Improving symptom burden and quality of life in patients with myelofibrosis: current strategies and future directions

Abstract

Introduction: Myelofibrosis (MF) is a complex and aggressive hematologic malignancy resulting from JAK/STAT-driven myeloproliferation and abnormal fibrogenesis. The clinical manifestations are heterogeneous and negatively impact quality of life and survival. JAK inhibitors improve symptoms and splenomegaly to a variable degree in a proportion of patients, but the effects for many patients are insufficient or short-lived. Areas covered: This review examines the constellation of symptoms that befall patients with MF, describes methods to quantify and serially monitor these symptoms, and evaluates pharmacologic and non-pharmacologic interventions for disease-related symptoms. The review also includes a discussion of areas of unmet medical need, and proposes future methods for meeting this need. Expert opinion: The treatment landscape for MF is evolving rapidly. The most effective therapies or combinations of therapies will likely simultaneously impact both the malignant hematopoietic stem cell and mechanisms of aberrant fibrogenesis that drive this disease. The goals of treatment for patients with myelofibrosis should be to improve length and quality of life. Clinical trials must be designed with these goals in mind, with endpoints focused on overall survival and symptom reduction, as opposed to surrogate endpoints such as spleen volume reduction.