Abstract
The Janus kinase inhibitor ruxolitinib is approved for the treatment of myelofibrosis (MF) and improved overall survival (OS) versus control therapy in the phase 3 COMFORT trials. The aim of this retrospective analysis was to examine the real-world impact of ruxolitinib on OS in patients with MF. The US Medicare Fee-for-Service claims database (parts A/B/D) was used to identify patients with ≥ 1 inpatient or ≥ 2 outpatient claims with an MF diagnosis (January 2010–December 2017). Eligible patients with MF were ≥ 65 years old (intermediate-1 or higher risk based on age). Patients were divided into 3 groups based on ruxolitinib approval status at diagnosis and ruxolitinib exposure: (1) preapproval, ruxolitinib-unexposed; (2) post-approval, ruxolitinib-unexposed; and (3) post-approval, ruxolitinib-exposed. In total, 1677 patients with MF were included (preapproval [all ruxolitinib-unexposed], n = 278; post-approval, n = 1399 [ruxolitinib-unexposed, n = 1127; ruxolitinib-exposed, n = 272]). Overall, median age was 78 years, and 39.8% were male. Among patients with valid death dates (preapproval, n = 119 [42.8%]; post-approval, ruxolitinib-unexposed, n = 382 [33.9%]; post-approval ruxolitinib-exposed, n = 54 [19.9%]), 1-year survival rates were 55.6%, 72.5%, and 82.3%, and median OS was 13.2 months, 44.4 months, and not reached, respectively. Risk of mortality was significantly lower post- versus preapproval regardless of exposure to ruxolitinib (ruxolitinib-unexposed: adjusted hazard ratio [HR], 0.67; ruxolitinib-exposed: adjusted HR, 0.36; P < 0.001 for both); post-approval, mortality risk was significantly lower in ruxolitinib-exposed versus ruxolitinib-unexposed patients (adjusted HR, 0.61; P = 0.002). Findings from this study complement clinical data of ruxolitinib in MF by demonstrating a survival benefit in a real-world setting.
Highlights
Myelofibrosis (MF) is a chronic myeloproliferative neoplasm characterized by hyperproliferation of myeloid cells, bone marrow fibrosis, and burdensome constitutional symptoms [1,2,3]
In this real-world study of US patients diagnosed with intermediate- to high-risk MF, overall survival (OS) was significantly longer for patients who received ruxolitinib than for those who did not receive ruxolitinib, providing real-world data to complement the survival benefit observed in the COMFORT studies
Few other real-world studies have evaluated the effect of ruxolitinib or diagnosis period on survival in patients with MF
Summary
Myelofibrosis (MF) is a chronic myeloproliferative neoplasm characterized by hyperproliferation of myeloid cells, bone marrow fibrosis, and burdensome constitutional symptoms [1,2,3]. Ruxolitinib, a Janus kinase (JAK) 1 and JAK2 inhibitor, was approved by the US Food and Drug Administration in November 2011 for the treatment of adult patients with intermediate- to high-risk primary or secondary MF based on data from the phase 3 COMFORT-I and COMFORTII trials [9, 10]. Ruxolitinib significantly reduced spleen volume and improved MF-related symptoms and quality of life compared with placebo or best available therapy in the COMFORT studies [11, 12]. In the pooled analysis of COMFORT data, patients treated with ruxolitinib demonstrated improved OS compared with patients in the control arm (placebo, COMFORT-I; best available therapy, COMFORT-II; 5.3 versus 3.8 years, respectively; hazard ratio [HR], 0.70; 95% CI, 0.54–0.91; P = 0.007), and when patients were censored at crossover, the survival advantage was even greater (5.3 versus 2.4 years, respectively; HR, 0.53; 95% CI, 0.36–0.78; P = 0.001) [10]. Median OS for patients with MF enrolled in JUMP was 4.9 and 2.8 years among patients with intermediate-2 and high-risk MF, respectively [14]
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
