POS1380 LONG-TERM EFFICACY AND SAFETY OF CANAKINUMAB IN PATIENTS WITH TRAPS (TUMOR NECROSIS FACTOR RECEPTOR-ASSOCIATED PERIODIC SYNDROME) - INTERIM ANALYSIS OF THE RELIANCE REGISTRY
Authors: N. Blank, J. Henes, T. Kallinich, P. Oommen, C. Schuetz, M. Borte, J. Weber-Arden, J. B. Kuemmerle-Deschner
Journal: Annals of the Rheumatic Diseases (2021)

Background: Tumor necrosis factor receptor-associated periodic syndrome (TRAPS) is a rare autoinflammatory condition characterized by severe systemic and organ inflammation. In a phase 3 pivotal trial ( CLUSTER study), TRAPS patients have been successfully treated with the interleukin-1β inhibitor canakinumab. 45% of patients reached clinical remission after 16 weeks (primary endpoint) 1 . Canakinumab has been approved and applied for the treatment of TRAPS patients since 2017 2 . Objectives: The present study explores the long-term efficacy and safety of canakinumab under routine clinical practice conditions in pediatric (age ≥2 years) and adult TRAPS patients. Methods: RELIANCE is a prospective, non-interventional, multi-center, observational study based in Germany with a 3-year follow-up period. Patients with clinically confirmed diagnoses of TRAPS, CAPS, FMF or HIDS/MKD who routinely receive canakinumab are enrolled in order to evaluate efficacy and safety of canakinumab under standard clinical practice conditions. Disease activity and remission by physician assessment, disease activity and fatigue by patient assessment, days absent from school/work due to study indication, inflammatory markers, and AIDAI (Auto-Inflammatory Diseases Activity Index) score were assessed at baseline and at 6-monthly intervals. Results: The interim analysis of TRAPS patients enrolled by December 2020 includes baseline (N=16, including 1 patient with atypical TRAPS) and preliminary 18-month data. Mean age in this cohort was 23 years (3−43 years) and the median duration of prior CAN treatment was 1.0 year (0−4 years). Physician assessment indicated 60-80% remission and laboratory parameters were within normal range. Disease control by patient assessment showed no major changes regarding the analyzed parameters (Table 1, Figure 1). Of the three serious adverse events reported none was classified as drug-related. Conclusion: Preliminary analysis of 18 month interim data of TRAPS patients treated with CAN available from the RELIANCE study indicate stable efficacy and safety of CAN long-term treatment. References: [1]De Benedetti F, et al. Canakinumab for the treatment of autoinflammatory recurrent fever syndromes. N Engl J Med 2018;378:1908–19. [2]Ilaris, INN-canakinumab (europa.eu) Table 1. Baseline characteristics and interim analysis data of patients with TRAPS Baseline 6 months 12 months 18 months Number of patients, N 16 13 10 6 Median age, years (min; max) 23 (3; 43) 17 (3; 43) 16 (4; 38) 25 (4; 43) Females (%) 11 (69) 9 (69) 7 (70) 3 (50) Median duration of prior CAN therapy at baseline, years (min; max) 1.0 (0; 4) 1.0 (0; 4) 1.0 (0; 4) 1.5 (0; 2) Number (%) of patients in disease remission (physician assessment) 9 (60.0) 9 (81.8) 7 (77.8) 4 (80.0) Physician Global Assessment, percentage of absent/mild-moderate/severe rating 40 / 53 / 0 82 / 9 / 0 44 / 44 / 11 80 / 20 / 0 Patient assessment of current disease activity; 0–10, median (min; max) 1.5 (0; 5) 1.0 (0; 4) 1.0 (0; 6) 0.0 (0; 3) Patient assessment of current fatigue; 0–10, median (min; max) 2.0 (0; 8) 1.0 (0; 7) 2,5 (0; 8) 4.0 (0; 7) Number (%) of patients without impairment of social life by the disease 4 (50) 5 (63) 2 (33) 3 (60) Number (%) of patients with days absent from work/school during last 6 months 8 (50) 5 (39) 5 (56) 3 (50) CRP, median (mg/dl) 0.1 0.1 0.1 0.0 SAA, median (mg/dl) 0.5 0.4 0.4 0.3 ESR, median (mm/h) 7.0 5.0 5.0 5.0 SAE Number of events Incidence rate per 100 patient years Total 3 14.7 Circulatory collapse (non fatal) 1 4.9 Dizziness 1 4.9 Headache 1 4.9 CRP, c-reactive protein; ESR , erythrocyte sedimentation rate; SAA, serum amyloid A; SAE, serious adverse event Disclosure of Interests: Norbert Blank Consultant of: Novartis, Sobi, Lilly, Pfizer, Abbvie, BMS, MSD, Actelion, UCB, Boehringer-Ingelheim, Roche, Grant/research support from: Novartis, Sobi, Jörg Henes Consultant of: Novartis, AbbVie, Sobi, Roche, Janssen, Boehringer-Ingelheim, Grant/research support from: Novartis, Roche, Tilmann Kallinich Consultant of: Sobi, Novartis, Roche, Grant/research support from: Novartis, Prasad Oommen Grant/research support from: Novartis, Catharina Schuetz: None declared, Michael Borte Grant/research support from: Pfizer, Shire, Julia Weber-Arden Employee of: Novartis, J. B. Kuemmerle-Deschner Consultant of: Novartis, AbbVie, Sobi, Grant/research support from: Novartis, AbbVie, Sobi