Tian Zhang,1 Yue Yu,2 Jiajing Chen,2 Zhao Zhao,2 Moting Qian,2 Yufei Zhang,2 Chunlai Ma,2 Feng Chang3 

1Institute of Science and Technology, China Pharmaceutical University, Nanjing, 211198, People’s Republic of China; 2Department of Pharmacy, Huashan Hospital, Fudan University, Shanghai, 200040, People’s Republic of China; 3School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing, 211198, People’s Republic of China

Correspondence: Feng Chang, Email cfcpu@163.com Chunlai Ma, Email chunlaima@126.com

Objective: Cryopyrin-associated periodic syndromes (CAPS), a group of interleukin-1 (IL-1) mediated autoinflammatory disorders, were incorporated into China’s “Second List of Rare Diseases” in 2023. Notably, rilonacept, an IL-1 inhibitor approved in the United States since 2008, has been a key treatment for CAPS. However, comprehensive analysis of its efficacy and safety are limited. This systematic review aims to assess rilonacept for CAPS treatment and provide evidence-based therapeutic recommendations.
Methods: We used medical subject headings terms and free-text keywords related to rilonacept and CAPS to perform a systematic literature search. This search covered databases including PubMed, Ovid/Embase, The Cochrane Library, and Chinese databases (CNKI, Wanfang, and VIP) from their inception to September 30, 2024. A multidimensional systematic review was then conducted, collating the efficacy, safety, cost-effectiveness, innovativeness, suitability, and accessibility of rilonacept in treating CAPS.
Results: Out of 1223 screened publications, three clinical studies including two sequential randomized controlled trials were selected based on the established criteria. Notably, no economic evaluations were identified. Treatment with rilonacept resulted in a reduction of approximately two points in key symptom scores for patients, with significant improvements in all outcome measures such as the number of flare days and high-sensitivity C-reactive protein levels. Adverse reactions were mostly mild to moderate, and with favorable long-term tolerability. Rilonacept meets current clinical needs due to its ease of use, demonstrating strong innovativeness and suitability. Its cost-effectiveness and accessibility warrant further examination post-market entry, yet it exhibits considerable potential for widespread use.
Conclusion: Rilonacept demonstrates significant effectiveness in treating CAPS with a favorable overall safety profile. It shows high innovativeness and acceptable suitability, with the potential for improved cost-effectiveness and accessibility. We anticipate that the effective treatment of CAPS with rilonacept will encourage further clinical and fundamental research, offering valuable insights for the treatment of other autoinflammatory diseases.

Keywords: rilonacept, cryopyrin-associated periodic syndromes, interleukin-1 systematic review, clinical comprehensive summary