{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,9,13]],"date-time":"2025-09-13T15:59:52Z","timestamp":1757779192285},"reference-count":35,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2015,4,16]],"date-time":"2015-04-16T00:00:00Z","timestamp":1429142400000},"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":[[2015,6]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p><jats:styled-content style=\"fixed-case\">HSCT<\/jats:styled-content> is the only curative treatment for <jats:styled-content style=\"fixed-case\">MIOP<\/jats:styled-content>. We prospectively investigated the outcome of <jats:styled-content style=\"fixed-case\">HSCT<\/jats:styled-content> using intravenous busulfan\u2010based conditioning regimen from 2008 to 2013. Nineteen patients (median age\u00a0=\u00a017\u00a0months) underwent transplantation from <jats:styled-content style=\"fixed-case\">HLA<\/jats:styled-content>\u2010matched related donors (n\u00a0=\u00a014), <jats:styled-content style=\"fixed-case\">HLA<\/jats:styled-content>\u2010haploidentical related donors (n\u00a0=\u00a02), partially matched cord blood donors (n\u00a0=\u00a02), and <jats:styled-content style=\"fixed-case\">HLA<\/jats:styled-content>\u2010matched unrelated donor (n\u00a0=\u00a01). Bone marrow (n\u00a0=\u00a09), peripheral blood (n\u00a0=\u00a08), and cord blood (n\u00a0=\u00a02) were used as stem cell sources. All but one patient demonstrated primary engraftment. Two patients experienced secondary graft failure. During the follow\u2010up period, three patients showed mixed chimerism (45%, 45%, and 70% of donor cells were engrafted in each one of these patients) but are disease free. Two\u2010yr <jats:styled-content style=\"fixed-case\">OS<\/jats:styled-content> and <jats:styled-content style=\"fixed-case\">DFS<\/jats:styled-content> were 84.2% and 73.7%, respectively. Improvement of visual acuity and partial reversal of mild conductive hearing loss occurred in two and four patients, respectively. The causes of death among three patients were infection, <jats:styled-content style=\"fixed-case\">G<\/jats:styled-content>v<jats:styled-content style=\"fixed-case\">HD<\/jats:styled-content>, and disease progression. In conclusion, due to major side effects of <jats:styled-content style=\"fixed-case\">MIOP<\/jats:styled-content> such as visual and hearing loss, early treatment using myeloablative conditioning without irradiation <jats:styled-content style=\"fixed-case\">HSCT<\/jats:styled-content> is suggested. The use of an <jats:styled-content style=\"fixed-case\">HLA<\/jats:styled-content>\u2010matched related donor seems to be highly successful in this regard. Also, according to results of our study, mixed chimerism may be sufficient to resolve symptoms of disease.<\/jats:p>","DOI":"10.1111\/petr.12476","type":"journal-article","created":{"date-parts":[[2015,4,16]],"date-time":"2015-04-16T07:03:12Z","timestamp":1429167792000},"page":"422-427","source":"Crossref","is-referenced-by-count":9,"title":["Non\u2010total body irradiation myeloablative conditioning with intravenous busulfan and cyclophosphamide in hematopoietic stem cell transplantation for malignant infantile osteopetrosis"],"prefix":"10.1111","volume":"19","author":[{"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. 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