在单基因自身炎症性疾病患者中，卡那奴单抗的长期安全性和有效性：RELIANCE 登记处的中期分析结果。
Long-term safety and effectiveness of canakinumab in patients with monogenic autoinflammatory diseases: results from the interim analysis of the RELIANCE registry.
机构信息
Division of Paediatric Rheumatology and autoinflammation reference centre Tübingen, Department of Paediatrics, University Hospital Tübingen, Tübingen, Germany
Department of Paediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité Universitätsmedizin and Deutsches Rheuma-Forschungszentrum (DRFZ), an Institute of the Leibniz Association, Berlin, Germany.
出版信息
RMD Open. 2024 Feb 15;10(1):e003890. doi: 10.1136/rmdopen-2023-003890.
OBJECTIVE
Interim analysis of the RELIANCE registry, an on-going, non-interventional, open-label, multicentre, prospective study evaluating the long-term safety, dosing regimens and effectiveness of canakinumab in patients with cryopyrin-associated periodic syndromes (CAPS), familial Mediterranean fever (FMF), tumour-necrosis factor receptor-associated periodic syndrome (TRAPS) or mevalonate-kinase deficiency (MKD)/hyperimmunoglobulin-D syndrome (HIDS).
METHODS
From September 2017 for patients with CAPS, and June 2018 for patients with FMF, TRAPS or MKD/HIDS, the registry enrolled paediatric (aged ≥2 years) and adult patients (aged ≥18 years) receiving canakinumab as part of their routine medical care. Safety, canakinumab dose, disease activity and quality of life outcome measures were evaluated at baseline and every 6 months until end of study visit.
RESULTS
At the analysis cut-off date (December 2020), 168 patients (91 CAPS, 54 FMF, 16 TRAPS and 7 MKD/HIDS) were enrolled. 85 (50.9%) patients were female and 72 (43.1%) were children (<18 years). The median patient age was 20.0 years (range 2.0-79.0 years). In the CAPS cohort, serious infections and serious adverse drug-reactions were more common in patients receiving higher than the recommended starting dose (SD) of canakinumab. A trend to receive >SD of canakinumab was observed in the pooled population. The majority of patients were reported as having either absent or mild/moderate disease activity (physician's global assessment) from baseline to Month 30, with a stable proportion of patients (~70%) in remission under canakinumab treatment. Patient-reported disease activity (Visual Analogue Scale (VAS), Autoinflammatory Disease Activity Index), fatigue (VAS); markers of inflammation (C-reactive protein, serum amyloid A and erythrocyte sedimentation rate) remained well-controlled throughout.
CONCLUSION
Data from this analysis confirm the long-term safety and effectiveness of canakinumab for the treatment of CAPS, FMF, TRAPS and MKD/HIDS.
目的
RELIANCE 注册研究是一项正在进行的、非干预性的、开放性标签的、多中心前瞻性研究，旨在评估卡那单抗在 Cryopyrin 相关周期性综合征（CAPS）、家族性地中海热（FMF）、肿瘤坏死因子受体相关周期性综合征（TRAPS）或甲羟戊酸激酶缺乏症（MKD）/高免疫球蛋白 D 综合征（HIDS）患者中的长期安全性、给药方案和疗效。
方法
自 2017 年 9 月起，针对 CAPS 患者，自 2018 年 6 月起，针对 FMF、TRAPS 或 MKD/HIDS 患者，该注册研究招募了接受卡那单抗作为常规医疗护理一部分的儿科（≥2 岁）和成人（≥18 岁）患者。在基线和每 6 个月评估安全性、卡那单抗剂量、疾病活动度和生活质量结局指标，直至研究访问结束。
结果
在分析截止日期（2020 年 12 月），共纳入 168 例患者（91 例 CAPS、54 例 FMF、16 例 TRAPS 和 7 例 MKD/HIDS）。85 例（50.9%）为女性，72 例（43.1%）为儿童（＜18 岁）。中位患者年龄为 20.0 岁（范围 2.0-79.0 岁）。在 CAPS 队列中，接受高于推荐起始剂量（SD）的卡那单抗治疗的患者更常见严重感染和严重药物不良反应。在汇总人群中观察到接受＞SD 卡那单抗的趋势。从基线到第 30 个月，大多数患者报告疾病活动度（医生总体评估）为无或轻度/中度，在卡那单抗治疗下缓解的患者比例稳定（约 70%）。患者报告的疾病活动度（视觉模拟量表（VAS）、自身炎症性疾病活动指数）、疲劳（VAS）；炎症标志物（C 反应蛋白、血清淀粉样蛋白 A 和红细胞沉降率）在整个过程中均得到良好控制。
结论
该分析数据证实了卡那单抗治疗 CAPS、FMF、TRAPS 和 MKD/HIDS 的长期安全性和有效性。
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