POS0220 LONG-TERM SAFETY AND EFFECTIVENESS OF CANAKINUMAB IN CRYOPYRIN-ASSOCIATED PERIODIC SYNDROMES – 36-MONTH DATA FROM THE RELIANCE REGISTRY

Background The cryopyrin-associated periodic fever syndromes (CAPS) are hereditary monogenic autoinflammatory diseases with severe systemic and organ inflammation due to increased production of Interleukin-1β (IL-1β). subcutaneously administered monoclonal antibody canakinumab (CAN) effectively inhibits IL-1β results in rapid remission CAPS symptoms clinical trials as well real-life. Objectives RELIANCE registry is designed explore long-term safety effectiveness CAN under routine practice conditions pediatric (≥2 years) adult patients CAPS, including Muckle-Wells syndrome (MWS), familial cold (FCAS), neonatal onset multisystem inflammatory disease (NOMID)/chronic infantile neurological cutaneous articular (CINCA). Methods This prospective, non-interventional, observational study a 3-year follow-up enrolls Germany clinically confirmed diagnoses routinely receiving CAN. In 6-monthly visits, data, physician assessments patient-reported outcomes evaluated starting at baseline. Results 98 (52% female; 15 [15%] NOMID/CINCA subtypes) were enrolled by December 2021 (Table 1). At baseline, median age was 20 years duration prior treatment 6 years. the 36 months visit, 74% reached physicians´ assessment along increasing rates absent activity (patient’s assessment, 2.0 baseline 0.0 month 36). addition, reported low levels fatigue (absent mild/moderate: 87% 95% impaired social life 47% (37% 36) 33% (23% days off from school/work. Lab parameters within normal limits. Remission control sustained remained stable or even decreased over time. Table 1. Patient laboratory markers Baseline 12 Number patients, N 72 40 (%) (physician assessment) 64 (68) 48 (70) 28 (74) Patient’s current activity; 0–10, (min; max) (0; 7) 6) fatigue; 3.0 9) 8) 1.0 without impairment 34 (53) 35 (65.0) 17 (63) CRP (mg/dl) | SAA (mg/dl); 0.1 0.3 0.5 disease-related inclusion into Fever 75 (80) 14 (15) 19 (28) 4 (11) Fatigue 84 (89) 49 (52) (46) Conjunctivitis/Uveitis 63 (67) 27 (29) 21 (30) 7 (19) Headache 68 (72) 30 (32) (43) 9 (24) Arthralgia/arthritis 80 (85) 32 (34) (38) Impairment hearing (37) 23 (25) 18 (26) 11 Trigger (cold, stress, infections, vaccinations, hormones) 71 (76) 3 (8) SAE events Incidence rate * per 100 patient All types SADR 28# 25.98 11.55 CRP, c-reactive protein; ESR, erythrocyte sedimentation rate; n. a., not annotated; SAA, serum amyloid A; SADR, serious adverse drug reaction; SAE, event *Incidence = number 36,525 / sum observation (=88,558) #Abdominal pain, Alport’s syndrome, appendicitis, arthralgia, blister, cardiovascular disorder, chest circulatory collapse, dehydration, diplopia, dyspnoea, erythema, febrile convulsion, gastroenteritis, glomerulonephritis, haemophilus test positive, myalgia, oedema, pneumonia, premature delivery, skin discoloration, tonsillectomy, tonsillitis bacterial, streptococcal, vision blurred (all N=1 event), pyrexia (3 events) Conclusion 36-month interim analysis demonstrates that safe effective independent subtype severity. Disclosure Interests J. B. Kuemmerle-Deschner Consultant of: Novartis, AbbVie, Sobi, Grant/research support from: Birgit Kortus-Goetze Paid instructor for: Prasad Oommen Ales Janda: None declared, Jürgen Rech Speakers bureau: Abbvie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD; Mylan, Roche, Sanofi, UCB, MSD, Catharina Schuetz: Tilmann Kallinich Frank Weller-Heinemann: Gerd Horneff Bayer, Merck Sharp & Dohme, Pfizer, Ivan Foeldvari Hexal, Medac, Florian Meier Michael Borte Shire, Tobias Krickau Julia Weber-Arden Employee Norbert Blank Actelion, Boehringer-Ingelheim, Sobi