Koru Lutfiye, Erten Elif Selcen Yabanci, Dursun Hatice Kubra, Dizman Eda Nur, Kaya Feray, Kucuk Elif, Aydin Zelal, Balci Merve Ozen, Ozturk Kubra, Haslak Fatih
Department of Pediatric Rheumatology, Istanbul Medeniyet University Istanbul, Istanbul, Turkey.
Department of Pediatrics, Istanbul Medeniyet University, Istanbul, Turkey.
Eur J Pediatr. 2025 May 2;184(5):322. doi: 10.1007/s00431-025-06158-w.
We aimed to evaluate the efficacy of prophylactic colchicine treatment in periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) using the Auto-Inflammatory Diseases Activity Index (AIDAI) score measured before and during treatment. We also aimed to determine the optimal treatment duration for observing clinical response and to identify factors predicting response to treatment. Children with PFAPA receiving colchicine were included in the study. We utilized AIDAI scoring system by evaluating the sore throat, tonsillitis, and oral aphthae under the general symptoms heading. This form of AIDAI was given to the families, and they were asked to document their symptoms on a daily basis. AIDAI scores were calculated for the month before colchicine initiation and for the first and third months after. Data were obtained retrospectively. The mean age of patients was 4.4 ± 2.13 years, and almost two third of them (50/76) were male. The difference in AIDAI scores between the month before colchicine treatment and the 1st month after the treatment was significant, while the difference between the 1st and 3rd month after the treatment was not (p = 0.002 vs. p = 0.463). Patient treatment responses were: 44 (57.9%) complete response, 15 (19.7%) partial response, and 17 (22.4%) unresponsive to colchicine.
Based on this finding, we can conclude that in the case of partial response to colchicine treatment after the first month, dose escalation, review of treatment compliance and the need for prophylaxis should be reevaluated.
• Colchicine prophylaxis is recommended in PFAPA patients; however, the optimal time point for response assessment remains unclear. • The AIDAI score is suggested for measuring disease activity in autoinflammatory conditions.
• This is the first repeated-measure study evaluating colchicine response in PFAPA patients using the AIDAI score. • Clinical response predominantly emerged within the first month, suggesting this period may be critical for evaluation.
我们旨在使用治疗前和治疗期间测量的自身炎症性疾病活动指数（AIDAI）评分，评估预防性秋水仙碱治疗周期性发热、阿弗他口炎、咽炎和颈淋巴结炎（PFAPA）的疗效。我们还旨在确定观察临床反应的最佳治疗持续时间，并确定预测治疗反应的因素。纳入研究的是接受秋水仙碱治疗的PFAPA患儿。我们通过在一般症状项下评估咽痛、扁桃体炎和口腔阿弗他溃疡来使用AIDAI评分系统。这种形式的AIDAI评分表发给了患儿家属，并要求他们每天记录症状。计算秋水仙碱开始使用前一个月以及开始使用后的第一个月和第三个月的AIDAI评分。数据为回顾性获取。患者的平均年龄为4.4±2.13岁，其中近三分之二（50/76）为男性。秋水仙碱治疗前一个月与治疗后第一个月的AIDAI评分差异有统计学意义，而治疗后第一个月与第三个月的差异无统计学意义（p = 0.002对比p = 0.463）。患者的治疗反应为：44例（57.9%）完全缓解，15例（19.7%）部分缓解，17例（22.4%）对秋水仙碱无反应。
基于这一发现，我们可以得出结论，在第一个月后秋水仙碱治疗出现部分反应的情况下，应重新评估剂量增加、治疗依从性以及预防的必要性。
• 推荐对PFAPA患者进行秋水仙碱预防；然而，反应评估的最佳时间点仍不明确。• 建议使用AIDAI评分来测量自身炎症性疾病的疾病活动度。
• 这是第一项使用AIDAI评分评估PFAPA患者秋水仙碱反应的重复测量研究。• 临床反应主要在第一个月内出现，表明该时期可能对评估至关重要。
