{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T15:29:46Z","timestamp":1787671786924,"version":"build-2736575974"},"reference-count":45,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2026,7,23]],"date-time":"2026-07-23T00:00:00Z","timestamp":1784764800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,7,23]],"date-time":"2026-07-23T00:00:00Z","timestamp":1784764800000},"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":["Pediatric Blood &amp;amp; Cancer"],"published-print":{"date-parts":[[2026,10]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Graft failure and transplant\u2010related mortality remain major barriers to haploidentical donor hematopoietic stem cell transplantation\u00a0(HID\u2010HSCT) in children and adolescents aged \u22657\u00a0years with transfusion\u2010dependent thalassemia (TDT). Thus, a novel low\u2010toxicity myeloablative conditioning regimen was developed to improve transplantation outcomes.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>In this prospective, single\u2010center study (clinical trial registration ChiCTR2300071890), TDT patients aged \u22657\u00a0years were enrolled to evaluate the safety and efficacy of a conditioning regimen comprising fludarabine, busulfan, thiotepa, and melphalan (F\u2010BMT). For HID recipients, graft\u2010versus\u2010host disease (GVHD) prophylaxis included half\u2010dose PTCY combined with the in vivo T\u2010cell depletion (porcine anti\u2010human lymphocyte immunoglobulin \nor rabbit anti\u2010human thymocyte globulin), along with other immunosuppressive agents. The primary endpoint was the 2\u2010year thalassemia\u2010free survival (TFS) rate.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>From June 2023 to August 2024, 29 TDT patients aged \u22657\u00a0years undergoing HID\u2010HSCT were enrolled (14 males, 15 females). The 2\u2010year TFS and overall survival rates were both 93.1%. Neutrophil and platelet engraftment were achieved in all patients, with no reported graft failure. The observed incidence of Grade II\u2013IV acute GVHD was 17.2%. Chronic GVHD occurred in 6.9% of the patients. Regimen\u2010related toxicities were manageable, with only one case of sinusoidal obstruction syndrome and three cases of hemorrhagic cystitis, all of which resolved with supportive treatment. No mixed chimerism was observed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>The F\u2010BMT regimen provides effective myeloablation with reduced toxicity, supporting its feasibility for HID\u2010HSCT in TDT patients aged \u22657\u00a0years.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/1545-5017.70563","type":"journal-article","created":{"date-parts":[[2026,7,23]],"date-time":"2026-07-23T13:56:20Z","timestamp":1784814980000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Fludarabine, Busulfan, Thiotepa, and Melphalan: A Novel Low\u2010Toxicity Myeloablative Conditioning Regimen for Haploidentical HSCT in Children and Adolescents Aged \u22657\u00a0Years With Transfusion\u2010Dependent 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