Efficacy and Safety of Canakinumab in Patients with Systemic Juvenile Idiopathic Arthritis: Results from an Open-Label Long-Term Follow-up Study

Hermine I. Brunner¹, Nicolino Ruperto², Pierre Quartier³, Tamás Constantin², Ekaterina Alexeeva⁴, Rayfel Schneider¹, Isabelle Kone-Paut², Kenneth Schikler¹, Katherine Marzan¹, Nico Wulffraat⁴, Shai Padeh⁴, Vyacheslav Chasnyk², Carine Wouters⁴, Jasmin B. Kuemmerle-Deschner⁴, Tilmann Kallinich⁴, Bernard Lauwerys⁵, Elie Haddad¹, Evgeny L Nasonov⁴, Maria Trachana⁴, Olga Vougiouka⁴, Karolynn Leon⁶, Antonio Speziale⁷, Karine Lheritier⁷, Eleni Vritzali⁸, Daniel J Lovell¹, Alberto Martini² and PRINTO/PRCSG, ¹PRCSG, Cincinnati, OH, ²PRINTO-Istituto Gaslini, Genoa, Italy, ³Hôpital Necker-Enfants Malades, Paris, France, ⁴PRINTO-Istituto Gaslini, Genova, Italy, ⁵Cliniques Universitaires Saint-Luc and Université Catholique de Louvain, Brussels, Belgium, ⁶Novartis Pharmaceuticals Corporation, East Hanover, NJ, ⁷Novartis Pharma AG, Basel, Switzerland, ⁸Immunology and Dermatology Franchise, Novartis Pharma AG, Basel, Switzerland

Meeting: 2016 ACR/ARHP Annual Meeting

Date of first publication: September 28, 2016

Keywords: Systemic JIA 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: Canakinumab (CAN), a highly selective human anti-IL1 β monoclonal antibody, had demonstrated its efficacy and safety in patients (pts) with active systemic juvenile idiopathic arthritis (SJIA) in a comprehensive global clinical program consisting of one phase II and two phase III trials.^1,2However, limited data was available on long-term efficacy and safety of CAN in SJIA. The study objective was to assess the long-term efficacy and safety of CAN treated SJIA pts over a 5-year (yr) follow-up observational period.

Methods: This was an open-label extension (OLE) study (NCT00891046) of SJIA pts participating in the global clinical trials of CAN.³Pts, 2 to <20 yrs of age at the time of enrollment in study, received subcutaneous CAN 4 mg/kg every 4 weeks. Baseline was defined as the starting point of the extension trial. Efficacy assessments were done every 3 months, including adapted pediatric response criteria (aACR), clinical inactive disease, and clinical remission on medication (continuous 12 months of clinical inactive disease). Safety assessments included adverse events (AEs) and serious AEs (SAEs).

Results: Overall, 147 pts to the OLE study had a median treatment duration of 3.2 yrs; total treatment exposure was approximately 365 pt-yrs. Of 147 pts, 100 (68%) completed 96 weeks of treatment, whereas 47 (32%) pts discontinued the study. Another 25 pts (17%) discontinued the study after Week 96. Of the 107 pts with an aACR 30 at entry to the OLE study, 61.7%, 79.4%, and 86.0% have had aACR 100, 90, and 70 responses, respectively at last assessment. At baseline, 32.7% of patients were with inactive disease which increased up to 60%-70% between Week 36 and Week 168. Clinical remission on medication was achieved in 43% pts. In total, 137 (93.2%) pts reported at least 1 AE during the 3.2 yrs median exposure in the study corresponding to 2.009 AEs/100 pt-days (733.6 AEs/100 pt-yrs) with infections (202.7 per 100 pt-yrs) being the most common AE. Overall, 47 (32.0%) pts had at least 1 SAE corresponding to 0.089 SAE/100 pt-days (32.6 SAE/100 pt-yrs) with the most common being JIA (14 pts) denoting disease flares or worsening of SJIA. Ten patients (6.8%) with a total of 12 macrophage activation syndrome (MAS) events were reported as SAE and 7 patients among them discontinued the study. No deaths were reported.

