OP0061 Kineret® (Anakinra) Controls Disease Symptoms in Patients with Severe Cryopyrin-Associated Periodic Syndromes (CAPS): Up to 5-Year Follow-Up Data

Background In severe forms of the rare monogenic autoinflammatory syndrome CAPS (NOMID, also denoted CINCA), persistent systemic and organ specific inflammation includes rash, central nervous system, and eye and cochlear inflammation leading to progressive hearing and vision loss. CAPS is caused by an uncontrolled IL-1β release, demonstrated by the often complete response after treatment with the IL-1 blocker anakinra (Kineret®) in all three entities of CAPS 1,2,3 Objectives To characterize the long-term efficacy and safety of Kineret® in patients with severe CAPS. Methods A prospective, open-label withdrawal design study with long-term extension including 43 patients (7 adult and 36 pediatric) was conducted at the NIH (study 03-AR-0298). Diary disease sum scores (DSSS), inflammatory serum markers, CNS inflammation, hearing, vision, joint status, quality of life, and safety were evaluated for up to 5 years, and the pharmacokinetics and dosing of Kineret® in CAPS patients were evaluated. Results The median follow up time for the 43 patients was 4.9 years (range Conclusions Kineret® treatment for up to 5 years in severe CAPS patients was safe and resulted in the control of disease symptoms, inflammatory markers, CNS inflammation, and stabilized organ function. Careful monitoring with rapid individual dosing adjustments is important to achieve optimal control of organ inflammation. References Hawkins PN et al. N Engl J Med 2003;348:2583–4 Hoffman HM et al. Lancet 2004;364:1779–85. Goldbach-Mansky et al.N Engl J Med 2006;355:581–92. Sibley CH et al. Arthritis Rheum 2012;64:2375–2386. Disclosure of Interest H. Olivecrona Employee of: Swedish Orphan Biovitrum AB, M. Alden Raboisson Employee of: Swedish Orphan Biovitrum AB, K. Soderberg Employee of: Swedish Orphan Biovitrum AB, B. Hallen Employee of: Swedish Orphan Biovitrum AB, M. Leinonen Consultant for: Swedish Orphan Biovitrum AB, C. Sibley: None Declared, N. Plass: None Declared, C. Brewer: None Declared, K. King: None Declared, C. Zalewski: None Declared, J. Kim: None Declared, R. Bishop: None Declared, S. Hill: None Declared, S. Paul: None Declared, D. Stone: None Declared, D. Chapelle: None Declared, J. Butman: None Declared, R. Goldbach-Mansky Grant/research support from: Swedish Orphan Biovitrum AB, Regeneron, and Novartis