Session: (0252–0282) Miscellaneous Rheumatic & Inflammatory Diseases Poster I
0276: Disease Control in Patients with Monogenetic Autoinflammatory Diseases Under Canakinumab Treatment – Comparison of 30 Months Interim Data from the RELIANCE Registry
Hamburger Zentrum für Kinder- und Jugendrheumatologie Hamburg, Germany
Disclosure(s): Novartis: Consultant (Ongoing)
Ivan Foeldvari1, Tilmann Kallinich2, Norbert Blank3, Joerg Henes4, Birgit Kortus-Goetze5, Prasad T. Oommen6, Anne Pankow7, Tobias Krickau8, Catharina Schuetz9, Gerd Horneff10, Juergen Rech11, Frank Weller-Heinemann12, Ales Janda13, Markus Hufnagel14, Florian M. Meier15, Frank Dressler16, Michael Borte17, Ioana Andreica18, Peter Wasiliew19, Michael Fiene20, Daniel Windschall21, Martin Krusche22, Tania Kuempfel23, Julia Weber-Arden24 and Jasmin B. Kuemmerle-Deschner25, 1Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg, Germany, 2Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité Universitätsmedizin Berlin, Nuremberg, Germany, 3University Hospital Heidelberg, Heidelberg, Germany, 4University Hospital Tuebingen, Tuebingen, Germany, 5Department of Internal Medicine, Division of Nephrology,University Hospital of Giessen and Marburg, Marburg, Germany, 6Department of Pediatric Oncology, Hematology and Clinical Immunology, Center for Child and Adolescent Health,Medical Faculty Heinrich-Heine-University Duesseldorf, Duesseldorf, Germany, 7Department of Rheumatology and Clinical Immunology,Charité-Universitätsmedizin Berlin, Berlin, Germany, 8Pediatrics, Friedrich-Alexander University Erlangen-Nuernberg (FAU), Erlangen, Germany, 9Department of Pediatrics, Medizinische Fakultät Carl Gustav Carus,Technische Universität Dresden, Dresden, Germany, 10Asklepios Klinik Sankt Augustin GmbH, Bonn, Germany, 11University Clinic Erlangen, Erlangen, Germany, 12Division of Pediatric Rheumatology, Prof. Hess Children's Hospital, Bremen, Bremen, Germany, 13Department of Pediatrics and Adolescent Medicine, University Medical Center Ulm, Ulm, Germany, 14Division of Pediatric Infectious Diseases and Rheumatology, Department of Pediatrics and Adolescent Medicine, University Medical Center, Medical Faculty, University of Freiburg, Freiburg, Germany, 15Department of General Pharmacology and Toxicology, Goethe University Hospital and Goethe University Frankfurt, Frankfurt am Main, Germany, 16Department of Paediatric Pneumology, Allergology and Neonatology, Children's Hospital, Hannover Medical School, Hannover, Germany, 17Hospital for Children & Adolescents, St. Georg Hospital, Leipzig, Germany, 18Rheumazentrum Ruhrgebiet Herne, Herne, Germany, 19Division of Pediatric Rheumatology and autoinflammation reference center Tuebingen, Department of Pediatrics, University Hospital Tuebingen, Tuebingen, Germany, 20Rheumatology Center Greifswald, Greifswald, Germany, 21Clinic of Paediatric and Adolescent Rheumatology, St. Josef-Stift Sendenhorst, Northwest German Center for Rheumatology, Sendenhorst, Germany, 22UKE, Hamburg, Germany, 23Institute of Clinical Neuroimmunology, Biomedical Center and University Hospital, Ludwig-Maximilians Universität München, Muenchen, Germany, 24Novartis Innovative Medicines, Nuernberg, Germany, 25med.uni-tuebingen, Tübingen, Germany
Background/Purpose: Treatment of autoinflammatory periodic diseases (AID) with the interleukin-1β inhibitor canakinumab (CAN) has been shown to be safe and effective in controlled trials and real-world setting. In the RELIANCE registry, long-term safety and efficacy of CAN in patients with cryopyrin-associated periodic syndromes (CAPS), familial Mediterranean fever (FMF), hyper-IgD syndrome/mevalonate kinase deficiency (HIDS/MKD) and tumor necrosis factor receptor-associated periodic syndrome (TRAPS) on CAN therapy were investigated in routine clinical practice.