Conclusion: In patients previously treated with CAN in pivotal trials, response to treatment was sustained or improved during long-term treatment in the OLE study. Safety profile of CAN was consistent with safety findings from previous studies. References: 1. Ruperto N, et al. N Engl J Med. 2012;367(25):2396-406. 2. Ringold S, et al. Arthritis & Rheum. 2013;65(10):2499-512. 3. Ruperto N, et al. Ann Rheum Dis. 2015;74(2):608.

Disclosure: H. I. Brunner, Novartis, Roche, Pfizer, UCB, Celgene, Regeneron, Amgen, Astrazeneca, GSK and BMS, 5,Novartis and Roche, 8; N. Ruperto, 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, Biogenidec, Astellas, Alter, AstraZeneca, Boehringer, BMS, CDPharma, Celgene, CrescendoBio, EMD Serono,Hoffman-La Roche, Italfarmaco, Janssen, MedImmune, Medac, Novartis, Novo Nordisk, Pfizer, Sanofi Aventis, Servier, Takeda and Ver, 8; P. Quartier, Abbvie, Chugai-Roche, Novartis, and Pfizer, 2,Abbvie, Novartis, Sobi and Roche, 5,Abbvie, Novartis, Pfizer, Roche and Sobi, 8; T. Constantin, None; E. Alexeeva, Roche, Abbott, Novartis, Pfizer, Bristol-Myers Squibb and Centocor, 2,Roche, Novartis, Merck Sharp Dohme, Bristol-Myers Squibb, Medac and Pfizer, 8; R. Schneider, Novartis, Novimmune, Sobi and Innomar Strategies, 5; I. Kone-Paut, SOBI, Novartis and Roche, 2,Novartis, SOBI, Pfizer, Abbvie and Roche/Chugai, 5; K. Schikler, Novartis, Novimmune, Sobi and Innomar Strategies, 5; K. Marzan, Novartis, Abbvie, 2; N. Wulffraat, Novartis Pharmaceutical Corporation, 2; S. Padeh, None; V. Chasnyk, None; C. Wouters, GSK, Novartis and Roche, 2; J. B. Kuemmerle-Deschner, Novartis Pharmaceutical Corporation, 2,Novartis, SOBI and Baxalta, 5; T. Kallinich, Novartis, 2,Sobi, Novartis, BMS and Roche, 8; B. Lauwerys, None; E. Haddad, None; E. L. Nasonov, None; M. Trachana, Novartis, Abbvie, Bristol Meyers, 2,Novartis, Roche and Pfizer, 5; O. Vougiouka, None; K. Leon, Novartis Pharmaceuticals Corporation, 3; A. Speziale, Novartis Pharma AG, 3; K. Lheritier, Novartis Pharma AG, 1,6Novartis Pharma AG, 3; E. Vritzali, Novartis Pharma AG, 3; D. J. Lovell, National Institutes of Health, 2,Astra-Zeneca, Bristol Meyers Squibb, AbbVee, Pfizer, Roche, Novartis, UCB, Forest Research Institute, Horizon, Johnson & Johnson, Biogen, Takeda, Genentech, Glaxo Smith Kline, Boehringer Ingelheim, Celgene and Jannsen, 5; 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.

To cite this abstract in AMA style:

Brunner HI, Ruperto N, Quartier P, Constantin T, Alexeeva E, Schneider R, Kone-Paut I, Schikler K, Marzan K, Wulffraat N, Padeh S, Chasnyk V, Wouters C, Kuemmerle-Deschner JB, Kallinich T, Lauwerys B, Haddad E, Nasonov EL, Trachana M, Vougiouka O, Leon K, Speziale A, Lheritier K, Vritzali E, Lovell DJ, Martini A. Efficacy and Safety of Canakinumab in Patients with Systemic Juvenile Idiopathic Arthritis: Results from an Open-Label Long-Term Follow-up Study [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). https://acrabstracts.org/abstract/efficacy-and-safety-of-canakinumab-in-patients-with-systemic-juvenile-idiopathic-arthritis-results-from-an-open-label-long-term-follow-up-study/. Accessed .

« Back to 2016 ACR/ARHP Annual Meeting

ACR Meeting Abstracts - https://acrabstracts.org/abstract/efficacy-and-safety-of-canakinumab-in-patients-with-systemic-juvenile-idiopathic-arthritis-results-from-an-open-label-long-term-follow-up-study/