Pediatric Respiratory Unit - San Marco Hospital, Department of Clinical and Experimental Medicine, University of Catania, Viale Carlo Azeglio Ciampi, 95121, Catania, Italy.
Ital J Pediatr. 2020 Jul 25;46(1):106. doi: 10.1186/s13052-020-00871-y.
Despite to PFAPA syndrome is considered a benign and self-limited condition in childhood its impact on patients and families can be remarkable in many cases. Currently, the therapeutic options for managing are non-specific and no consensus exists about the best treatment to use. Pidotimod has been suggested as a new potential treatment in PFAPA syndrome for its immunodulatory effects. We conducted a preliminary, prospective, controlled, open, cross-over trial to assess the efficacy and the safety of Pidotimod in the treatment of children with PFAPA syndrome.
22 children with PFAPA syndrome were randomly allocated to treatment with pidotimod (with 2 vials of 400 mg daily) in combination with betamethasone 0.5-1 mg on need, based on parents/caregivers' decision (group A) or betamethasone 0.5-1 mg on need, based on parents/caregivers' decision (group B). Each treatment period was for 3 months (Phase 1), after that patients were switched to the other arm for other 3 months (Phase 2). Efficacy was expressed in terms of number of episodes of fever, pharyngitis, or aphthous stomatitis, as well as the additional use of betamethasone on need. Safety and tolerability of the Pidotimod were evaluated on the basis of the number and type of adverse events (AEs) recorded during the treatment.
Patients receiving Pidotimod and use betametasone showed a significant decrease in frequency of fevers (p = 0.002); number of episodes of pharyngitis (p = 0.049); aphthous stomatitis (p = 0.036) as well as the betamethasone use on need (p = 0.007). Overall, 19/22 (86.4%) showed benefits from Pidotimod administration. The safety profile of Pidotimod was excellent as no serious adverse events have been reported in the treated groups.
We firstly showed that high dosage of Pidotimod could be an effective and safe to reduce the PFAPA attacks in children.
尽管 PFAPA 综合征被认为是儿童期一种良性且自限性疾病，但在许多情况下，它对患者和家庭的影响可能非常显著。目前，管理该病的治疗方法是非特异性的，并且对于使用哪种最佳治疗方法尚无共识。吡咯烷酮二硫代氨基甲酸盐因其免疫调节作用而被提议作为 PFAPA 综合征的一种新的潜在治疗方法。我们进行了一项初步的、前瞻性的、对照的、开放的交叉试验，以评估吡咯烷酮二硫代氨基甲酸盐在治疗 PFAPA 综合征儿童中的疗效和安全性。
22 名 PFAPA 综合征患儿被随机分为两组，一组接受吡咯烷酮二硫代氨基甲酸盐（每天 2 瓶 400mg）联合家长/照顾者决定按需使用倍他米松 0.5-1mg（A 组），另一组接受家长/照顾者决定按需使用倍他米松 0.5-1mg（B 组）。每个治疗期为 3 个月（第 1 阶段），之后患者切换到另一组再治疗 3 个月（第 2 阶段）。疗效以发热、咽炎或口腔溃疡发作次数以及按需使用倍他米松的次数来表示。根据治疗期间记录的不良事件（AE）的数量和类型评估吡咯烷酮二硫代氨基甲酸盐的安全性和耐受性。
接受吡咯烷酮二硫代氨基甲酸盐和倍他米松治疗的患者发热频率（p=0.002）、咽炎发作次数（p=0.049）、口腔溃疡（p=0.036）以及按需使用倍他米松的次数（p=0.007）均显著减少。总体而言，22 名患者中有 19 名（86.4%）从吡咯烷酮二硫代氨基甲酸盐治疗中获益。吡咯烷酮二硫代氨基甲酸盐的安全性良好，治疗组未报告严重不良事件。
我们首次表明，高剂量吡咯烷酮二硫代氨基甲酸盐可有效且安全地减少儿童 PFAPA 发作。
