Expert review of hematology · 2026
Efficacy of pacritinib versus momelotinib in patients with thrombocytopenic myelofibrosis: a matching-adjusted indirect treatment comparison.
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Pemmaraju Naveen, Lucchesi Alessandro, Oh Stephen T, Wilson Koo, Hakimi Zalmai, Buckley Sarah A, Suthar Purvi, Parkitny Mikolaj, Wojciechowski Piotr, Kolonko Klaudia, Mascarenhas John O
Аннотация
BACKGROUND: Thrombocytopenia is a prognostic marker in patients with myelofibrosis, associated with increased morbidity and reduced survival. We conducted a matching-adjusted-indirect-comparison (MAIC) of pacritinib-vs-momelotinib for treatment of patients with thrombocytopenic myelofibrosis.
RESEARCH DESIGN AND METHODS: Data were from Phase 3 randomized controlled trials of patients with thrombocytopenic myelofibrosis treated with pacritinib 200 mg BID (PERSIST-2 [NCT02055781]) with baseline platelet count 24‒99 × 109/L matched on prognostic indicators with patients on momelotinib 200 mg QD (pooled post-hoc analysis of patients with platelet count <100 × 109/L (MOMENTUM [NCT04173494]; SIMPLIFY-1 [NCT01969838]). Week-24 endpoints included % patients achieving: ≥50% total symptom score reduction (TSS-50), ≥35% spleen volume reduction (SVR-35) and red-blood-cell-transfusion-independence (RBC-TI). Reweighted effects were compared with momelotinib using logistic regression/odds ratio (OR [95% confidence interval, CI]) and Cox regression/hazard ratio (HR [95% CI]) for overall survival (OS).
RESULTS: After matching, baseline characteristics balanced; 76‒81% of matched pacritinib patients had moderate thrombocytopenia (platelet count 50‒99 × 109/L). All outcomes favored pacritinib on ORs (95% CI) for TSS-50: 1.75 (0.60, 5.09); SVR-35: 1.40 (0.32, 6.04); RBC-TI: 1.19 (0.36, 3.97); and OS: HR (95% CI) 0.35 (0.12, 1.05); however, none had statistical significance.
CONCLUSIONS: This MAIC revealed consistent, nominal trends toward pacritinib efficacy benefits and OS in patients with myelofibrosis, most with moderate thrombocytopenia.
RESEARCH DESIGN AND METHODS: Data were from Phase 3 randomized controlled trials of patients with thrombocytopenic myelofibrosis treated with pacritinib 200 mg BID (PERSIST-2 [NCT02055781]) with baseline platelet count 24‒99 × 109/L matched on prognostic indicators with patients on momelotinib 200 mg QD (pooled post-hoc analysis of patients with platelet count <100 × 109/L (MOMENTUM [NCT04173494]; SIMPLIFY-1 [NCT01969838]). Week-24 endpoints included % patients achieving: ≥50% total symptom score reduction (TSS-50), ≥35% spleen volume reduction (SVR-35) and red-blood-cell-transfusion-independence (RBC-TI). Reweighted effects were compared with momelotinib using logistic regression/odds ratio (OR [95% confidence interval, CI]) and Cox regression/hazard ratio (HR [95% CI]) for overall survival (OS).
RESULTS: After matching, baseline characteristics balanced; 76‒81% of matched pacritinib patients had moderate thrombocytopenia (platelet count 50‒99 × 109/L). All outcomes favored pacritinib on ORs (95% CI) for TSS-50: 1.75 (0.60, 5.09); SVR-35: 1.40 (0.32, 6.04); RBC-TI: 1.19 (0.36, 3.97); and OS: HR (95% CI) 0.35 (0.12, 1.05); however, none had statistical significance.
CONCLUSIONS: This MAIC revealed consistent, nominal trends toward pacritinib efficacy benefits and OS in patients with myelofibrosis, most with moderate thrombocytopenia.