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Momelotinib is an emerging agent that inhibits ACVR1, in addition to JAK1 and JAK2, resulting in improvements in erythropoiesis.

Pacritinib has recently been approved for patients with a platelet count of less than 50, a group for whom prior JAKi were contraindicated based on the FDA label. Nonetheless, pretreatment or treatment-emergent anemia and/or thrombocytopenia remain complicating factors.Įxcitingly, new JAKi are emerging, with the potential to extend the benefit of JAKi to cytopenic patients with MF. In the COMFORT studies, half of patients had discontinued treatment by 3 years,3 whereas real-world data have demonstrated a median duration of treatment as low as 13 months.4 Novel ruxolitinib dosing strategies in patients with anemia may improve tolerability,5 and anemia-directed therapies can also be employed. Ruxolitinib has the longest track record of both safety and success, initially demonstrated by the COMFORT trials (NCT00952289, NCT00934544).1,2 However, cytopenias can be a challenge, and real-world data have highlighted the frequency of ruxolitinib dose interruptions and early treatment discontinuation. JAKi have consistently demonstrated improvements in splenomegaly and disease-related symptoms, with long-term follow-up also showing an overall survival benefit. However, in the absence of a suitable clinical trial, treatment with a JAKi is the standard of care in this setting. Patients with significant symptomatology, should be assessed for clinical trials. Moreover, hematopoietic stem cell transplant candidacy should be considered at diagnosis, as this remains the only potentially curative therapy for patients with MF. With multiple JAKi now options in this treatment space, patient- and disease-specific factors are vital when determining the most appropriate therapy plans. Symptom and spleen assessments, the presence of cytopenias, and risk stratification are key factors to consider prior to treatment with a JAKi.

Following the FDA approvals of the JAK inhibitors (JAKi) ruxolitinib (Jakafi), fedratinib (Inrebic), and pacritinib (Vonjo), the treatment landscape of myelofibrosis (MF) continues to grow with the use of these agents with an additional FDA review planned for momelotinib in September 2023.