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Leukemia research · 2026

Association of hypomethylating agents + venetoclax in the real-life treatment of Myeloproliferative Neoplasms in blastic phase.

СтатьяМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
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Latagliata Roberto, Pugliese Novella, Lame Dorela, Cristiano Gianluca, De Luca Giulia, Strazimiri Alda, Cavalca Fabrizio, Rizzo Lorenzo, Zoletto Simone, Federico Vincenzo, Cattaneo Daniele, Crugnola Monica, Biagi Annalisa, Niscola Pasquale, Palmieri Raffaele, Mulas Olga, Vangone Beatrice Esposito, Santopietro Michelina, Bruzzese Antonella, Tiribelli Mario, Di Veroli Ambra, Stagno Fabio, Ciuffreda Lucia, Iurlo Alessandra, Di Renzo Nicola, Binotto Gianni, Salutari Prassede, Riva Marta, Bonifacio Massimiliano, Elli Elena Maria, Curti Antonio, Poloni Antonella

Аннотация

BACKGROUND: Patients (pts) with Myeloproliferative Neoplasms evolved in blastic phase (MPN-BP) have a dismal overall survival (OS). Very few data on the association of hypomethylating agents (HMA) and venetoclax (VEN) are available at present in homogeneous cohort of MPN-BP pts.

METHODS: Data of 61 pts with MPN-BP treated frontline with HMA + VEN in 21 hematologic Centers in Italy outside clinical trials from 11/2018 to 8/2024 were retrospectively collected and analysed.

RESULTS: Pts were treated for a median of 4 courses (IQR 2-8). Overall, 56 pts (91.8%) had at least one hematologic toxicity of grade 3-4: in particular, severe neutropenia (PMN < 0.5 × 109/l) was reported in 50 pts (81.9%). Thirty-two pts (52.4%) had at least one infective episode during the treatment: pulmonary infections were reported in 13 pts (21.3%). Overall Response Rate (ORR) was 60.6%, with a median response duration of 9.4 months (95%CI 4.3-18.3): in particular, 26 patients (42.6%) achieved ALR-C/Ci, with a median response duration of 18.3 months (95%CI 1.2-35.3). Median OS of the whole cohort was 10.5 months (95%CI 7.7-13.3). Pts with any type of response had a significantly longer OS compared to pts with progressive/stable disease [13.6 (95%CI 10.3-21.9) versus 7.1 (95%CI 4.4-11.7) months, respectively (p < 0.001)].

CONCLUSIONS: Our real-life data confirm that HMA + VEN combination could have a role in MPN-BP: however, response duration is still very short, with a persistently poor median OS. As a consequence, addition of other targeted therapies should be explored.

Предметные рубрики MeSH

ИП Урсолов Владлен Сергеевич

ИНН 500118482352 · ОГРНИП 314500117700049

Реквизиты и контакты