Selected studies of treatment outcomes in MPN-BP
| Therapeutic regimen . | Patient population . | Study design . | Key outcomes . | Reference . |
|---|---|---|---|---|
| Intensive chemotherapy | ||||
| Intensive chemotherapy (not specified) | MPN-BP | Retrospective cohort study | Median OS 10.2 mo | 61 |
| “7 + 3” cytarabine plus an anthracycline | MPN-BP | Retrospective cohort study | CR rate 67%; median OS 19.2 mo for allo-HCT vs 3.8 mo for nontransplant patients | 5 |
| AML-like induction chemotherapy | MPN-BP | Retrospective cohort study | CR rate 35%; 3-y OS 32% for allo-HCT vs 19% for nontransplant patients | 4 |
| HMA monotherapy | ||||
| Azacitidine | MPN-BP/AP | Retrospective cohort study | ORR 61.5%; median OS 13.5 mo | 43 |
| MPN-BP | Retrospective cohort study | ORR 68%; median OS 9.9 mo | 42 | |
| MPN-BP | Retrospective cohort study | Median OS 9 mo | 61 | |
| Azacitidine or decitabine | MPN-BP | Retrospective cohort study | Median OS 6.7 mo | 5 |
| Decitabine | MPN-BP, MPN-AP, high-risk PMF | Retrospective cohort study | MPN-BP: 29% ORR, 10.5 mo median OS MPN-AP: 62% ORR, 11.8 mo median OS | 41 |
| Ruxolitinib monotherapy | ||||
| Ruxolitinib | MPN-BP | Phase 2 clinical trial | ORR 16.6% | 48 |
| HMA + ruxolitinib | ||||
| Ruxolitinib + decitabine | MPN-AP, MPN-BP | Phase 2 clinical trial | ORR 44%; median OS 9.5 mo | 49 |
| MPN-BP | Phase 1/2 clinical trial | ORR 45%; median OS 8.4 mo | 50 | |
| HMA + venetoclax | ||||
| HMA + venetoclax | MPN-BP | Retrospective cohort study | Newly diagnosed: ORR 50%; median OS 7 mo R/R: no objective responses; median OS 3 mo | 45 |
| MPN-BP | Retrospective cohort study | ORR 44%; median OS 8 mo | 44 | |
| MPN-AP, MPN-BP | Retrospective multicenter cohort study | ORR: 52%; median OS 6 mo | 47 | |
| IDH1/2 inhibitors | ||||
| Enasidenib | IDH2-mutant MPN-AP, MPN-BP | Retrospective cohort study | ORR: 37.5% per ELN 2017 and 75% per MPN-BP response criteria | 54 |
| Enasidenib or ivosidenib | IDH1 or IDH2-mutant MPN-AP, MPN-BP | Retrospective cohort study | 43% CR rate among newly diagnosed patients; no objective responses in R/R cohort | 53 |
| Therapeutic regimen . | Patient population . | Study design . | Key outcomes . | Reference . |
|---|---|---|---|---|
| Intensive chemotherapy | ||||
| Intensive chemotherapy (not specified) | MPN-BP | Retrospective cohort study | Median OS 10.2 mo | 61 |
| “7 + 3” cytarabine plus an anthracycline | MPN-BP | Retrospective cohort study | CR rate 67%; median OS 19.2 mo for allo-HCT vs 3.8 mo for nontransplant patients | 5 |
| AML-like induction chemotherapy | MPN-BP | Retrospective cohort study | CR rate 35%; 3-y OS 32% for allo-HCT vs 19% for nontransplant patients | 4 |
| HMA monotherapy | ||||
| Azacitidine | MPN-BP/AP | Retrospective cohort study | ORR 61.5%; median OS 13.5 mo | 43 |
| MPN-BP | Retrospective cohort study | ORR 68%; median OS 9.9 mo | 42 | |
| MPN-BP | Retrospective cohort study | Median OS 9 mo | 61 | |
| Azacitidine or decitabine | MPN-BP | Retrospective cohort study | Median OS 6.7 mo | 5 |
| Decitabine | MPN-BP, MPN-AP, high-risk PMF | Retrospective cohort study | MPN-BP: 29% ORR, 10.5 mo median OS MPN-AP: 62% ORR, 11.8 mo median OS | 41 |
| Ruxolitinib monotherapy | ||||
| Ruxolitinib | MPN-BP | Phase 2 clinical trial | ORR 16.6% | 48 |
| HMA + ruxolitinib | ||||
| Ruxolitinib + decitabine | MPN-AP, MPN-BP | Phase 2 clinical trial | ORR 44%; median OS 9.5 mo | 49 |
| MPN-BP | Phase 1/2 clinical trial | ORR 45%; median OS 8.4 mo | 50 | |
| HMA + venetoclax | ||||
| HMA + venetoclax | MPN-BP | Retrospective cohort study | Newly diagnosed: ORR 50%; median OS 7 mo R/R: no objective responses; median OS 3 mo | 45 |
| MPN-BP | Retrospective cohort study | ORR 44%; median OS 8 mo | 44 | |
| MPN-AP, MPN-BP | Retrospective multicenter cohort study | ORR: 52%; median OS 6 mo | 47 | |
| IDH1/2 inhibitors | ||||
| Enasidenib | IDH2-mutant MPN-AP, MPN-BP | Retrospective cohort study | ORR: 37.5% per ELN 2017 and 75% per MPN-BP response criteria | 54 |
| Enasidenib or ivosidenib | IDH1 or IDH2-mutant MPN-AP, MPN-BP | Retrospective cohort study | 43% CR rate among newly diagnosed patients; no objective responses in R/R cohort | 53 |