OP0254\u2005CANAKINUMAB IMPROVES PATIENT-REPORTED OUTCOMES IN PATIENTS WITH RECURRENT FEVER SYNDROMES: RESULTS FROM A PHASE 3 TRIAL (CLUSTER)

Abstract

Background Recurrent fever syndromes have a significant impact on health-related quality of life (HRQoL).1 Canakinumab (CAN) has demonstrated efficacy and safety in patients with colchicine-resistant familial Mediterranean fever (crFMF), hyper-immunoglobulin D syndrome/mevalonate kinase deficiency (HIDS/MKD) and tumour necrosis factor receptor-associated periodic syndrome (TRAPS) in the pivotal, Phase 3, CLUSTER trial (NCT02059291),2 but there are limited published data on the impact of CAN on the HRQoL, work/school and social life of these patients. Objectives To evaluate effect of CAN on HRQoL, work/school and social life of patients in the 3 disease cohorts (crFMF, HIDS/MKD, and TRAPS) in a double blinded randomised study. Methods The detailed study design was reported previously.2 The HRQoL of patients treated with CAN was assessed at Baseline (BL), Week 17 (Wk17) and Week 41 (Wk41) in patients who fully responded (absence of flares), either to 150 mg q4w CAN, or to 300 mg q4w CAN after up dosing. Methods used were the Child Health Questionnaire (CHQ)-PF50 physical (PhS) and psychosocial (PsS) summary scores (children >5–<18 years), Short-Form health survey (SF-12) physical component summary (PCS) and mental component summary (MCS; adults ≥18 years) scores. An increase from baseline of 2, 5, and 8 points in the CHQ-PF50 physical and psychological component summary scores corresponds to a small, moderate and large treatment effect, respectively. Functional impairment related to work/school, social life and family life/home responsibilities was assessed by Sheehan Disability Scale (SDS). A score of 5 or higher in SDS is associated with significant impairment. Results Patients showed a high impairment of HRQoL at baseline in all 3 cohorts (crFMF n=31, HIDS/MKD n=37 and TRAPS n=22). At Wk17, a moderate to large treatment effect, either with 150 mg or 300 mg q4w CAN, was observed by an improvement in the CHQ-PF50 PhS and PsS (increased >5), SF-12 PCS (increased >5) and SDS scores (decreased below 2). At Wk41, the number of patients on CAN was limited (crFMF n=9, HIDS/MKD n=6 and TRAPS n=4) due to the design of the trial (randomised withdrawal part). For the patients who remained on CAN, the improvement in HRQoL, work/school and social life was sustained. Conclusion Treatment with CAN led to sustained improvement of HRQoL, work/school and social life in patients with crFMF, HIDS/MKD and TRAPS. References [1] Sahin, et al. Eur Rev Med Pharmacol Sci. 2013;17:958–963. [2] De Benedetti, et al. NEJM 2018;378:1908–1990. Disclosure of Interests Helen J. Lachmann Grant/research support from: SOBI, Novartis, Consultant for: Novartis, Takeda, Speakers bureau: SOBI. Novartis, Bernard Lauwerys: None declared, Paivi Miettunen: None declared, Tilmann Kallinich Grant/research support from: Novartis, Speakers bureau: Sobi, Roche, Novartis, CLB, Gerd Horneff: None declared, Riva Brik: None declared, Rafaelle Manna: None declared, Sara Murias: None declared, Sinisa Savic Grant/research support from: Novartis and Sobi, Serge Smeets Employee of: Novartis, Fabrizio De Benedetti Grant/research support from: Abbvie, SOBI, Novimmune, Roche, Novartis, Sanofi, Pfizer, Anna Simon: None declared