Abstract:
Background: Cryopyrin-associated periodic syndromes (CAPS) is a group of rare autoinflammatory diseases. Little is known about the burden of disease, patients\' views on treatment, and adverse events (AEs) with current therapy. Objectives: The main study objective was to quantify the patients\' burden of disease in terms of flares and resource use and to characterize patient symptomatology and tolerability of treatment with anakinra. A secondary objective included comparing chart review and patient recall of symptoms and AEs. Methods: A retrospective medical chart review and concurrent online patient survey was conducted in four European countries. Data 12 months prior to initiation of/during anakinra treatment were entered into web-based case report forms by study groups. Results: Forty-two patients received/were receiving anakinra as primary treatment for at least 12 months. Patients experienced a 79.5% reduction in flares after commencing anakinra treatment. During the past 12 months on anakinra, four of five (80%) patients who recalled experiencing flares reported cancelling social activities and staying home as the most common courses of action. Most common AEs were injection site pain upon treatment initiation and weight gain. According to patient recall, 12 of 21 patients (57.1%) discontinued anakinra to enter another clinical trial; of the 12, eight (38%) specifically discontinued anakinra only for that reason, and four patients cited entering a clinical trial as one of many reasons for discontinuing anakinra. Conclusions: To our knowledge, this is the most comprehensive survey of patient experience with CAPS. Although improved, CAPS treatment remains suboptimal and a significant burden is placed upon patients, caregivers, and the healthcare system. With new agents available, it will be important to compare outcomes in patients using all therapeutic options.
摘要:
背景:Cryopyrin相关周期性综合征(CAPS)是一组罕见的自身炎症性疾病。人们对疾病的负担知之甚少,患者对治疗的看法,和当前治疗的不良事件(AE)。目标:主要研究目标是根据耀斑和资源使用来量化患者的疾病负担,并描述患者的症状学和用阿那酚酮治疗的耐受性。次要目标包括比较图表回顾和患者对症状和AE的回忆。方法:在四个欧洲国家进行了回顾性医学图表审查和并发的在线患者调查。在开始anakinra治疗之前的12个月/期间的数据由研究组输入到基于网络的病例报告表中。结果:42例患者接受/正在接受anakinra作为主要治疗至少12个月。开始anakinra治疗后,患者的耀斑减少了79.5%。在阿纳金拉的过去12个月里,回忆经历耀斑的五名患者中有四名(80%)报告说,取消社交活动和呆在家里是最常见的行动方案。最常见的AE是治疗开始时的注射部位疼痛和体重增加。根据患者回忆,21名患者中有12名(57.1%)停止了anakinra进入另一项临床试验;在12名患者中,有8名(38%)仅出于该原因而专门停止了anakinra,四名患者称进入临床试验是停用anakinra的众多原因之一。结论:据我们所知,这是对CAPS患者体验的最全面调查.虽然改进了,CAPS治疗仍然欠佳,患者负担很大,看护者,和医疗保健系统。有了新的代理,在使用所有治疗方案的患者中比较结局将是重要的.