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

Authors

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

Published in

Investigational new drugs. Jun 15, 2026. Epub Jun 15, 2026.

Abstract

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.

PMID:
42295574
Bibliographic data and abstract were imported from PubMed on 15 Jun 2026.

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