{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,2]],"date-time":"2026-09-02T00:14:47Z","timestamp":1788308087777,"version":"build-2803163510"},"reference-count":33,"publisher":"Wiley","license":[{"start":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T00:00:00Z","timestamp":1788220800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T00:00:00Z","timestamp":1788220800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>\n                    We report the results of an open\u2010label, single\u2010centre phase 4 clinical trial evaluating haploidentical haematopoietic stem cell transplantation (Haplo\u2010HSCT) in patients with transfusion\u2010dependent thalassaemia (TDT). A total of 134 patients with TDT were enrolled; the median age was 8.7\u2009years (interquartile range [IQR], 6.0\u201311.7\u2009years; range, 3\u201320\u2009years). All patients received a uniform conditioning regimen consisting of busulfan, cyclophosphamide, fludarabine and anti\u2010thymocyte globulin (ATG). Graft\u2010versus\u2010host disease (GVHD) prophylaxis included methotrexate (MTX), mycophenolate mofetil (MMF) and tacrolimus. The primary end\u2010point was 2\u2010year overall survival (OS). Median follow\u2010up is 23.4\u2009months (IQR: 11.5\u201338.7; range: 6.1\u201381.5). The 2\u2010year OS, event\u2010free survival (EFS) and graft\u2010versus\u2010host disease\u2010free, relapse\u2010free survival (GRFS) rates were 93.4%, 92.6% and 77.5% respectively. The cumulative incidence rates of aGVHD, grade 2\u20134 aGVHD and grade 3\u20134 aGVHD were 47.8%, 30.9% and 12.5% respectively. Donor\u2010specific antibodies (DSAs) and male\u2010recipient\/female\u2010donor pairing were associated with inferior OS (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.021;\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.010) and EFS (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.040;\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.020). Hepatomegaly (\u22655\u2009cm) was associated with inferior OS (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.037). Collectively, these findings support Haplo\u2010HSCT as a key curative option for TDT patients in the absence of fully matched donors.\n                  <\/jats:p>","DOI":"10.1111\/bjh.70819","type":"journal-article","created":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T23:34:55Z","timestamp":1788305695000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Haploidentical haematopoietic stem cell transplantation for thalassaemia major: A phase\n                    <scp>IV<\/scp>\n                    , open\u2010label, single\u2010centre trial"],"prefix":"10.1111","author":[{"ORCID":"https:\/\/orcid.org\/0009-0004-3529-0455","authenticated-orcid":false,"given":"Xiaoxuan","family":"Lai","sequence":"first","affiliation":[{"name":"The First Affiliated Hospital of Guangxi Medical University  Nanning Guangxi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5531-8006","authenticated-orcid":false,"given":"Rongrong","family":"Liu","sequence":"additional","affiliation":[{"name":"The First Affiliated Hospital of Guangxi Medical University  Nanning Guangxi China"},{"name":"NHC Key Laboratory of Thalassemia Medicine  Nanning Guangxi China"},{"name":"Guangxi Key Laboratory of Thalassemia Research  Nanning Guangxi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2363-0294","authenticated-orcid":false,"given":"Hongwen","family":"Xiao","sequence":"additional","affiliation":[{"name":"The First Affiliated Hospital of Guangxi Medical University  Nanning Guangxi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lianjin","family":"Liu","sequence":"additional","affiliation":[{"name":"The First Affiliated Hospital of Guangxi Medical University  Nanning Guangxi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2197-2047","authenticated-orcid":false,"given":"Zhenbin","family":"Wei","sequence":"additional","affiliation":[{"name":"The First Affiliated Hospital of Guangxi Medical University  Nanning Guangxi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lingling","family":"Shi","sequence":"additional","affiliation":[{"name":"The First Affiliated Hospital of Guangxi Medical University  Nanning Guangxi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Meiqing","family":"Wu","sequence":"additional","affiliation":[{"name":"The First Affiliated Hospital of Guangxi Medical University  Nanning Guangxi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhongming","family":"Zhang","sequence":"additional","affiliation":[{"name":"The First Affiliated Hospital of Guangxi