Effect of Canakinumab Treatment on Health-Related Quality of Life in Patients with Periodic Fever Syndromes

Anna Simon¹, Anna Shcherbina², Jordi Anton³, Eldad Ben-Chetrit⁴, Fabrizio De Benedetti⁵, Joost Frenkel⁶, Marco Gattorno⁷, Ryoki Hara⁸, Philip J Hashkes⁹, Michaël Hofer¹⁰, Hal M. Hoffman¹¹, Isabelle Koné-Paut¹², Helen Lachmann¹³, Alberto Martini¹⁴, Seza Ozen¹⁵, Andrew Zeft¹⁶, Antonio Speziale¹⁷, Guido Junge¹⁷ and Jill Gregson¹⁷, ¹General Internal Medicine, Radboud University Nijmegen Medical Centre, Nijmegen, Netherlands, ²Clinical immunology Department, Center of Children's Hematology n.a. D. Rogachev, Moscow, Russian Federation, ³Hospital Sant Joan de Déu, Barcelona, Spain, ⁴Rheumatology Unit, Hadassah—Hebrew University Medical Center, Jerusalem, Israel, ⁵IRCCS Ospedale Pediatrico Bambino Gesú, Rome, Italy, ⁶University Medical Center Utrecht, Utrecht, Netherlands, ⁷Pediatric Rheumatology, G. Gaslini Institute, Genoa, Italy, ⁸Yokohama City University, Yokohama, Japan, ⁹Pediatrics Rheumatology; Shaare-Zedek Medical Center, Jerusalem, Israel, ¹⁰Pediatric Rheumatology of Western Switzerland, CHUV, University of Lausanne, Lausanne, Switzerland, ¹¹University of California at San Diego, San Diego, CA, ¹²Hopital Kremlin Bicetre, University of Paris SUD, Paris, France, ¹³UK National Amyloidosis Centre, University College London Medical School, London, United Kingdom, ¹⁴Istituto G. Gaslini Pediatria II Reumatologia, Genova, Italy, ¹⁵Department of Pediatrics, Hacettepe University Faculty of Medicine, Ankara, Turkey, ¹⁶Pediatrics Rheumatology, Cleveland Clinic, Cleveland, OH, ¹⁷Novartis Pharma AG, Basel, Switzerland

Meeting: 2016 ACR/ARHP Annual Meeting

Date of first publication: September 28, 2016

Keywords: Traps and canakinumab

Session Information

Date: Tuesday, November 15, 2016

Title: Pediatric Rheumatology – Clinical and Therapeutic Aspects - Poster III: Systemic JIA, Autoinflammatory Syndromes, Scleroderma, Vasculitis, Miscellaneous

Session Type: ACR Poster Session C

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

Background/Purpose: Open label studies have shown the efficacy of canakinumab (CAN), a fully human and highly specific anti-IL-1β monoclonal antibody, in patients (pts) with Periodic Fever Syndromes (PFS), which include colchicine-resistant Familial Mediterranean Fever (crFMF), Hyper-IgD Syndrome/Mevalonate Kinase Deficiency (HIDS/MKD) and TNF-Receptor Associated Periodic Syndrome (TRAPS). Currently, no data is available on the effect of CAN on Health-Related Quality of Life (HRQoL) in pts with PFS. The study objective was to assess the effect of CAN on HRQoL using Child Health Questionnaire-Parent Form 50 (CHQ-PF50) and Short Form-12 Health Survey (SF-12) in pts with PFS.

Methods: In this Phase 3, randomized, placebo-controlled study of CAN in pts with PFS (NCT02059291), SF-12 Physical Component Summary (PCS) and Mental Component Summary (MCS) scores were assessed in adults, whereas CHQ-PF50 Physical Summary (PhS) and Psychosocial Summary (PsS) scores were assessed in children (>5–<18 years).

