Abstract

Previous studies have reported the survival benefit after ruxolitinib treatment in patients with myelofibrosis (MF). However, population-based data of its efficacy are limited. We analyzed the effects of ruxolitinib in MF patients with data from the Korean National Health Insurance Database. In total, 1199 patients diagnosed with MF from January 2011 to December 2017 were identified, of which 731 were included in this study. Patients who received ruxolitinib (n = 224) were matched with those who did not receive the drug (n = 507) using the 1:1 greedy algorithm. Propensity scores were formulated using five variables: age, sex, previous history of arterial/venous thrombosis, and red blood cell (RBC) or platelet (PLT) transfusion dependence at the time of diagnosis. Cox regression analysis for overall survival (OS) revealed that ruxolitinib treatment (hazard ratio (HR), 0.67; p = 0.017) was significantly related to superior survival. In the multivariable analysis for OS, older age (HR, 1.07; p < 0.001), male sex (HR, 1.94; p = 0.021), and RBC (HR, 3.72; p < 0.001) or PLT (HR, 9.58; p = 0.001) transfusion dependence were significantly associated with poor survival, although type of MF did not significantly affect survival. Considering evidence supporting these results remains weak, further studies on the efficacy of ruxolitinib in other populations are needed.

Highlights

  • The present study aimed to evaluate efficacy of ruxolitinib based on real-world population data from the Korean National Health Insurance (NHI) Database (NHID)

  • In our nationwide population-based analysis, ruxolitinib treatment appeared to be associated with better survival in patients with MF

  • Previous phase 3 trials have reported the favorable effects of ruxolitinib in patients with MF [6,7]

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Summary

Introduction

Publisher’s Note: MDPI stays neutral with regard to jurisdictional claims in published maps and institutional affiliations. Primary myelofibrosis (MF) is a subgroup of stem cell-derived clonal myeloproliferative neoplasms, characterized by clinical manifestations including severe anemia, thrombocytosis, bleeding, and splenomegaly [1]. MF that occurs following prior polycythemia vera (PV) or essential thrombocythemia (ET) is known as secondary MF [2]. According to the Korea National Cancer Incidence Database, the incidence rates of MF increased in the country from 0.08 per 100,000 person-years in 2003 to 0.15 per 100,000 person-years in

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