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Background <p>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.</p> Methods <p>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).</p> Results <p>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.</p> Conclusions <p>Our findings indicate that HMA/VEN combination therapy shows promise as a bridging strategy to allo-SCT in HR-MDS and CMML.</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年4月26日

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  • 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

摘要

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.