Geng Liangquan, Chen Erling, Ji Yanping, Liu Huilan, Sun Zimin
Department of Hematology, The First Affiliated Hospital of University of Science and Technology of China, Hefei, People's Republic of China.
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, People's Republic of China.
Blood Lymphat Cancer. 2025 Aug 1;15:103-113. doi: 10.2147/BLCTT.S527790. eCollection 2025.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only curative treatment for higher-risk myelodysplastic syndromes (MDS), but optimal timing and donor selection remain controversial.
We conducted a retrospective analysis of 70 higher-risk MDS patients classified by the revised International Prognostic Scoring System (IPSS-R) undergoing allo-HSCT. Patients were stratified by: 1) the interval from diagnosis to allo-HSCT (early: <6 months vs later: ≥6 months); 2) pre-transplant treatment cycles (fewer: <2 vs more: ≥2); 3) remission status (complete remission [CR] / partial remission [PR] vs non-remission [NR]), and 4) donor type (sibling vs unrelated cord blood [UCB]).
The results showed a significantly higher 3-year overall survival (OS) in the early HSCT group (70% vs 50%, p = 0.05) with lower transplant-related mortality (TRM) (22.7% vs 46.5%, p = 0.0205). Although more pre-transplant treatment cycles were linked to a lower relapse rate (2.3% vs 15.4%, p = 0.0403), they did not significantly affect OS or TRM. Early HSCT emerged as the only significant factor influencing both OS (Hazard Ratio [HR] 2.84, p = 0.01) and TRM (HR 3.21, p = 0.01). While no significant differences were noted between sibling HSCT and unrelated cord blood transplantation (UCBT) for OS and TRM, UCBT demonstrated a lower incidence of chronic graft-versus-host disease (cGVHD) (19.0% vs 52.9%, p = 0.003).
Our findings suggest early allo-HSCT may optimize outcomes in higher-risk MDS. In settings where sibling donors are unavailable, UCBT could serve as a potential alternative, though this observation requires validation in prospective multicenter studies to account for inherent selection biases and confounding factors.
异基因造血干细胞移植（allo-HSCT）是高危骨髓增生异常综合征（MDS）的唯一治愈性治疗方法，但最佳时机和供体选择仍存在争议。
我们对70例根据修订的国际预后评分系统（IPSS-R）分类的接受allo-HSCT的高危MDS患者进行了回顾性分析。患者按以下因素分层：1）从诊断到allo-HSCT的间隔时间（早期：<6个月 vs 晚期：≥6个月）；2）移植前治疗周期数（较少：<2 vs 较多：≥2）；3）缓解状态（完全缓解[CR]/部分缓解[PR] vs 未缓解[NR]），以及4）供体类型（同胞 vs 无关脐血[UCB]）。
结果显示，早期HSCT组的3年总生存率（OS）显著更高（70% vs 50%，p = 0.05），移植相关死亡率（TRM）更低（22.7% vs 46.5%，p = 0.0205）。虽然更多的移植前治疗周期与较低的复发率相关（2.3% vs 15.4%，p = 0.0403），但它们对OS或TRM没有显著影响。早期HSCT是影响OS（风险比[HR] 2.84，p = 0.01）和TRM（HR 3.21，p = 0.01）的唯一显著因素。虽然同胞HSCT和无关脐血移植（UCBT）在OS和TRM方面没有显著差异，但UCBT的慢性移植物抗宿主病（cGVHD）发生率较低（19.0% vs 52.9%，p = 0.003）。
我们的研究结果表明，早期allo-HSCT可能会优化高危MDS的治疗结果。在没有同胞供体的情况下，UCBT可以作为一种潜在的替代方法，不过这一观察结果需要在前瞻性多中心研究中进行验证，以考虑内在的选择偏倚和混杂因素。
