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.
Association of hypomethylating agents + venetoclax in the real-life treatment of Myeloproliferative Neoplasms in blastic phase
Tiribelli M.;
2026-01-01
Abstract
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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
