Novartis Pharma AG, Basel, Switzerland.
Orphanet J Rare Dis. 2013 Sep 10;8:139. doi: 10.1186/1750-1172-8-139.
The Cryopyrin-Associated Periodic Syndromes (CAPS) are a group of rare hereditary autoinflammatory diseases and encompass Familial Cold Autoinflammatory Syndrome (FCAS), Muckle-Wells Syndrome (MWS), and Neonatal Onset Multisystem Inflammatory Disease (NOMID). Canakinumab is a monoclonal antibody directed against IL-1 beta and approved for CAPS patients but requires post-approval monitoring due to low and short exposures during the licensing process. Creative approaches to observational methodology are needed, harnessing novel registry strategies to ensure Health Care Provider reporting and patient monitoring.
A web-based registry was set up to collect information on long-term safety and effectiveness of canakinumab for CAPS.
Starting in November 2009, this registry enrolled 241 patients in 43 centers and 13 countries by December 31, 2012. One-third of the enrolled population was aged < 18; the overall population is evenly divided by gender. Enrolment is ongoing for children.
Innovative therapies in orphan diseases require post-approval structures to enable in depth understanding of safety and natural history of disease. The rarity and distribution of such diseases and unpredictability of treatment require innovative methods for enrolment and follow-up. Broad international practice-based recruitment and web-based data collection are practical.
Cryopyrin 相关周期综合征（CAPS）是一组罕见的遗传性自身炎症性疾病，包括家族性冷自身炎症综合征（FCAS）、穆勒-韦尔斯综合征（MWS）和新生儿发病多系统炎症性疾病（NOMID）。Canakinumab 是一种针对 IL-1β的单克隆抗体，已被批准用于 CAPS 患者，但由于在许可过程中暴露量低且短暂，因此需要进行上市后监测。需要采用创造性的观察方法学方法，利用新的登记策略来确保医疗保健提供者报告和患者监测。
建立了一个基于网络的登记处，以收集有关 Canakinumab 治疗 CAPS 的长期安全性和有效性的信息。
从 2009 年 11 月开始，该登记处到 2012 年 12 月 31 日在 43 个中心和 13 个国家共登记了 241 名患者。登记人口中有三分之一的年龄<18 岁；总体人口在性别上平分秋色。正在为儿童进行登记。
孤儿病的创新疗法需要建立上市后结构，以便深入了解安全性和疾病自然史。此类疾病的罕见性和分布以及治疗的不可预测性需要创新的招募和随访方法。广泛的国际基于实践的招募和基于网络的数据收集是切实可行的。
