SAT0510 LONG-TERM EFFECTIVENESS OF CANAKINUMAB IN AID – INTERIM ANALYSIS OF THE CAPS SUBGROUP FROM THE RELIANCE REGISTRY

Autor: Frank Weller-Heinemann, Norbert Blank, B. Kortus-Goetze, Catharina Schuetz, G. Horneff, I. Foeldvari, Michael Borte, Tilmann Kallinich, Prasad T. Oommen, J. Weber-Arden, J. B. Kuemmerle-Deschner
Rok vydání: 2020
Předmět:
Zdroj: Annals of the Rheumatic Diseases. 79:1211.2-1211
ISSN: 1468-2060
0003-4967
Popis: 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.1-3Objectives: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 (table 1). The results demonstrate sustained remission and disease control as evaluated parameters remained stable over time. Serious adverse events were reported for 9 patients including papillitis, pyrexia, tonsillitis (n=2), appendicitis, chest pain, circulatory collapse, skin disorders, and preterm delivery.Table 1.Patient and physician assessment of clinical CAPS disease activity and laboratory markers over timeBaseline6 months12 months18 monthsNumber of patients, N78514229Mean 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 0-102.9 (0; 9)2.4 (0; 8)2.8 (0; 8)1.7 (0; 7)Number (%) of patients without impairment of social life by disease16 (49)29 (76)20 (61)14 (67)Number (%) of patients with days absent from school/work25 (32.5)11 (22)14 (34)15 (52)Inflammatory markers, CRP/SAA, mean (mg/dL)0.43.20.42.10.30.80.20.5Number (%) 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):1908-1919Disclosure 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
Databáze: OpenAIRE
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