Hypomethylating agents plus venetoclax for high-risk MDS and CMML as bridge therapy to transplant: a GESMD study

Hypomethylating agents plus venetoclax for high-risk MDS and CMML as bridge therapy to transplant: a GESMD study

Abstract Background 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...

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Main Authors: Ines Zugasti, Monica Lopez-Guerra, Sandra Castaño-Díez, Daniel Esteban, Alejandro Avendaño, Helena Pomares, Ana Perez, Sara García-Ávila, Irene Padilla Conejo, Cristina de la Fuente Montes, Alexandra Martínez-Roca, Beatriz Merchán, Carlos Jiménez-Vicente, Francesca Guijarro, Jose Ramón Álamo, Albert Cortes-Bullich, Victor Torrecillas, Lucia Mont, Esther Carcelero, Gisela Riu, Lurdes Zamora, Joan Bargay, Ana Triguero, Maria Suarez-Lledó, Maria Queralt Salas, Felix López-Cadenas, Fernando Ramos, Blanca Xicoy, David Valcárcel, Montserrat Arnan, Carmen Martínez, Montserrat Rovira, Francesc Fernández-Avilés, Maria Díez-Campelo, Jordi Esteve, Marina Díaz-Beyá
Format: Article
Language:English
Published: BMC 2025-04-01
Series:Experimental Hematology & Oncology
Subjects:
MDS
CMML
MDS/MPN
Allo-SCT
Bridge therapy
Cytoreductive therapy
Online Access:https://doi.org/10.1186/s40164-025-00652-5
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Summary:Abstract Background 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. Methods 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). Results 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. Conclusions Our findings indicate that HMA/VEN combination therapy shows promise as a bridging strategy to allo-SCT in HR-MDS and CMML.
ISSN:2162-3619

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