目的:RELIANCE注册中心的中期分析,一个正在进行的,非干预性,开放标签,多中心,评估长期安全性的前瞻性研究,canakinumab在冷冻比林相关周期性综合征(CAPS)患者中的给药方案和有效性,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)。
方法:自2017年9月起,针对CAPS患者,和2018年6月FMF患者,陷阱或MKD/HIDS,该研究纳入了接受canakinumab作为常规医疗服务一部分的儿科(≥2岁)和成年(≥18岁)患者.安全,canakinumab剂量,在基线时和每6个月评估疾病活动和生活质量结局指标,直至研究访视结束.
结果:在分析截止日期(2020年12月),168例患者(91例CAPS,54FMF,纳入16个TRAPS和7个MKD/HIDS)。85例(50.9%)患者为女性,72例(43.1%)为儿童(<18岁)。患者年龄中位数为20.0岁(范围2.0-79.0岁)。在CAPS队列中,在接受高于推荐起始剂量(SD)的canakinumab的患者中,严重感染和严重药物不良反应更为常见.在汇集的群体中观察到接受>SD的卡纳单抗的趋势。据报道,大多数患者从基线到第30个月无或轻度/中度疾病活动(医生的全球评估),在canakinumab治疗下缓解的患者比例稳定(~70%)。患者报告的疾病活动(视觉模拟量表(VAS),自身炎性疾病活动指数),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)始终得到良好控制。
结论:这项分析的数据证实了卡纳单抗治疗CAPS的长期安全性和有效性,FMF,陷阱和MKD/HIDS。
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.
CONCLUSIONS: Data from this analysis confirm the long-term safety and effectiveness of canakinumab for the treatment of CAPS, FMF, TRAPS and MKD/HIDS.