Ruxolitinib Strengthens Survival Outcomes Among High-Risk Patients With Myelofibrosis

A Population-Based Retrospective Cohort Study

Ruxolitinib treatment strengthened survival outcomes among high-risk patients with myelofibrosis (MF), according to findings from a population-based retrospective cohort study published in Blood. 

Barbara Mora, MD, ASST Sette Laghi, Varese, Italy, and coauthors stated, “Patients in the higher prognostic risk categories have a median overall survival (OS) ranging between 2 and 4 years after diagnosis,” of myelofibrosis. Since being approved for the treatment of MF, the incorporation of ruxolitinib has shown an increase in patients’ OS in a variety of trials. 

In this study, Dr Mora and study authors aimed to analyze the outcomes of higher-risk patients with MF treated with ruxolitinib in an unselected real-world population. The primary end point was OS, defined as the time between the start of ruxolitinib treatment and death for any cause, censoring due to migration, end of data availability, or stem cell transplant. The secondary end point was a measure of the risk factors associated with mortality. 

A total of 652 high-risk or intermediate-2 patients with myelofibrosis from the Regional Health Service were included in this study, utilizing their databases from the Lombardy, Tuscany, and Lazio regions in Italy. Ruxolitinib was administered at an initial dose of 5 mg BID among 32% of cases and at least 20 mg BID among 25.6% of the entire cohort. Results indicated that the median OS was 47.7 months. At a median follow-up of 39.8 months, 55.8% of patients (n = 364) died. The OS rate at 1 year was 85%, at 3 years was 63%, and at 6 years was 34%. 

The study authors noted that the factors independently associated with mortality included gender, older age, high Multisource Comorbidity Score (MCS), and receiving less than 20 mg BID ruxolitinib as an initial dose. In terms of safety, the most frequent adverse events were infections, bleeding events, and thrombosis. 

Mora and study authors concluded, “In this study on 652 [intermediate-3] (INT-2) and [high-risk] (HR) MF patients observed for a minimum follow-up period of 3.5 years, [ruxolitinib] (RUX) demonstrates to have a robust effect on survival, underpinning its use at appropriate doses in the management of these patients.”

Source: 

Mora B, Pellegrini G, Franchi M, et al. A Population-Based Study on Survival of 652 Higher Risks Myelofibrosis Patients Treated with Ruxolitinib in Italy. Blood. Published online: November 28, 2023. doi: 10.1182/blood-2023-182107