Medical University  Nanning Guangxi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8306-1954","authenticated-orcid":false,"given":"Yongrong","family":"Lai","sequence":"additional","affiliation":[{"name":"The First Affiliated Hospital of Guangxi Medical University  Nanning Guangxi China"},{"name":"NHC Key Laboratory of Thalassemia Medicine  Nanning Guangxi China"},{"name":"Guangxi Key Laboratory of Thalassemia Research  Nanning Guangxi China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,9,1]]},"reference":[{"issue":"6","key":"e_1_2_10_2_1","doi-asserted-by":"crossref","first-page":"1296","DOI":"10.1182\/blood-2009-05-218982","article-title":"Purified T\u2010depleted, CD34+ peripheral blood and bone marrow cell transplantation from haploidentical mother to child with thalassemia","volume":"115","author":"Sodani P","year":"2010","journal-title":"Blood"},{"issue":"20","key":"e_1_2_10_3_1","doi-asserted-by":"crossref","first-page":"1908","DOI":"10.1056\/NEJMra1404415","article-title":"The \u03b1\u2010Thalassemias","volume":"371","author":"Piel FB","year":"2014","journal-title":"N Engl J Med"},{"issue":"8","key":"e_1_2_10_4_1","doi-asserted-by":"crossref","first-page":"727","DOI":"10.1056\/NEJMra2021838","article-title":"\u03b2\u2010Thalassemias","volume":"384","author":"Taher AT","year":"2021","journal-title":"N Engl J Med"},{"issue":"5","key":"e_1_2_10_5_1","doi-asserted-by":"crossref","first-page":"682","DOI":"10.1038\/s41409-025-02546-w","article-title":"\u03b1\u00df T\u2010cell depleted haploidentical stem cell transplantation for pediatric and young adult patients with transfusion\u2010dependent thalassemia","volume":"60","author":"Kleinschmidt K","year":"2025","journal-title":"Bone Marrow 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in chronic graft\u2010versus\u2010host disease: I. The 2014 diagnosis and staging working group report","volume":"21","author":"Jagasia MH","year":"2015","journal-title":"Biol Blood Marrow Transplant"},{"issue":"17","key":"e_1_2_10_15_1","doi-asserted-by":"crossref","first-page":"2562","DOI":"10.1182\/bloodadvances.2019000291","article-title":"Related and unrelated donor transplantation for \u03b2\u2010thalassemia major: results of an international survey","volume":"3","author":"Eapen M","year":"2019","journal-title":"Blood Adv"},{"issue":"18","key":"e_1_2_10_16_1","doi-asserted-by":"crossref","first-page":"1663","DOI":"10.1056\/NEJMoa2309673","article-title":"Exagamglogene Autotemcel for transfusion\u2010dependent \u03b2\u2010thalassemia","volume":"390","author":"Locatelli F","year":"2024","journal-title":"N Engl J Med"},{"issue":"8","key":"e_1_2_10_17_1","doi-asserted-by":"crossref","first-page":"1969","DOI":"10.1002\/ajh.70382","article-title":"Correction of ineffective erythropoiesis and normalization of iron homeostasis after Exagamglogene Autotemcel in transfusion\u2010dependent beta\u2010thalassemia","volume":"101","author":"Sheth S","year":"2026","journal-title":"Am J Hematol"},{"key":"e_1_2_10_18_1","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-026-69756-8","article-title":"A multi\u2010center clinical trial of allogeneic hematopoietic stem cell transplantation in transfusion\u2010dependent thalassemia","volume":"17","author":"Liu R","year":"2026","journal-title":"Nat Commun"},{"issue":"6","key":"e_1_2_10_19_1","doi-asserted-by":"crossref","first-page":"813","DOI":"10.1038\/bmt.2016.7","article-title":"Hematopoietic stem cell transplantation for homozygous \u03b2\u2010thalassemia and \u03b2\u2010thalassemia\/hemoglobin E patients from haploidentical donors","volume":"51","author":"Anurathapan U","year":"2016","journal-title":"Bone Marrow 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tertiary referral Center in South India","volume":"26","author":"Swaminathan VV","year":"2020","journal-title":"Biol Blood Marrow Transplant"},{"issue":"8","key":"e_1_2_10_26_1","doi-asserted-by":"crossref","first-page":"1392","DOI":"10.1016\/j.bbmt.2015.05.001","article-title":"Complement\u2010binding donor\u2010specific anti\u2010HLA antibodies and risk of primary graft failure in hematopoietic stem cell transplantation","volume":"21","author":"Ciurea SO","year":"2015","journal-title":"Biol Blood Marrow Transplant"},{"issue":"8","key":"e_1_2_10_27_1","doi-asserted-by":"crossref","first-page":"1019","DOI":"10.1097\/TP.0b013e3181b9d710","article-title":"High risk of graft failure in patients with anti\u2010HLA antibodies undergoing haploidentical stem\u2010cell transplantation","volume":"88","author":"Ciurea 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