Table 2 Hypomethylating agent (HMA) combination therapy in patients with accelerated or blast phase myeloproliferative neoplasms (MPN-AP/BP) (References cited in the text).
From: Blast phase myeloproliferative neoplasm: contemporary review and 2024 treatment algorithm
Study | Study design MPN type | Treatment regimen | Response rates (duration) | Toxicity | Median survival |
|---|---|---|---|---|---|
HMA + Ruxolitinib | |||||
Mascarenhas et al. Blood Advances 2020 N = 25 | Phase 2 MPN-AP (n = 10) MPN-BP (n = 15) | HMA+ruxolitinib | CR 0% CRi 8% PR 36% ORR 44% (3.4 months) | Grade 3/4 Febrile neutropenia 28% Pneumonia 24% Neutropenia 16% Anemia 16% Bone pain 8% Thrombocytopenia 8% | 9.5 months |
Rampal et al. Blood Advances 2018 N = 21 | Phase 1 MPN-AP (n = 8) MPN-BP (n = 13) | HMA+ruxolitinib | CRi 24% PR 29% ORR 53% | Grade 3/4 Febrile neutropenia 33% Pneumonia 29% Thrombocytopenia 19% Anemia 14% Sepsis 14% | 7.9 months |
Bose et al. Leukemia 2020 N = 14 (Phase 1) N = 18 (Phase 2) | Phase 1/2 MPN-BP | HMA+ruxolitinib | CR 7% CRi 34% PR 3% ORR 45% (1.7 months) | Grade 1/2 Fatigue Pruritus Diarrhea Nausea | 6.2 months |
Drummond et al. Blood 2020 N = 34 | Phase 1/2 MPN-AP (n = 19) MPN-BP (n = 15) | HMA+ruxolitinib | MPN-AP CR 5% Marrow CR 21% PR 5% (10.7 months) MPN-BP PR 27% (6.6 months) | - | 1-year survival 42% |
HMA + Venetoclax | |||||
Gangat et al. Haematologica 2022 N = 47 | Retrospective MPN-BP (N = 47) ND (n = 32) Relapsed (n = 15) | HMA+Ven | CR 26% CRi 17% PR 11% ORR 53% (5 months) | Pancytopenia 62% Neutropenic fever 47% Major hemorrhage 2% Gastrointestinal 11% | 7 months 1-year survival 28% |
Masarova et al. Blood Advances 2021 N = 31 | Retrospective MPN-BP ND (n = 14) Relapsed (n = 17) | HMA+Ven Ven+other Cladribine LDAC IDHi CPX-351 CLIA FLAG(+/-ida) | CR 10% CRi 10% PR 3% ORR 23% Relapsed pts 0% | ≥Grade 3 infection 84% Severe hemorrhage 45% CNS hemorrhage 19% | 4 months 1-year survival 16% |
King et al. AJH 2021 N = 27 | Retrospective MPN-BP (n = 21) ND (n = 8) MPN-AP (n = 6) | HMA+Ven LDAC+Ven | MPN-BP CR 24% PR 19% ORR 52% (2.9 months) MPN-AP CR 50% 1.8 months | MPN-BP Infection 28% Grade 3 hemorrhage 19% MPN-AP Neutropenic fever 50% | 6 months |
Tremblay et al. Leuk Res 2020 N = 9 | Retrospective MPN-BP (n = 8) MPN-AP (n = 1) ND (n = 2) | HMA+Ven | CR 11% CRi 22% | ≥Grade 3 Infection 78% Hemorrhage 56% | 4.2 months |
