ACR Meeting Abstracts

ACR Meeting Abstracts

Abstract Number: 0191

Influence of Canakinumab Dosing on Long-term Efficacy and Safety in Patients with Cryopyrin-associated Periodic Syndromes (CAPS) – 30-months Interim Analysis of the RELIANCE Registry

Jasmin Kuemmerle-Deschner1, Birgit Kortus-Goetze2, Prasad T. Oommen3, Aleš Janda4, Juergen Rech5, Tilmann Kallinich6, Frank Weller-Heinemann7, Gerd Horneff8, Ivan Foeldvari9, Catharina Schuetz10, Michael Borte11, Axel Braner12, Julia Weber-Arden13 and Norbert Blank14, 1Pediatric Rheumatology and Autoinflammatory Reference Center, University Children's Hospital Tuebingen, Tuebingen, Germany, 2Division of Nephrology, University of Marburg, Marburg, Germany, 3Clinic of Pediatric Hematology, Oncology and Clinical Immunology, Heinrich-Heine-University Duesseldorf, Duesseldorf, Germany, 4Department of Pediatrics, University Hospital Ulm, Ulm, Germany, 5Friedrich-Alexander University (FAU) Erlangen-Nuernberg and Universitaetsklinikum Erlangen, Department of Internal Medicine 3 - Rheumatology and Immunology, Germany, Erlangen, Germany, 6Charité University Medicine, Nuremberg, Germany, 7Klinikum Bremen-Mitte, Prof. Hess Kinderklinik, Bremen, Germany, 8Paediatric Rheumatology International Trials Organisation (PRINTO), Sankt Augustin, Germany, 9Hamburger Zentrum fuer Kinder- und Jugendrheumatologie, Hamburg, Germany, 10Pediatrics, Medizinische Fakultaet Carl Gustav Carus, Technische Universitaet Dresden, Dresden, Germany, 11ImmunoDeficiencyCenter Leipzig (IDCL), Hospital St. Georg gGmbH Leipzig, Leipzig, Germany, 12Department of Medicine II, Rheumatology, University Hospital Frankfurt, Germany, Frankfurt a.M., Germany, 13Novartis Pharma GmbH, Nuremberg, Germany, 14Rheumatology, University Hospital Heidelberg, Eppelheim, Germany

Meeting: ACR Convergence 2021

Keywords: , , , ,

Session Information

Date: Saturday, November 6, 2021

Title: Miscellaneous Rheumatic & Inflammatory Diseases Poster I (0183–0209)

Session Type: Poster Session A

Session Time: 8:30AM-10:30AM

Background/Purpose: The IL-1 inhibitor canakinumab (CAN) induces rapid remission of symptoms of CAPS (Cryopyrin-associated periodic syndromes), a monogenic autoinflammatory disease with severe systemic inflammation, both in clinical trials and in routine practice.The aim of the present study is to investigate the long-term efficacy and safety of CAN in pediatric (≥ 2 years) and adult patients with CAPS (incl. Muckle-Wells syndrome [MWS], familial cold-induced autoinflammatory syndrome [FCAS], and neonatal onset inflammatory systemic disease [NOMID]/chronic infantile neurologic-cutaneous-articular syndrome [CINCA]) in relation to weight-dependent CAN dosing under routine clinical practice conditions.

Methods: The prospective, non-interventional, observational RELIANCE study with 3-year follow-up includes patients with clinically confirmed CAPS diagnosis who routinely receive CAN. Clinical data, physician assessment, and patient-reported outcomes are evaluated at 6-month visits from baseline.

Results: The interim analysis included data from 91 CAPS patients (50% females; 14 [15%] NOMID/CINCA subtypes) enrolled by December 2020. At baseline, median age was 20.5 years and median duration of prior CAN treatment was 6 years. According to physician assessment, 66% of patients achieved disease remission with increasing absence of disease activity according to PGA (physician global assessment, 50-60%). Patients reported stable, low levels of disease activity, fatigue, and Auto-Inflammatory Diseases Activity Index (AIDAI) scores. CAPS affected the social life of 50% of patients and 50% reported days of absence from school/work. At baseline, 2% of patients received less than the standard dose* CAN (SD; < 87.5% of SD), 49% received standard dose CAN (SD CAN; 150 mg and 2mg/kg respectively per 8 weeks) and another 49% received higher than SD CAN ( >112.5% of SD). At 30 months, 82% of patients received higher than SD CAN. The laboratory parameters as well as physicians´ and patients´ rating of disease activity remained stable over time, showing sustained remission and disease control in all dosing groups (Table 1).

Treatment-related severe adverse events (SAE) occurred in 8 patients, none of them under standard dosing (in 1 patient: less than SD, in 7 patients more than SD).

Conclusion: The 30-month interim analysis of the RELIANCE study shows that long-term treatment with CAN is safe and effective in patients with CAPS. A clear trend towards updosing to more than SD CAN could be observed.

Table 1: Stratification of efficacy parameters for CAN dosing and weight (N=91)#.
*Body weight >40kg: Standard dose is 150 mg per 8 weeks; Body weight ≤40 kg: Standard dose is 2mg per kg per 8 weeks; #Numbers and percentages do not sum up to N=91 or 100%, respectively, due to the number of patients with unknown weight.

Disclosures: J. Kuemmerle-Deschner, Novartis, 1, 2, 5, 6, SOBI, 1, 2, 5, 6; B. Kortus-Goetze, Novartis, 2; P. Oommen, Novartis, 5; A. Janda, None; J. Rech, Novartis, 2, 5, 6, SOBI, 2, 5, 6, AbbVie, 2, 6, Biogen, 2, 6, BMS, 2, 6, Chugai, 2, 6, GSK, 2, 6, Janssen, 2, 6, Lilly, 2, 6, MSD, 2, 6, Mylan, 2, 6, Roche, 2, 6, Sanofi, 2, 6, UCB, 2, 6; T. Kallinich, None; F. Weller-Heinemann, None; G. Horneff, Novartis, 5, 6, Janssen, 5, 6, Roche, 5, Eli-Lilly, 6, Glaxo Smith and Kline, 6, Pfizer, 6, Sobi, 6; I. Foeldvari, Novartis, 4; C. Schuetz, None; M. Borte, Shire, 5, Pfizer, 5; A. Braner, Novartis, 2, SOBI, 2; J. Weber-Arden, Novartis, 3; N. Blank, Novartis, 2, 5, SOBI, 2, 5, Lilly, 2, Pfizer, 2, AbbVie, 2, BMS, 2, MSD, 2, Actelion, 2, UCB, 2, Boehringer Ingelheim, 2, Roche, 2.

To cite this abstract in AMA style:

Kuemmerle-Deschner J, Kortus-Goetze B, Oommen P, Janda A, Rech J, Kallinich T, Weller-Heinemann F, Horneff G, Foeldvari I, Schuetz C, Borte M, Braner A, Weber-Arden J, Blank N. Influence of Canakinumab Dosing on Long-term Efficacy and Safety in Patients with Cryopyrin-associated Periodic Syndromes (CAPS) – 30-months Interim Analysis of the RELIANCE Registry [abstract]. Arthritis Rheumatol. 2021; 73 (suppl 9). https://acrabstracts.org/abstract/influence-of-canakinumab-dosing-on-long-term-efficacy-and-safety-in-patients-with-cryopyrin-associated-periodic-syndromes-caps-30-months-interim-analysis-of-the-reliance-registry/. Accessed .

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