An interim analysis of post-marketing all-patients surveillance of canakinumab in Japanese patients with Familial Mediterranean fever

Abstract

Objective: A post-marketing all-patient surveillance of canakinumab is underway to assess its safety and effectiveness in Japanese patients with colchicine-resistant Familial Mediterranean fever（crFMF）. We report an interim analysis 2.5 years post-launch of all crFMF patients who received canakinumab in daily medical practice. Methods: Thirty-nine patients were included in a data lock as of 30 June 2019, and all patients were included in the safety set and effectiveness set. Results: The mean age of the patients at treatment initiation was 40 years. The majority of patients were atypical cases with MEFV gene mutation（non-Exon10）（n=22）, followed by typical cases with gene mutation（Exon10）（n=10）, atypical cases without gene mutation（n=5）, and typical cases with unknown gene mutation（n=2）. Adverse drug reactions（ADRs）were reported in 25.64%（n=10）, and serious ADRs were reported in 10.26%（n=4）（two events of osteoarthritis and one event each of diverticulitis, haemosiderosis, enterovesical fistula, bursitis, bursal fluid accumulation, oedema, and pain）. Although the proportion of responders as defined in this survey was 20.51%（n=8/39）, the proportion of clinical remission was 65.71%（n=23/35）and that of serological remission was 85.29%（n=29/34）. Conclusion: This interim analysis showed no new safety concern. We will continue to assess the safety and effectiveness of canakinumab through this surveillance.