Long-term safety and effectiveness of canakinumab in patients with monogenic autoinflammatory diseases: results from the interim analysis of the RELIANCE registry
Tilmann Kallinich,
Juergen Rech,
Ivan Foeldvari,
Norbert Blank,
Frank Weller-Heinemann,
Gerd Horneff,
Ioana Andreica,
Joerg Henes,
Jasmin B Kuemmerle-Deschner,
Florian Meier,
Frank Dressler,
Michael Borte,
Markus Hufnagel,
Birgit Kortus-Götze,
Prasad T Oommen,
Tobias Krickau,
Aleš Janda,
Catharina Schuetz,
Michael Fiene,
Julia Weber-Arden
Affiliations
Tilmann Kallinich
1BIKER-Registry, Sankt Augustin, Germany
Juergen Rech
Department of Internal Medicine 3 - Rheumatology and Immunology, Friedrich-Alexander University (FAU) Erlangen-Nürnberg and Universitätsklinikum Erlangen, Erlangen, Germany
Ivan Foeldvari
Centre for Pediatric and Adolescent Rheumatology, Hamburg, Germany
Center of Interdisciplinary Rheumatology, Immunology and autoimmune diseases (INDIRA), University Hospital Tübingen, Tübingen, Germany
Jasmin B Kuemmerle-Deschner
Division of Paediatric Rheumatology and autoinflammation reference centre Tübingen, Department of Paediatrics, University Hospital Tübingen, Tübingen, Germany
Florian Meier
Department of Medicine II, Rheumatology, University Hospital Frankfurt, Goethe University, Frankfurt, Germany
Frank Dressler
1BIKER-Registry, Sankt Augustin, Germany
Michael Borte
BIKER-Registry, Sankt Augustin, Germany
Markus Hufnagel
1BIKER-Registry, Sankt Augustin, Germany
Birgit Kortus-Götze
Department of Internal Medicine, Division of Nephrology, University Hospital of Giessen and Marburg, Marburg, Germany
Prasad T Oommen
Department of Paediatric Oncology, Haematology and Clinical Immunology, Division of Paediatric Rheumatology, Medical Faculty, Heinrich Heine University Düsseldorf, Düsseldorf, Germany
Tobias Krickau
DZI (Deutsches Zentrum für Immuntherapie), Erlangen, Germany
Aleš Janda
Department of Paediatrics and Adolescent Medicine, University Medical Centre Ulm, Ulm, Germany
Objective Interim 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).Methods From 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.Results At 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.Conclusion Data from this analysis confirm the long-term safety and effectiveness of canakinumab for the treatment of CAPS, FMF, TRAPS and MKD/HIDS.
