1096-7192(03)00007-6"},{"key":"e_1_2_6_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/jcpt.12136"},{"key":"e_1_2_6_7_1","doi-asserted-by":"crossref","unstructured":"PrasadVK KurtzbergJ.Transplant outcomes in mucopolysaccharidoses.Seminars in hematology. 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