Zugasti Ines, Lopez-Guerra Monica, Castaño-Díez Sandra, Esteban Daniel, Avendaño Alejandro, Pomares Helena, Perez Ana, García-Ávila Sara, Conejo Irene Padilla, de la Fuente Montes Cristina, Martínez-Roca Alexandra, Merchán Beatriz, Jiménez-Vicente Carlos, Guijarro Francesca, Álamo Jose Ramón, Cortes-Bullich Albert, Torrecillas Victor, Mont Lucia, Carcelero Esther, Riu Gisela, Zamora Lurdes, Bargay Joan, Triguero Ana, Suarez-Lledó Maria, Salas Maria Queralt, López-Cadenas Felix, Ramos Fernando, Xicoy Blanca, Valcárcel David, Arnan Montserrat, Martínez Carmen, Rovira Montserrat, Fernández-Avilés Francesc, Díez-Campelo Maria, Esteve Jordi, Díaz-Beyá Marina
Hospital Clínic Barcelona, Barcelona, Spain.
Grupo Español de Síndromes Mielodisplásicos (GESMD), Madrid, Spain.
Exp Hematol Oncol. 2025 Apr 26;14(1):61. doi: 10.1186/s40164-025-00652-5.
High-risk myelodysplastic syndromes (HR-MDS) and chronic myelomonocytic leukemia (CMML) remain therapeutic challenges with suboptimal outcomes. The only potentially curative treatment is allogeneic stem cell transplantation (allo-SCT). The most frequent pre-allo-SCT treatment is monotherapy with hypomethylating agents (HMA), but approximately 40% of patients cannot proceed to allo-SCT, mainly due to disease progression. Recent evidence suggests that combining HMA with venetoclax (HMA/VEN) could increase HMA efficacy in HR-MDS but it remains unclear if this combination could bridge more patients to allo-SCT.
We retrospectively evaluated HMA/VEN as a bridge to allo-SCT in 30 patients with HR-MDS or CMML eligible for transplant. Eighteen patients were treatment-naïve and 12 were refractory or relapsed (R/R).
As defined by the IWG 2023 criteria, the overall response rate (ORR) was 90% and the composite complete response rate was 77%. For the R/R patients, ORR was 83%. The allo-SCT rate was 83%, and the allo-SCT rate of those patients treated exclusively with HMA/VEN without further bridge therapies was 76%. One- and two-year post-allo-SCT survival was 75% and two-year cumulative incidence of relapse was 30.5%. Follow-up of measurable residual disease identified some molecular relapses that were controlled with preemptive treatment.
Our findings indicate that HMA/VEN combination therapy shows promise as a bridging strategy to allo-SCT in HR-MDS and CMML.
高危骨髓增生异常综合征（HR-MDS）和慢性粒单核细胞白血病（CMML）仍然是治疗挑战，治疗效果欠佳。唯一可能治愈的治疗方法是异基因干细胞移植（allo-SCT）。allo-SCT前最常用的治疗是使用去甲基化药物（HMA）进行单药治疗，但约40%的患者无法进行allo-SCT，主要原因是疾病进展。最近的证据表明，将HMA与维奈克拉联合使用（HMA/VEN）可提高HMA在HR-MDS中的疗效，但尚不清楚这种联合治疗能否使更多患者过渡到allo-SCT。
我们回顾性评估了HMA/VEN作为30例符合移植条件的HR-MDS或CMML患者allo-SCT的桥梁治疗。18例患者既往未接受过治疗，12例为难治性或复发性（R/R）患者。
根据IWG 2023标准定义，总缓解率（ORR）为90%，完全缓解率为77%。对于R/R患者，ORR为83%。allo-SCT率为83%，仅接受HMA/VEN治疗而无进一步桥梁治疗的患者allo-SCT率为76%。allo-SCT后1年和2年生存率分别为75%，2年累积复发率为30.5%。对可测量残留病的随访发现了一些通过抢先治疗得到控制的分子复发。
我们的研究结果表明，HMA/VEN联合治疗作为HR-MDS和CMML患者allo-SCT的桥梁策略显示出前景。
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