{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,7,30]],"date-time":"2025-07-30T10:04:39Z","timestamp":1753869879277,"version":"3.41.2"},"reference-count":22,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2018,3,7]],"date-time":"2018-03-07T00:00:00Z","timestamp":1520380800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81370603"],"award-info":[{"award-number":["81370603"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2018,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>The objective of this study was to evaluate the feasibility of a modified conditioning regimen for the treatment of patients with \u03b2<jats:bold>\u2010<\/jats:bold>thalassaemia major (TM), using unrelated donor peripheral blood stem cell transplantation (UD\u2010PBSCT).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>A modified conditioning regimen based on intravenous busulfan, cyclophosphamide, fludarabine, and antithymocyte globulin was performed in 50 consecutive childhood patients with \u03b2<jats:bold>\u2010<\/jats:bold>TM and a median age of 4.6 years (range, 2\u201312 years). According to Pesaro's classification, three classes of risk are identified using the criteria of degree of hepatomegaly, portal fibrosis, and quality of the chelation treatment. Patients with three adverse criteria constituted class III, none of the adverse criteria constituted class I, and one or two of the adverse criteria formed class II. Ten patients were class I, 36 class II, and four class III. All patients were transplanted with UDs containing 37 of 10\/10 human leukocyte antigen (HLA)\u2010matched pairs, 11 of 9\/10 matched pairs, and two of 8\/10 matched pairs. The median follow\u2010up was 36 months (range, 9\u201396 months).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>All patients successfully achieved engraftment, two of whom developed persistent\u00a0thrombocytopaenia. The incidence of acute graft\u2010versus\u2010host disease (aGVHD) grade III\u2010IV and chronic graft\u2010versus\u2010host disease (cGVHD) were 12% and 8%, respectively. However, 8.3% of HLA\u2010matched and 15.4% of HLA\u2010mismatched patients developed aGVHD. The incidence of severe bacterial infections and fungal pneumonia was 12% and 20%, respectively. The 3\u2010year overall survival, disease\u2010free survival, graft rejection, and transplant\u2010related mortality were 94%, 92%, 2%, and 6%, respectively.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>This modified conditioning protocol effectively improved outcomes of UD\u2010PBSCT for patients with \u03b2\u2010TM.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.27026","type":"journal-article","created":{"date-parts":[[2018,3,7]],"date-time":"2018-03-07T10:22:41Z","timestamp":1520418161000},"source":"Crossref","is-referenced-by-count":7,"title":["Modified conditioning regimen improves outcomes of unrelated donor peripheral blood stem cell transplantation for \u03b2\u2010thalassaemia major patients"],"prefix":"10.1002","volume":"65","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1854-7840","authenticated-orcid":false,"given":"Ke","family":"Huang","sequence":"first","affiliation":[{"name":"Department of Pediatrics Sun Yat\u2010sen Memorial Hospital Sun Yat\u2010sen University  Guangzhou People's Republic of China"},{"name":"Key Laboratory of Malignant Tumor Gene Regulation and Target Therapy of Guangdong Higher Education Institutes Sun Yat\u2010sen University  Guangzhou People's Republic of China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dun\u2010Hua","family":"Zhou","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Sun Yat\u2010sen Memorial Hospital Sun Yat\u2010sen University  Guangzhou People's Republic of China"},{"name":"Key Laboratory of Malignant Tumor Gene Regulation and Target Therapy of Guangdong Higher Education Institutes Sun Yat\u2010sen University  Guangzhou People's Republic of China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yang","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Sun Yat\u2010sen Memorial Hospital Sun Yat\u2010sen University  Guangzhou People's Republic of China"},{"name":"Key Laboratory of 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Institutes Sun Yat\u2010sen University  Guangzhou People's Republic of China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Chun","family":"Chen","sequence":"additional","affiliation":[{"name":"Department of Pediatrics The Seventh Affiliated Hospital of Sun Yat\u2010sen University  Shenzhen People's Republic of China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hong\u2010Man","family":"Xue","sequence":"additional","affiliation":[{"name":"Department of Pediatrics The Seventh Affiliated Hospital of Sun Yat\u2010sen University  Shenzhen People's Republic of China"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hai\u2010Xia","family":"Guo","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Southern Medical University Nan fang Hospital  Guangzhou People's Republic of 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