OP0092 LONG-TERM SAFETY AND EFFECTIVENESS OF CANAKINUMAB IN CRYOPYRIN-ASSOCIATED PERIODIC SYNDROMES (CAPS) – 30-MONTH DATA FROM THE RELIANCE REGISTRY

Background: In clinical trials as well in real-life, the IL-1ß inhibitor canakinumab leads to rapid remission of symptoms treatment CAPS, a monogenic autoinflammatory disease with severe systemic and organ inflammation. Objectives: The RELIANCE registry is designed explore long-term safety effectiveness under routine practice conditions pediatric (≥2 years) adult patients including Muckle-Wells syndrome (MWS), familial cold (FCAS), neonatal onset multisystem inflammatory (NOMID)/chronic infantile neurological cutaneous articular (CINCA). Methods: This prospective, non-interventional, observational study 3-year follow-up enrolls clinically confirmed diagnoses CAPS routinely receiving canakinumab. 6-monthly visits, data, physician assessments patient-reported outcomes are evaluated starting at baseline last update 30 months total cohort subtypes (NOMID/CINCA). Results: 91 (50% female; 14 [15%] NOMID/CINCA subtypes) were enrolled by December 2020 (Table 1). At baseline, median age was 20.5 years duration prior 6 years. 11 drug related adverse events per 100 patient reported. 68% reached physicians´ assessment along rates 40-61% absent activity PGA. Patients reported stable low level activity, fatigue Auto-Inflammatory Diseases Activity Index scores (AIDAI, figure impairing social life 50% another days off from school/work. Lab parameters within normal limits. Table 1. Patient laboratory markers over time. Baseline 12 Total NOMID/ CINCA Number patients, N 67 8 28 4 (%) work/school during (34) (29) (42) 2 (25) 17 (61) (100) (physician assessment) 61 (68.5) (78.6) 42 (66.7) 19 (67.9) (100.0) Physician Global Assessment, percentage absent/mild-moderate/severe rating, % 40 / 53 57 36 0 33 60 50 39 75 25 current activity; 0–10, (min; max) 2.0 (0; 7) 1.0 6) 5) 0.0 4) fatigue; 3.0 9) 8) 4.0 without impairment 32 (52.5) (50.0) 31 (62.0) 3 (42.9) (47.8) 1 (33.3) CRP, (mg/dl) 0.1 0.2 0.5 SAA, 0.3 0.4 0.9 ESR, (mm/h) 5.0 6.0 3.5 5.5 SAE Incidence rate All types 14.72 11.72 SADR 20 # 10.69 0.00 Alport’s syndrome, appendicitis, blister, cardiovascular disorder, chest pain, circulatory collapse, erythema, febrile convulsion, glomerulonephritis, haemophilus test positive, pneumonia, premature delivery, skin discolouration, tonsillectomy, tonsillitis bacterial, streptococcal (all N=1 event), pyrexia (N=3 events), not yet coded (inpatient admission, event) c-reactive protein; erythrocyte sedimentation rate; n. a., annotated; serum amyloid A; SADR, serious reaction; SAE, event Conclusion: 30-month interim analysis demonstrates that safe effective any subtype CAPS. However, school/work still exists. Disclosure Interests: J. B. Kuemmerle-Deschner Consultant of: Novartis, AbbVie, Sobi, Grant/research support from: Birgit Kortus-Goetze Prasad Oommen Ales Janda: None declared, Jürgen Rech Speakers bureau: bbvie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD; Mylan, Roche, Sanofi, UCB, Abbvie, MSD, Tilmann Kallinich Frank Weller-Heinemann: Gerd Horneff Bayer, Merck Sharp & Dohme, Pfizer, Ivan Foeldvari Catharina Schuetz: Michael Borte Shire, Axel Braner Novartis SOBI, Julia Weber-Arden Employee Norbert Blank Actelion, Boehringer-Ingelheim, Sobi