{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T02:25:18Z","timestamp":1777861518834,"version":"3.51.4"},"reference-count":30,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T00:00:00Z","timestamp":1777507200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Oncol."],"abstract":"<jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Non\u2013Down syndrome acute megakaryoblastic leukemia (non\u2013DS\u2013AMKL) is a rare but high-risk subtype of pediatric AML with dismal outcomes. Allo-HSCT in first complete remission (CR1) is recommended as the preferred strategy for improving survival, yet the benefit of BU\/CY intensification with melphalan (MEL) or thiotepa (TT) remains unclear.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We retrospectively analyzed 70 pediatric non\u2013DS\u2013AMKL patients undergoing ATG\/G-CSF\u2013based haplo-HSCT between March 2010 and February 2024, comparing standard BU\/CY (n=25) with intensified BU+MEL\/TT (n=45). Endpoints included OS, LFS, RI, NRM, and TRM. Survival was estimated by Kaplan\u2013Meier and compared with the log-rank test, while cumulative incidences were analyzed using Gray\u2019s test. Cox proportional hazards and Fine\u2013Gray competing-risk regression models were applied for univariate and multivariate analyses, as appropriate.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Baseline clinical characteristics were comparable between groups, whereas graft CD34+ (median, 10.40\u00d710\n                      <jats:sup>6<\/jats:sup>\n                      \/kg vs 5.85\u00d710\n                      <jats:sup>6<\/jats:sup>\n                      \/kg) and CD3+ (median, 4.54\u00d710\n                      <jats:sup>8<\/jats:sup>\n                      \/kg vs 1.89\u00d710\n                      <jats:sup>8<\/jats:sup>\n                      \/kg) cell doses were significantly higher in the BU+MEL\/TT group (both P&amp;lt;0.001). Median follow-up was 25 months (range, 13\u201391.2). Day-100 TRM was similar between groups (6.67% vs 8.00%,\n                      <jats:italic>P<\/jats:italic>\n                      \u00a0=\u00a00.81), with no significant differences in engraftment, GVHD, viral reactivations. No VOD\/SOS or TMA events were observed. Among patients transplanted in CR, the BU+MEL\/TT group demonstrated superior 5-year OS (75.2% vs 50.0%,\n                      <jats:italic>P<\/jats:italic>\n                      \u00a0=\u00a00.045) and LFS (76.3% vs 50.0%,\n                      <jats:italic>P<\/jats:italic>\n                      \u00a0=\u00a00.039), with numerically lower RI and NRM. In contrast, patients transplanted in NR\/PR had poor outcomes in both groups, with 1-year OS and LFS of approximately 20%. In multivariable Cox analyses, intensified conditioning (BU+MEL\/TT vs BU) improved OS (HR 0.20, P\u00a0=\u00a00.047) and LFS (HR 0.29, P\u00a0=\u00a00.034). Complex cytogenetics (OS: HR 2.38, P\u00a0=\u00a00.016; LFS: HR 2.58, P\u00a0=\u00a00.011) and pre-transplant NR\/PR status (OS: HR 5.53, P\u00a0=\u00a00.001; LFS: HR 3.55, P\u00a0=\u00a00.004) were independent adverse factors. In competing-risk models, conditioning was not associated with RI or NRM, whereas NR\/PR status significantly increased relapse (HR 3.39, P\u00a0=\u00a00.019) and NRM (HR 3.73, P\u00a0=\u00a00.007).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>In pediatric non\u2013DS\u2013AMKL, intensified conditioning (BU+MEL\/TT) independently improved OS and LFS in patients transplanted in CR without increasing early NRM. Pre-transplant NR\/PR status was the strongest adverse factor, markedly increasing RI and NRM, underscoring the importance of achieving remission before transplantation.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3389\/fonc.2026.1741238","type":"journal-article","created":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T04:21:15Z","timestamp":1777522875000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":0,"title":["Superior long-term survival with acceptable safety of ATG\/G-CSF\u2013based haplo-HSCT with intensified BU+MEL\/TT conditioning in CR Pediatric Non-DS\u2013AMKL"],"prefix":"10.3389","volume":"16","author":[{"given":"Fei","family":"Pan","sequence":"first","affiliation":[{"name":"Beijing Lu Daopei Hospital","place":["Beijing, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jing","family":"Long","sequence":"additional","affiliation":[{"name":"Beijing Lu Daopei Hospital","place":["Beijing, China"]},{"name":"Beijing Lu Daopei Institute of Hematology","place":["Beijing, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xing-yu","family":"Cao","sequence":"additional","affiliation":[{"name":"Beijing Lu Daopei Hospital","place":["Beijing, China"]},{"name":"Hebei Yanda Lu Daopei Hospital","place":["Langfang, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yue","family":"Lu","sequence":"additional","affiliation":[{"name":"Beijing Lu Daopei Hospital","place":["Beijing, China"]},{"name":"Hebei Yanda Lu Daopei Hospital","place":["Langfang, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jian-ping","family":"Zhang","sequence":"additional","affiliation":[{"name":"Beijing Lu Daopei Hospital","place":["Beijing, China"]},{"name":"Hebei Yanda Lu Daopei Hospital","place":["Langfang, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yan-li","family":"Zhao","sequence":"additional","affiliation":[{"name":"Beijing Lu Daopei Hospital","place":["Beijing, China"]},{"name":"Hebei Yanda Lu Daopei Hospital","place":["Langfang, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Min","family":"Xiong","sequence":"additional","affiliation":[{"name":"Beijing Lu Daopei Hospital","place":["Beijing, China"]},{"name":"Hebei Yanda Lu Daopei Hospital","place":["Langfang, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fang-min","family":"Pan","sequence":"additional","affiliation":[{"name":"Beijing Lu Daopei Hospital","place":["Beijing, China"]},{"name":"Beijing Lu Daopei Institute of Hematology","place":["Beijing, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Guan-lan","family":"Yue","sequence":"additional","affiliation":[{"name":"Hebei Yanda Lu Daopei Hospital","place":["Langfang, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kang","family":"Gao","sequence":"additional","affiliation":[{"name":"Hebei Yanda Lu Daopei Hospital","place":["Langfang, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zhi-jie","family":"Wei","sequence":"additional","affiliation":[{"name":"Beijing Lu Daopei Hospital","place":["Beijing, China"]},{"name":"Hebei Yanda Lu Daopei Hospital","place":["Langfang, China"]}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1965","published-online":{"date-parts":[[2026,4,30]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jtct.2022.06.005","article-title":"Hematopoietic cell transplantation in the treatment of pediatric acute myelogenous leukemia and myelodysplastic syndromes: guidelines from the American Society of Transplantation and Cellular Therapy","volume":"28","author":"Tarlock","year":"2022","journal-title":"Transplant Cell 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