The role of therapy in the outcome of patients with myelofibrosis

• Published in: Cancer Vol. 129; no. 18; pp. 2828 - 2835
• Main Authors: Masarova, Lucia, Bose, Prithviraj, Pemmaraju, Naveen, Daver, Naval G., Sasaki, Koji, Chifotides, Helen T., Zhou, Lingsha, Kantarjian, Hagop M., Estrov, Zeev, Verstovsek, Srdan
• Format: Journal Article
• Language: English
• Published: United States Wiley Subscription Services, Inc 15.09.2023
• Subjects: Fibrosis; Immunomodulation; Inhibitors; initial therapy; JAK inhibitors; Myelofibrosis; Oncology; outcome; Pharmacology; Survival; Therapy; initial therapy; myelofibrosis; JAK inhibitors; outcome
• ISSN: 0008-543X; 1097-0142
• DOI: 10.1002/cncr.34851

BACKGROUND The 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. METHODS In 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. RESULTS Overall, 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). CONCLUSIONS This study demonstrated improved outcomes of patients with MF who received treatment with the JAK inhibitor ruxolitinib. This single‐center study suggests that ruxolitinib played a central role in improved survival among patients with myelofibrosis. Patients who are not eligible for ruxolitinib require novel therapies.