Long-term safety and effectiveness of canakinumab in patients with monogenic autoinflammatory diseases: results from the interim analysis of the RELIANCE registry

• Published in: Rheumatic & musculoskeletal diseases open Vol. 10; no. 1; p. e003890
• Main Authors: Kuemmerle-Deschner, Jasmin B, Kallinich, Tilmann, Henes, Joerg, Kortus-Götze, Birgit, Oommen, Prasad T, Rech, Juergen, Krickau, Tobias, Weller-Heinemann, Frank, Horneff, Gerd, Janda, Aleš, Foeldvari, Ivan, Schuetz, Catharina, Dressler, Frank, Borte, Michael, Hufnagel, Markus, Meier, Florian, Fiene, Michael, Andreica, Ioana, Weber-Arden, Julia, Blank, Norbert
• Format: Journal Article
• Language: English
• Published: England EULAR 15-02-2024; BMJ Publishing Group LTD; BMJ Publishing Group
• Series: Original research
• Subjects: Adults; Autoinflammatory Disorders; Clinical medicine; Clinical trials; Cryopyrin-Associated Periodic Syndromes; Cytokines; Disease control; Drug dosages; Familial Mediterranean Fever; Fever; Inflammation; Kinases; Monoclonal antibodies; Mutation; Pathogenesis; Patients; Pediatrics; Quality of life; Rheumatology; Standard of care; Steroids; Germany; Cryopyrin-Associated Periodic Syndromes; Inflammation; Familial Mediterranean Fever
• ISSN: 2056-5933; 2056-5933
• DOI: 10.1136/rmdopen-2023-003890

ObjectiveInterim analysis of the RELIANCE registry, an on-going, non-interventional, open-label, multicentre, prospective study evaluating the long-term safety, dosing regimens and effectiveness of canakinumab in patients with cryopyrin-associated periodic syndromes (CAPS), familial Mediterranean fever (FMF), tumour-necrosis factor receptor-associated periodic syndrome (TRAPS) or mevalonate-kinase deficiency (MKD)/hyperimmunoglobulin-D syndrome (HIDS).MethodsFrom September 2017 for patients with CAPS, and June 2018 for patients with FMF, TRAPS or MKD/HIDS, the registry enrolled paediatric (aged ≥2 years) and adult patients (aged ≥18 years) receiving canakinumab as part of their routine medical care. Safety, canakinumab dose, disease activity and quality of life outcome measures were evaluated at baseline and every 6 months until end of study visit.ResultsAt the analysis cut-off date (December 2020), 168 patients (91 CAPS, 54 FMF, 16 TRAPS and 7 MKD/HIDS) were enrolled. 85 (50.9%) patients were female and 72 (43.1%) were children (<18 years). The median patient age was 20.0 years (range 2.0–79.0 years). In the CAPS cohort, serious infections and serious adverse drug-reactions were more common in patients receiving higher than the recommended starting dose (SD) of canakinumab. A trend to receive >SD of canakinumab was observed in the pooled population. The majority of patients were reported as having either absent or mild/moderate disease activity (physician’s global assessment) from baseline to Month 30, with a stable proportion of patients (~70%) in remission under canakinumab treatment. Patient-reported disease activity (Visual Analogue Scale (VAS), Autoinflammatory Disease Activity Index), fatigue (VAS); markers of inflammation (C-reactive protein, serum amyloid A and erythrocyte sedimentation rate) remained well-controlled throughout.ConclusionData from this analysis confirm the long-term safety and effectiveness of canakinumab for the treatment of CAPS, FMF, TRAPS and MKD/HIDS.