Disease Control in Patients with Monogenetic Autoinflammatory Diseases Under Canakinumab Treatment – Comparison of 30 Months Interim Data from the RELIANCE Registry

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

Meeting: ACR Convergence 2023

Keywords: Autoinflammatory diseases, Disease Activity, Innate Immunity Rheumatic Disease, Periodic Disease

Session Information

Date: Sunday, November 12, 2023

Title: (0252–0282) Miscellaneous Rheumatic & Inflammatory Diseases Poster I

Session Type: Poster Session A

Session Time: 9:00AM-11:00AM

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)

Disclosures: 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.

To cite this abstract in AMA style:

Foeldvari I, Kallinich T, Blank N, Henes J, Kortus-Goetze B, Oommen P, Pankow A, Krickau T, Schuetz C, Horneff G, Rech J, Weller-Heinemann F, Janda A, Hufnagel M, Meier F, Dressler F, Borte M, Andreica I, Wasiliew P, Fiene M, Windschall D, Krusche M, Kuempfel T, Weber-Arden J, Kuemmerle-Deschner J. Disease Control in Patients with Monogenetic Autoinflammatory Diseases Under Canakinumab Treatment – Comparison of 30 Months Interim Data from the RELIANCE Registry [abstract]. Arthritis Rheumatol. 2023; 75 (suppl 9). https://acrabstracts.org/abstract/disease-control-in-patients-with-monogenetic-autoinflammatory-diseases-under-canakinumab-treatment-comparison-of-30-months-interim-data-from-the-reliance-registry/. Accessed .

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