Making a case for disease-modifying agents in myelofibrosis

JAK inhibitors are currently the cornerstone of non-transplantation treatment strategies in intermediate and high-risk myelofibrosis.1 Ruxolitinib, the first approved JAK inhibitor in this setting, rapidly reduces splenomegaly and controls symptoms in a substantial proportion of patients,2,3 alongside conferring a survival benefit that seems to occur in the absence of a consistent disease-modifying ability.4 However, high discontinuation rates by the third year of treatment and dismal outcomes of patients after stopping ruxolitinib5 underline the need for effective second-line treatments and, ideally, for improved front-line options.