Results: Of the 181 pts (63 crFMF, 72 MKD/HIDS, 46 TRAPS) randomized to CAN or placebo, 71 were adults (age ≥18 years) and 110 were children (age ≥2–<18 years). Pts who initially received placebo and did not respond were switched to CAN. Treatment with CAN was associated with an early clinically meaningful improvement in SF-12 PCS scores reported at Week (Wk) 5, which were sustained and increased to a large effect size by Wk 16 for all indications (Table). Similarly, clinically meaningful improvement in SF-12 MCS, CHQ-PF50 PhS, and CHQ-PF50 PsS scores was observed for all indications, except for PsS in HIDS/MKD and TRAPS pts.

Conclusion: Canakinumab showed rapid improvement by Wk 5 in pt-reported outcomes in adults and children with PFS, which was sustained through Wk 16. References: 1. User’s manual for the SF-12v2 Health Survey, 3^rd ed.; 2012 2. Cohen J, et al. Statistical power analysis for the behavioural sciences, 2^nd ed.; 1988 3. HealthActCHQ. The CHQ Scoring and Interpretation Manual (Boston, MA: HealthActCHQ, 2013) 4. Maruish, M.E. ed. User’s manual for the SF-12v2 Health Survey, 3rd ed. (Lincoln, RI: QualityMetric Inc., 2012)

Disclosure: A. Simon, Novartis, Xoma/Servier, CSL Behring, 2,Novartis, Takeda, SOBI, Xoma, 5; A. Shcherbina, None; J. Anton, Novartis Pharma AG, 2,Novartis Pharma AG, 5; E. Ben-Chetrit, Novartis Pharmaceutical Corporation, 5; F. De Benedetti, Pfizer, Abbvie, Roche, Novartis, Novimmune and BMS and SOBI, 2; J. Frenkel, Novartis and SOBI, 2; M. Gattorno, Novartis and SOBI, 2,Novartis and SOBI, 5; R. Hara, None; P. J. Hashkes, Novartis Pharmaceutical Corporation, 2,Novartis Pharmaceutical Corporation, 5,Novartis Pharmaceutical Corporation, 8; M. Hofer, Novartis Pharmaceutical Corporation, 5; H. M. Hoffman, Novartis Pharmaceutical Corporation, 5,Novartis Pharmaceutical Corporation, 8; I. Koné-Paut, Novartis, SOBI and Roche, 2,Novartis, SOBI, Pfizer, Abbvie, Roche, 5; H. Lachmann, Novartis, SOBI, Takeda and GSK, 5,Novartis and SOBI, 8; A. Martini, Abbott, BMS, "Francesco Angelini", GlaxoSmithKline (GSK), Hoffman-La Roche, Italfarmaco, Janssen, Novartis, Pfizer, Sanofi Aventis, Schwarz Biosciences, Sobi, Xoma and Wyeth, 2,Abbott, Abbvie, Amgen, Biogenidecm Bristol MyersSquibb, Astellas, Behringer, Italfarmaco, Janssen, MedImmune, Novartis, NovoNordisk, Pfizer, Sanofi, Roche, Servier and Takeda, 8; S. Ozen, Novartis and SOBI, 8; A. Zeft, Merck, Opko Health, Arno therapeutics, 1,Novartis Pharmaceutical Corporation, 5; A. Speziale, Novartis Pharma AG, 3; G. Junge, Novartis Pharma AG, 3; J. Gregson, Novartis., 3.

To cite this abstract in AMA style:

Simon A, Shcherbina A, Anton J, Ben-Chetrit E, De Benedetti F, Frenkel J, Gattorno M, Hara R, Hashkes PJ, Hofer M, Hoffman HM, Koné-Paut I, Lachmann H, Martini A, Ozen S, Zeft A, Speziale A, Junge G, Gregson J. Effect of Canakinumab Treatment on Health-Related Quality of Life in Patients with Periodic Fever Syndromes [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). https://acrabstracts.org/abstract/effect-of-canakinumab-treatment-on-health-related-quality-of-life-in-patients-with-periodic-fever-syndromes/. Accessed .

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