Real-world outcomes of momelotinib in myelofibrosis patients with anaemia

Dr Lucia Perez-Lamas, a haematologist at the Puerta de Hierro University Hospital in Madrid, Spain, presented real-world evidence on the use of momelotinib in myelofibrosis (MF) patients with anaemia. 

A retrospective, multicenter study was conducted to evaluate the safety and efficacy of momelotinib in patients diagnosed with MF who presented with disease-related symptoms and/or splenomegaly and anaemia, and who received momelotinib through a managed-access program. Patients could have been either JAK inhibitor-naïve or previously treated with a JAK inhibitor. 

A total of 76 patients from 42 Spanish centres were enrolled, with a mean age at diagnosis of 69 years. 48.6% of patients had haemoglobin levels below 10 g/dL. Prior to starting momelotinib, 14 patients (18.4%) had not received any previous treatment, while the remaining patients had undergone an average of 1.45 prior therapies. A significant majority had used erythropoiesis-stimulating agents (ESA) before initiating momelotinib. The median duration on momelotinib was 120 days.

Prior to the initiation of momelotinib, patients had a mean haemoglobin level of 8.1 g/dL, with 74% of them being transfusion-dependent. Following 120 days of treatment, the mean haemoglobin level increased by approximately 1.5 g/dL. Additionally, 44% of transfusion-dependent patients achieved transfusion independence, with a higher rate observed in second-line therapy (45%) compared to first-line therapy (37%). For patients who remained transfusion-dependent, the average number of red blood cell units required per month decreased from 3.8 to 2.6 units.

Approximately 80% of second-line patients reported improvements in symptoms, with 68% experiencing reduced ischemia, 50% showing decreased anorexia, and 43% reporting weight stabilization or gain. These improvements were comparable in first-line patients, indicating consistent efficacy across treatment lines. Around half of the patients reported significant improvement in splenomegaly.

In terms of safety, the most common haematologic adverse event observed was thrombocytopenia, occurring in approximately 12% of patients, The most frequent non-haematologic adverse event was gastrointestinal symptoms, reported in around 30% of patients; however, all of these cases were mild. At the last follow-up, 79% of patients remained on momelotinib.

In conclusion, the data support a favourable efficacy profile for momelotinib in improving splenomegaly and symptoms in myelofibrosis patients, as well as significant improvements in anaemia, with nearly half of the patients achieving transfusion independence.