1. After a 24-week treatment period, a significantly greater proportion of patients randomized to momelotinib had symptom scores reduced by more than 50%.
2. Momelotinib was well tolerated overall with the most common adverse events being anemia and thrombocytopenia.
Evidence Rating Level: 1 (Excellent)
Study Rundown: Myelofibrosis is a malignancy of the bone marrow that typically causes a low red blood cell count. Patients with myelofibrosis can be affected by debilitating symptoms such as weakness and fatigue. Momelotinib is an activin A receptor type 1 (ACVR1) inhibitor that has shown promise in previous phase 1-3 clinical trials in myelofibrosis. This study aimed to efficacy of momelotinib versus danazol in reducing the symptoms of myelofibrosis. Participants were enrolled to receive either momelotinib or danazol for a total of 24 weeks. In result, a significantly greater proportion of patients in the momelotinib group compared to the danazol group reported symptom reductions by more than 50%. Adverse events were similar between the two groups, with the most common being anemia and thrombocytopenia. Limitations of this study include the inability to assess long-term survival benefits between the two groups. Nonetheless, this study supports the use of momelotinib for treating myelofibrosis-associated symptoms.
Click to read the study in The Lancet
In-Depth [randomized controlled trial]: MOMENTUM was an international, randomized controlled phase 3 trial evaluating momelotinib versus danazol in patients with anemia and myelofibrosis. Participants were aged 18 years or older with confirmed primary myelofibrosis, post-polycythemia vera, or post-essential thrombocythemia myelofibrosis. A total of 195 patients were randomized 2:1 to either momelotinib 200 mg orally once per day plus danazol placebo (n=130) or danazol 300 mg orally twice per day plus momelotinib placebo (n=65). The treatment duration was 24 weeks. The primary endpoint was defined as the response rate at week 24 measured by a >50% reduction in the Myelofibrosis Symptom Assessment Form (MFSAF) TTS compared to baseline. The primary endpoint occurred in a significantly greater portion of the momelotinib group compared to the danazol group (25% vs. 9%, proportional difference 16% [95% CI 6-26], p=0.0095). The most frequent grade 3 adverse events associated with momelotinib and danazol were anemia (61% vs 75%) and thrombocytopenia (28% vs 26%). Other non-hematological adverse events include acute kidney injury (3% vs 9%) and pneumonia (2% vs 9%).