IMPROVED HEALTH STATUS OF HAEMOPHILIA B PATIENTS TREATED WITH NONACOG BETA PEGOL, A NEW GLYCOPEGYLATED RECOMBINANT FIX PRODUCT WITH PROLONGED HALF-LIFE
Author(s)
Chowdary P1, Kearney S2, Yee DL3, Meunier J4, Slothuus U5, Hoxer CS5
1Royal Free Hospital, London, UK, 2Children's Hospital and Clinics of Minnesota, Minneapolis, MN, USA, 3Texas Children's Hemophilia & Thrombosis Center, Houston, TX, USA, 4Mapi, Lyon, France, 5Novo Nordisk A/S, Søborg, Denmark
OBJECTIVES: To assess the health status of haemophilia B patients treated with nonacog beta pegol, a novel recombinant Factor IX (FIX) product with prolonged half-life and less frequent dosing requirements. METHODS: Severe/moderate haemophilia B (FIX activity level ≤2%) is an inherited bleeding disorder characterised by recurrent, often spontaneous bleeding episodes, in particular musculoskeletal bleeds. Modern management includes self-infusion of FIX 2-3 times/week to prevent bleeds (prophylaxis) or on-demand treatment (OnD). Haemophilia B patients aged 13-70 years were included in a single-blind phase 3 trial evaluating safety and efficacy of nonacog beta pegol. Patients were treated OnD for 28 weeks, or randomised to once weekly prophylaxis with 10 or 40 IU/kg for 52 weeks. All patients completed the 3 level EuroQol-5 dimensions (EQ-5D-3L) at baseline and end-of-trial. EQ-5D VAS ranges from 0-100. Higher score represents better health status. Changes in scores were compared to no change using the Wilcoxon signed rank test. RESULTS: A significant improvement in EQ-5D index score was observed in the overall set (mean change =0.04, p=0.016), but no significant change was observed for the different dose groups. In subset analysis of the high dose prophylaxis arm, mean EQ-5D VAS was 74.3±18.0 at baseline and 83.9±14.5 at end-of-trial, leading to a statistically significant improved mean change in score (p=0.030). The change was non-significant in the OnD and low dose prophylaxis arms. CONCLUSIONS: In a phase 3 clinical trial, haemophilia B patients treated with the long-acting FIX nonacog beta pegol showed a significant improvement in their health status. Patients treated with the prophylaxis dose of 40 IU/kg/weekly demonstrated significant improvement in health status according to the EQ-5D VAS.
Conference/Value in Health Info
2015-05, ISPOR 2015, Philadelphia, PA, USA
Value in Health, Vol. 18, No. 3 (May 2015)
Code
PSY49
Topic
Patient-Centered Research
Topic Subcategory
Health State Utilities, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Systemic Disorders/Conditions