The role of therapy in the outcome of patients with myelofibrosis

Abstract

BACKGROUNDThe treatment of patients with myelofibrosis (MF) has evolved in the past decade, as reflected in an increased use of various therapeutic agents that could potentially impact patient outcomes.METHODSIn this retrospective study, the authors evaluated the pattern of therapy and its possible impact on the survival of patients with MF at their institution. Patients (n = 802) with newly diagnosed, chronic, overt MF (MF fibrosis grade ≥2, <10% blasts) seen at their cancer center between 2000 and 2020 were included.RESULTSOverall, 492 of the included patients (61%) initiated MF‐directed therapy during follow‐up. The most frequent initial therapy was the JAK inhibitor ruxolitinib (44% of treated patients), investigational agents excluding JAK inhibitors (21%), immunomodulatory agents (18%), other investigational JAK inhibitors (10%), and others (7%). Overall survival was superior for patients who received initial ruxolitinib therapy, with a median survival of 72 months versus approximately 50 months for the remaining approaches, excluding the last group. Thirty‐two percent of patients required subsequent therapy (n = 159). The longest survival since the start of second‐line therapy was observed in patients who initiated salvage ruxolitinib (median, 35 months; 95% CI, 25–45 months).CONCLUSIONSThis study demonstrated improved outcomes of patients with MF who received treatment with the JAK inhibitor ruxolitinib.

Association of Myelofibrosis Phenotypes with Clinical Manifestations, Molecular Profiles, and Treatments

“…Synergistic combination regimens, such as pelabresib or navitoclax with ruxolitinib, may increase the depth and duration of spleen and symptom responses and improve other aspects of the disease, such as driver mutation burden and bone marrow fibrosis in MF patients presenting with the myeloproliferative phenotype [ 79 , 87 ]. Prolongation of survival, currently the primary endpoint of the pivotal trial of imetelstat, would be a welcome advancement in the field preceded by the transformative impact of ruxolitinib [ 157 , 158 , 159 , 160 , 161 ]. Achievement of RBC transfusion independence is the primary endpoint of the INDEPENDENCE trial of luspatercept and is likely to be seen more and more in registrational trials [ 87 ], given that anemia is a cardinal feature of MF and achievement of RBC transfusion independence was correlated with superior OS in the SIMPLIFY-1 trial [ 162 ].…”