Methods: The RELIANCE registry is a prospective, non-interventional, observational study in Germany enrolling pediatric (age ≥2 years) and adult patients with a clinically confirmed diagnosis of AID who routinely receive CAN. Efficacy and safety parameters are recorded at baseline and assessed at 6-month intervals. To compare disease control between indications, parameters of 30 months visits were analyzed.
Results: In the present interim analysis, data were included from a total of n=232 patients with a diagnosis of CAPS, FMF, TRAPS and HIDS/MKD enrolled in the RELIANCE registry between October 2017 and December 2022. The median age of the total study cohort was 20.0 years (2−80 years). Most patients (n=198, 85 %) were CAN pre-treated when entering the study and the median duration of total CAN treatment before and during the study was 4 years (0−15 years).
Of 58/28/10/5 CAPS/FMF/TRAPS/HIDS patients with month 30 visits documented, 68/82/63/100% were in disease remission according to physician assessment. Patient-reported median disease activity and fatigue were low (1.5/1.5/1.5/0 and 3/2/4/0 on a 0−10 VAS scale). Inflammation markers (median) were within the limits of normal including neutrophil counts (2975/3420/3262/2897 n/µL). Statistical analysis confirmed similar efficacy across diseases in most parameters.
Even though these outcomes suggest an adequate disease control, an impact of the disease on patient´s social life was reported in 38/22/50/50% and negative influence on mood in 26/6/0/50% of patients.
Conclusion: The interim data of the RELIANCE study confirm sustained disease control of long-term treatment with CAN across all study indications. Even though disease activity measures suggest adequate disease control, patients´ social life and mood were negatively impacted by the disease.
Figure 1: Clinical disease activity in the RELIANCE study across study indications (n=232 patients)
Table 1: Overview of laboratory markers and dose categories in the RELIANCE study across study indications (n=232 patients)
I. Foeldvari: Novartis, 2; T. Kallinich: Roche, 6; N. Blank: Novartis, Sobi, 5, Novartis, Sobi, Lilly, Pfizer, Abbvie, BMS, MSD, Actelion, UCB, Boehringer-Ingelheim, Roche, 2; J. Henes: AbbVie/Abbott, 2, 6, Boehringer-Ingelheim, 2, 6, Bristol-Myers Squibb(BMS), 2, 6, GlaxoSmithKlein(GSK), 2, 6, Janssen, 2, 6, Novartis, 2, 6, Pfizer, 2, 6, UCB, 2, 6; B. Kortus-Goetze: Novartis, 2; P. Oommen: Novartis, 5; A. Pankow: None; T. Krickau: Novartis, 2, 5, 6; C. Schuetz: Novartis, 5; G. Horneff: GSK, 6, Janssen, 6, MSD, 5, 6, Novartis, 5, 6, Pfizer, 5, 6, Roche, 5, 6, Sanofi, 6, Sobi, 6; J. Rech: AbbVie, Biogen, BMS, Chugai, GSK, Janssen, Lilly, MSD; Mylan, Novartis, Roche, Sanofi, Sobi, UCB, 2, 6, Novartis, Sobi, 5; F. Weller-Heinemann: None; A. Janda: None; M. Hufnagel: Novartis, 5; F. Meier: Novartis, 6; F. Dressler: Abbvie, Mylan, Novartis, Pfizer, 2, Novartis, 5; M. Borte: Pfizer, Shire, 5; I. Andreica: AbbVie/Abbott, 1, 6, Amgen, 1, 6, AstraZeneca, 1, 6, Chugai, 6, Novartis, 1, 6, Sobi, 1, 6, UCB, 1, 6; P. Wasiliew: None; M. Fiene: None; D. Windschall: None; M. Krusche: Chugai, 2, 6; T. Kuempfel: None; J. Weber-Arden: Novartis, 3; J. Kuemmerle-Deschner: Novartis, AbbVie, Sobi, 2, 5.