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<p>Leukemia relapse after allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the leading death cause, with extramedullary disease history as an independent relapse risk factor in acute leukemia. We analyzed 10 patients with extramedullary disease history who received allo-HSCT with mitoxantrone liposome (Lipo-MIT)–containing conditioning at our center (Dec 2022–Jan 2025). Median age was 39.5 (14–63), 7 had acute myeloid leukemia (AML, including myeloid sarcoma), 2&#xa0;T lymphoblastic lymphoma/leukemia, and 1 B-cell acute lymphoblastic leukemia. Pre-transplant, 4 were in complete remission (CR) (1 with minimal residual disease [MRD] positivity) and 6 had residual lesions (2 with BM blasts ≥ 5%, 2 with MRD positivity). The conditioning regimen was Lipo-MIT + total body irradiation/busulfan + cyclophosphamide ± cytarabine, with a median Lipo-MIT dose of 26.1 (18.5–28.9) mg/m<sup>2</sup>. Mucosal injury showed a high incidence (9/10) after conditioning, but most were transient and controllable with supportive care. Engraftment succeeded in 9 patients, with a median neutrophil recovery time of 16 (11–21) days and platelet recovery time of 20 (11–39) days. Only 1 patient developed grade 2 skin acute graft-versus-host disease (aGVHD). All 9 engrafted patients achieved complete remission (CR) post-transplantation; one had MRD positivity at + 177&#xa0;days, which turned negative after chemotherapy combined with donor lymphocyte infusion. One patient relapsed at + 234&#xa0;days and died at + 272&#xa0;days. The patient with engraftment failure died at + 346&#xa0;days due to disease progression, with a history of polycythemia vera complicated by myelofibrosis. Lipo-MIT conditioning shows promising efficacy/safety for allo-HSCT in extramedullary disease patients. Randomized trials are needed.</p>

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Allogeneic hematopoietic stem cell transplantation with liposomal mitoxantrone conditioning for extramedullary leukemia

  • 期刊论文

  • 发表日期：

    2026年6月15日

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  • Lei Deng,
  • Xiaolin Yu,
  • Xiaocheng Song,
  • Wenjun Li,
  • Rui Guan,
  • Yixi Hou,
  • Yan Shao,
  • Yuerong Zhao,
  • Qianqian Xiao,
  • Jing Wang,
  • Yue Liu,
  • Bo Xin,
  • Fang Zhou

摘要

Leukemia relapse after allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the leading death cause, with extramedullary disease history as an independent relapse risk factor in acute leukemia. We analyzed 10 patients with extramedullary disease history who received allo-HSCT with mitoxantrone liposome (Lipo-MIT)–containing conditioning at our center (Dec 2022–Jan 2025). Median age was 39.5 (14–63), 7 had acute myeloid leukemia (AML, including myeloid sarcoma), 2 T lymphoblastic lymphoma/leukemia, and 1 B-cell acute lymphoblastic leukemia. Pre-transplant, 4 were in complete remission (CR) (1 with minimal residual disease [MRD] positivity) and 6 had residual lesions (2 with BM blasts ≥ 5%, 2 with MRD positivity). The conditioning regimen was Lipo-MIT + total body irradiation/busulfan + cyclophosphamide ± cytarabine, with a median Lipo-MIT dose of 26.1 (18.5–28.9) mg/m2. Mucosal injury showed a high incidence (9/10) after conditioning, but most were transient and controllable with supportive care. Engraftment succeeded in 9 patients, with a median neutrophil recovery time of 16 (11–21) days and platelet recovery time of 20 (11–39) days. Only 1 patient developed grade 2 skin acute graft-versus-host disease (aGVHD). All 9 engrafted patients achieved complete remission (CR) post-transplantation; one had MRD positivity at + 177 days, which turned negative after chemotherapy combined with donor lymphocyte infusion. One patient relapsed at + 234 days and died at + 272 days. The patient with engraftment failure died at + 346 days due to disease progression, with a history of polycythemia vera complicated by myelofibrosis. Lipo-MIT conditioning shows promising efficacy/safety for allo-HSCT in extramedullary disease patients. Randomized trials are needed.