Momelotinib efficacy and safety in MF: Retrospective real-world data

Results from a retrospective, multicenter, Italian real-world study, evaluating the safety and efficacy of momelotinib in 39 consecutive patients with myelofibrosis (MF) and anemia, regardless of prior therapy, were published in the European Journal of Haematology by Martorelli et al. The primary endpoint was improvement in anemia. Secondary endpoints were spleen longitudinal diameter reduction, safety, overall survival (OS), time on treatment (ToT), rate of acute myeloid leukemia (AML) transformation, symptom improvement, and rate of hemoglobin level increase >1.5 or 1 g/dL at 24 weeks.

Key data: The overall response rate (ORR) was 56% (major response, 37%; minor response, 19%) in transfusion-dependent (TD) patients, 54% (major response, 31%; minor response, 23%) in severely TD patients, and 46% (major response, 27%; minor response, 19%) in transfusion-independent (TI) patients. The median time to achieve transfusion independence was 55 days (range, 31–360 days). At 24 weeks of treatment, hemoglobin increase >1.5 g/dL was observed in 33% of patients, symptom improvement in 51%, and spleen volume reduction (SVR) >35% in 28%. The median OS was not reached (NR), while the 1-year OS was 85% (95% confidence interval [CI], 68–100%). Treatment discontinuation occurred in 18% of patients, attributed largely to hematological toxicity (2%), death (2%), lack of response (5%), other neoplasms (2%), and unknown reasons (2%).

Key learning: Momelotinib demonstrated substantial clinical efficacy and a favorable safety profile in heavily pretreated patients with MF and anemia in real-world clinical practice, supporting its potential as an effective therapeutic option for managing anemia in this challenging patient population.