Ten-year journey with myelofibrosis: real-world data and clinical outcomes from a tertiary care center

Aims: The aim of this study was to document the last decade of experience with primary myelofibrosis (PMF) and provide mature risk-stratified survival data and disease complication estimates. Methods: This retrospective study included all patients diagnosed with PMF according to World Health Organization criteria between 2009 and 2019 at İstanbul Medipol University. The follow-up period extended until March 2019. Results: A total of 74 patients (median age 60.5 years, range 30-85) were included, with 35 males (47.3%) and 39 females (52.7%). During the study period, 34 deaths (45.9%), 3 leukemic transformations (4%), and 3 thrombotic events (4.2%) were recorded, including bilateral pulmonary artery thrombosis, cephalic vein thrombosis, and left lower extremity deep vein thrombosis. The median overall survival was 6 years. There was no statistically significant difference in life expectancy between patients treated with ruxolitinib and those receiving other medications (erythropoiesis-stimulating agents, androgens, immunomodulatory agents, cytoreductive drugs such as hydroxyurea, and splenectomy) (chi-square statistic: 3.5149, p = 0.0608). One of the 11 patients treated with ruxolitinib died (9%), while the remaining 10 patients survived (91%). Conclusion: PMF remains a serious disease with high mortality and morbidity, requiring effective treatment interventions. Despite recent advances, treatment remains unsatisfactory for most patients. Although ruxolitinib, a JAK2 inhibitor, shows promise in managing symptoms, its impact on life expectancy remains unclear, highlighting the need for novel therapeutic strategies and well-designed clinical trials.