APHP, CHU de Bicêtre, Paediatric rheumatology and CEREMAIA, University of Paris Sud Saclay, Le Kremlin Bicêtre, France.
Department of Pediatrics, CHU Sainte Justine Research Centre, Sainte Justine University Hospital, University of Montreal, Montreal, QC, Canada.
Joint Bone Spine. 2022 Nov;89(6):105448. doi: 10.1016/j.jbspin.2022.105448. Epub 2022 Aug 6.
To evaluate the feasibility of the autoinflammatory disease activity index (AIDAI) as a tool to assess disease activity in patients with hereditary recurrent fever syndromes (HRFs) treated with canakinumab.
Patients with active colchicine-resistant familial Mediterranean fever (crFMF), mevalonate kinase deficiency (MKD), or tumor necrosis factor receptor-associated periodic syndrome (TRAPS) were enrolled in the phase III CLUSTER study and asked to complete the AIDAI questionnaire daily. All patients included in the analysis were treated with canakinumab, but regimens and periods of treatment varied per study protocol. The AIDAI for each patient was calculated weekly over the first 40 weeks of study, based on the diaries completed over 30 days. Disease-specific cut-off AIDAI values for inactive disease were calculated in a ROC analysis by comparing AIDAI scores with the occurrence of clinically inactive disease, based on the physician global assessments of disease activity and the occurrence of flares.
Sixty patients with crFMF, 70 with MKD, and 43 with TRAPS were included in the analysis. Median AIDAI scores were high during the first 4 weeks for the three disease cohorts, and decreased afterwards, with some differences between disease cohorts. AIDAI values of 12.0, 9.6 and 15.5 were obtained as the most optimal thresholds to discriminate patients with inactive disease, with sensitivity and specificity values mostly over 75%.
The AIDAI allows to discriminate between patients with active and inactive HRFs, and can be used in clinical practice to monitor the disease course of patients and the effect of medications.
评估自身炎症性疾病活动指数（AIDAI）作为一种工具，用于评估接受卡那单抗治疗的遗传性复发性发热综合征（HRF）患者的疾病活动。
在 III 期 CLUSTER 研究中，纳入了患有活性秋水仙碱耐药家族性地中海热（crFMF）、甲羟戊酸激酶缺乏症（MKD）或肿瘤坏死因子受体相关周期性综合征（TRAPS）的患者，并要求他们每天完成 AIDAI 问卷。所有纳入分析的患者均接受卡那单抗治疗，但治疗方案和治疗时间因研究方案而异。根据 30 天内完成的日记，每周计算一次每位患者的 AIDAI 值，共计算前 40 周的研究。通过比较 AIDAI 评分与临床无活性疾病的发生，根据医生对疾病活动的总体评估和疾病发作的情况，对疾病特异性无活性疾病的 AIDAI 切点值进行了 ROC 分析。
分析中包括 60 例 crFMF 患者、70 例 MKD 患者和 43 例 TRAPS 患者。在这三个疾病队列中，AIDAI 评分在最初的 4 周内较高，之后逐渐下降，但不同疾病队列之间存在差异。12.0、9.6 和 15.5 的 AIDAI 值被确定为区分无活性疾病患者的最佳阈值，敏感性和特异性值大多超过 75%。
AIDAI 可区分活动性和非活动性 HRF 患者，可用于临床实践中监测患者的疾病进程和药物的疗效。
