Zhang Tian, Yu Yue, Chen Jiajing, Zhao Zhao, Qian Moting, Zhang Yufei, Ma Chunlai, Chang Feng
Institute of Science and Technology, China Pharmaceutical University, Nanjing, 211198, People's Republic of China.
Department of Pharmacy, Huashan Hospital, Fudan University, Shanghai, 200040, People's Republic of China.
J Multidiscip Healthc. 2025 May 9;18:2591-2602. doi: 10.2147/JMDH.S500838. eCollection 2025.
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.
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.
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.
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.
冷吡啉相关周期性综合征（CAPS）是一组由白细胞介素-1（IL-1）介导的自身炎症性疾病，于2023年被纳入中国“第二批罕见病名录”。值得注意的是，自2008年起在美国获批的IL-1抑制剂rilonacept一直是治疗CAPS的关键药物。然而，对其疗效和安全性的综合分析有限。本系统评价旨在评估rilonacept治疗CAPS的效果，并提供循证治疗建议。
我们使用与rilonacept和CAPS相关的医学主题词和自由文本关键词进行系统的文献检索。该检索涵盖了包括PubMed、Ovid/Embase、Cochrane图书馆以及中国数据库（知网、万方和维普）在内的数据库，检索时间跨度从建库至2024年9月30日。随后进行了多维度系统评价，整理了rilonacept治疗CAPS的疗效、安全性、成本效益、创新性、适用性和可及性。
在1223篇筛选出的出版物中，根据既定标准选择了三项临床研究，其中包括两项序贯随机对照试验。值得注意的是，未发现经济评估方面的研究。使用rilonacept治疗使患者的关键症状评分降低了约2分，在所有结局指标上均有显著改善，如发作天数和高敏C反应蛋白水平。不良反应大多为轻至中度，且长期耐受性良好。Rilonacept因其使用方便，满足了当前临床需求，显示出很强的创新性和适用性。其成本效益和可及性在上市后值得进一步研究，但具有广泛应用的巨大潜力。
Rilonacept在治疗CAPS方面显示出显著疗效，总体安全性良好。它具有很高的创新性和可接受的适用性，具有改善成本效益和可及性的潜力。我们预计，rilonacept对CAPS的有效治疗将鼓励进一步的临床和基础研究，为其他自身炎症性疾病的治疗提供有价值的见解。
