fetching data ... 
Background: In the treatment of monogenic autoinflammatory diseases (AID), a heterogeneous group of diseases with excessive interleukin (IL)-1β release and severe systemic and organ inflammation, the anti-IL-1 inhibitor canakinumab (CAN) has been associated with rapid remission of symptoms in clinical trials as well as in real-life. 1-3
Objectives: The aim of the RELIANCE registry is to explore long-term effectiveness and safety of CAN under routine clinical practice conditions in pediatric and adult patients with CAPS (cryopyrin-associated periodic syndromes, including Muckle-Wells syndrome [MWS], familial cold autoinflammatory syndrome [FCAS], neonatal onset multisystem inflammatory disease [NOMID]/chronic infantile neurological cutaneous and articular syndrome [CINCA]), FMF (familial Mediterranean fever), TRAPS (tumor necrosis factor receptor-associated periodic syndrome) and HIDS/MKD (hyperimmunoglobulinemia D syndrome/mevalonate kinase deficiency).
Methods: This prospective, non-interventional, observational study is based in Germany with a 3-year follow-up and enrolls pediatric ≥2 years and adult patients with clinically confirmed diagnoses of CAPS, FMF, TRAPS and HIDS/MKD routinely receiving CAN. In 6-monthly visits, clinical data and patient-reported outcomes are assessed. Study endpoints are long-term effectiveness and safety of CAN. Here, the CAPS cohort was analyzed.
Results: This 18-month interim-analysis includes 78 CAPS patients (49% females) enrolled by September 2019. Mean age at baseline was 25 years and mean duration of prior CAN treatment was 5.7 years. 64 patients (82%) had MWS, 2 FCAS, 7 NOMID/CINCA, 3 atypical CAPS and 2 lacked subtype diagnosis. Disease activity, fatigue and social impairment by patients’ assessment, days absent from school/work, inflammatory markers, and remission by physician assessment were evaluated at 6-monthly intervals starting at baseline with last update at 18 months of follow-up (
Patient and physician assessment of clinical CAPS disease activity and laboratory markers over time
| Baseline | 6 months | 12 months | 18 months | |
| Number of patients, N | 78 | 51 | 42 | 29 |
| Mean age, years (SD) | 25 (4; 79) | 22 (4; 79) | 20 (4; 58) | 22 (4; 54) |
| Patient’s assessment of disease activity 0-10, mean (min; max) | 2.2 (0; 7) | 1.8 (0; 7) | 2.4 (0; 7) | 2.8 (0; 8) |
| Patient’s assessment of fatigue 0-10 | 2.9 (0; 9) | 2.4 (0; 8) | 2.8 (0; 8) | 1.7 (0; 7) |
| Number (%) of patients without impairment of social life by disease | 16 (49) | 29 (76) | 20 (61) | 14 (67) |
| Number (%) of patients with days absent from school/work | 25 (32.5) | 11 (22) | 14 (34) | 15 (52) |
| Inflammatory markers, CRP/SAA, mean (mg/dL) | 0.4
| 0.4
| 0.3
| 0.2
|
| Number (%) of patients in disease remission (physician assessment) | 55 (72) | 38 (76) | 29 (71) | 22 (76) |
Conclusion: The 18-month interim analysis of the RELIANCE study, the longest running real-life CAN registry, demonstrates that long-term CAN treatment is safe and effective in CAPS patients.
REFERENCES:
[1]Lachmann et al. N Engl J Med. 2009;360(23):2416-25
[2]Kuemmerle-Deschner et al. Rheumatology (Oxford). 2016;55(4):689-96
[3]De Benedetti et al. N Engl. J Med. 2018;378(20):1908-1919
Disclosure of Interests: Norbert Blank Grant/research support from: Novartis, Sobi, Consultant of: Novartis, Sobi, Lilly, Pfizer, Abbvie, BMS, MSD, Actelion, UCB, Boehringer-Ingelheim, Roche, Michael Borte Grant/research support from: Pfizer, Shire, Ivan Foeldvari Consultant of: Novartis, Gerd Horneff Grant/research support from: AbbVie, Chugai, Merck Sharp & Dohme, Novartis, Pfizer, Roche, Speakers bureau: AbbVie, Bayer, Chugai, Merck Sharp & Dohme, Novartis, Pfizer, Roche, Tilmann Kallinich Grant/research support from: Novartis, Consultant of: Sobi, Roche, Novartis, Birgit Kortus-Goetze Consultant of: Novartis, Prasad Oommen Consultant of: Novartis, Catharina Schuetz: None declared, Frank Weller-Heinemann: None declared, Julia Weber-Arden Employee of: I am employed by Novartis, J. B. Kuemmerle-Deschner Grant/research support from: Novartis, AbbVie, Sobi, Consultant of: Novartis, AbbVie, Sobi