Momelotinib for the treatment of myelofibrosis with anemia.
Douglas TremblayRuben A MesaPublished in: Future oncology (London, England) (2022)
Myelofibrosis is a myeloproliferative neoplasm characterized by splenomegaly, debilitating constitutional symptoms and bone marrow failure. Disease-related anemia is common and associated with an inferior quality of life and survival. Unfortunately, few therapies exist to improve hemoglobin in myelofibrosis patients. Momelotinib is a JAK1/JAK2 inhibitor that also antagonizes ACVR1, leading to downregulation of hepcidin expression and increased availability of iron for erythropoiesis. In clinical testing, momelotinib has demonstrated a unique ability to improve hemoglobin and reduce transfusion burden in myelofibrosis patients with baseline anemia, while producing reductions in spleen size and symptom burden. This review explores the preclinical rationale, clinical trial data and future role of momelotinib in the evolving therapeutic landscape of myelofibrosis.
Keyphrases
- iron deficiency
- chronic kidney disease
- end stage renal disease
- clinical trial
- bone marrow
- poor prognosis
- ejection fraction
- newly diagnosed
- risk factors
- electronic health record
- prognostic factors
- cell proliferation
- big data
- randomized controlled trial
- open label
- machine learning
- stem cells
- patient reported
- depressive symptoms
- cardiac surgery
- study protocol
- binding protein
- high grade