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

被引：0

作者：

Ines Zugasti ^{[1
]}

Monica Lopez-Guerra ^{[10
]}

Sandra Castaño-Díez ^{[12
]}

Daniel Esteban ^{[1
]}

Alejandro Avendaño ^{[11
]}

Helena Pomares ^{[13
]}

Ana Perez ^{[1
]}

Sara García-Ávila ^{[10
]}

Irene Padilla Conejo ^{[11
]}

Cristina de la Fuente Montes ^{[1
]}

Alexandra Martínez-Roca ^{[7
]}

Beatriz Merchán ^{[11
]}

Carlos Jiménez-Vicente ^{[2
]}

Francesca Guijarro ^{[10
]}

Jose Ramón Álamo ^{[3
]}

Albert Cortes-Bullich ^{[10
]}

Victor Torrecillas ^{[4
]}

Lucia Mont ^{[10
]}

Esther Carcelero ^{[5
]}

Gisela Riu ^{[10
]}

Lurdes Zamora ^{[6
]}

Joan Bargay ^{[10
]}

Ana Triguero ^{[7
]}

Maria Suarez-Lledó ^{[10
]}

Maria Queralt Salas ^{[1
]}

Felix López-Cadenas ^{[11
]}

Fernando Ramos ^{[1
]}

Blanca Xicoy ^{[11
]}

David Valcárcel ^{[1
]}

Montserrat Arnan ^{[11
]}

Carmen Martínez ^{[1
]}

Montserrat Rovira ^{[11
]}

Francesc Fernández-Avilés ^{[1
]}

Maria Díez-Campelo ^{[11
]}

Jordi Esteve ^{[1
]}

Marina Díaz-Beyá ^{[11
]}

机构：

[1] Hospital Clínic Barcelona, Barcelona

[2] Hospital Universitario de Salamanca, Salamanca

[3] Institut Català d’Oncologia, Hospital Duran I Reynals, L’Hospitalet de Llobregat, Barcelona

[4] Hospital Universitario Vall d´Hebrón, Barcelona

[5] Hospital del Mar, Barcelona

[6] Hospital Universitario de León, León

[7] Institut Català d’Oncologia (ICO), Hospital Germans Trias I Pujol, Badalona

[8] Universitat de Barcelona, Barcelona

[9] Hospital Son Llátzer, Palma

[10] Grupo Español de Síndromes Mielodisplásicos (GESMD), Madrid

[11] Fundació de Recerca Clínic Barcelona-Institut d’Investigacions Biomèdiques August Pi I Sunyer (FRCB-IDIBAPS), Barcelona

[12] Josep Carreras Leukemia Research Institute, Barcelona

[13] Centro de Investigación Biomédica en Red de Cáncer (CIBERONC), Madrid

[14] Universitat Autònoma de Barcelona, Barcelona

来源：

Experimental Hematology & Oncology | / 14卷 / 1期

关键词：

Allo-SCT; Bridge therapy; CMML; Cytoreductive therapy; HMA/VEN; MDS; MDS/MPN; Molecular follow-up; MRD;

D O I：

10.1186/s40164-025-00652-5

中图分类号：

学科分类号：

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. © The Author(s) 2025.

引用

共 54 条

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