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

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This study was supported by grants from the Instituto de Salud Carlos III grant number PI22/01660 (JE, MD-B) and co-funded by the European Union and Convocatoria d'Intensificacio Interna per als professionals de l'HCB 2023 Emili-Letang (MD-B).

Analysis of institutional authors

Lopez-Guerra, MonicaAuthorJimenez-Vicente, CarlosAuthorGuijarro, FrancescaAuthorCortés-Bullich, AlbertAuthorSalas, Maria QueraltAuthorRovira, MontserratAuthorEsteve, JordiAuthorDiaz-Beya, MarinaCorresponding Author

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Article

Publicated to:Experimental Hematology And Oncology. 14 (1): 61- - 2025-04-26 14(1), DOI: 10.1186/s40164-025-00652-5

Authors: Zugasti, Ines; Lopez-Guerra, Monica; Castano-Diez, Sandra; Esteban, Daniel; Avendano, Alejandro; Pomares, Helena; Perez, Ana; Conejo, Irene Padilla; de la Fuente Montes, Cristina; Martinez-Roca, Alexandra; Merchan, Beatriz; Jimenez-Vicente, Carlos; Guijarro, Francesca; Alamo, Jose Ramon; Cortes-Bullich, Albert; Torrecillas, Victor; Mont, Lucia; Carcelero, Esther; Riu, Gisela; Zamora, Lurdes; Bargay, Joan; Triguero, Ana; Suarez-Lledo, Maria; Salas, Maria Queralt; Lopez-Cadenas, Felix; Ramos, Fernando; Xicoy, Blanca; Valcarcel, David; Arnan, Montserrat; Martinez, Carmen; Rovira, Montserrat; Fernandez-Aviles, Francesc; Diez-Campelo, Maria; Esteve, Jordi; Diaz-Beya, Marina

Affiliations

Ctr Invest Biomed Red Canc CIBERONC, Madrid, Spain - Author

Fdn Recerca Clin Barcelona, Inst Invest Biomed August Pi i Sunyer, Barcelona, Spain - Author

Grp Espanol Sindromes Mielodisplas GESMD, Madrid, Spain - Author

Hosp Badalona Germans Trias & Pujol, Inst Catala Oncol ICO, Badalona, Spain - Author

Hosp Clin Barcelona, Barcelona, Spain - Author

Hosp Duran i Reynals, Inst Catala Oncol, Barcelona, Spain - Author

Hosp Mar, Barcelona, Spain - Author

Hosp Son Llatzer, Palma De Mallorca, Spain - Author

Hosp Univ Leon, Leon, Spain - Author

Hosp Univ Salamanca, Salamanca, Spain - Author

Hosp Univ Vall Hebron, Barcelona, Spain - Author

Josep Carreras Leukemia Res Inst, Barcelona, Spain - Author

Univ Autonoma Barcelona, Barcelona, Spain - Author

Univ Barcelona, Barcelona, Spain - Author

Abstract

BackgroundHigh-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.MethodsWe 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-na & iuml;ve and 12 were refractory or relapsed (R/R).ResultsAs 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.ConclusionsOur findings indicate that HMA/VEN combination therapy shows promise as a bridging strategy to allo-SCT in HR-MDS and CMML.

Keywords

Allo-sctAzacitidineBridge therapyCmmlCytoreductive therapyDecitabineDiseaseDonor availabilityHma/venMdsMds/mpnMolecular follow-uMolecular follow-upMrdMyelodysplastic syndromesOpen-labelProposaResponse criteriaStem-cell transplantation

Quality index

Bibliometric impact. Analysis of the contribution and dissemination channel

The work has been published in the journal Experimental Hematology And Oncology due to its progression and the good impact it has achieved in recent years, according to the agency WoS (JCR), it has become a reference in its field. In the year of publication of the work, 2025, it was in position 10/97, thus managing to position itself as a Q1 (Primer Cuartil), in the category Hematology. Notably, the journal is positioned above the 90th percentile.

Impact and social visibility

Leadership analysis of institutional authors

There is a significant leadership presence as some of the institution’s authors appear as the first or last signer, detailed as follows: Last Author (Díaz Beyá, Marina).