Abstract
BackgroundDespite 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.Methods22 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.ResultsPatients 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.ConclusionsWe firstly showed that high dosage of Pidotimod could be an effective and safe to reduce the PFAPA attacks in children.
Highlights
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
Firstly described by Marshall et al in 1987 [1], periodic fever, aphthous stomatitis, pharyngitis, adenopathy (PFAPA) syndrome is an autoinflammatory disease belonging to the heterogeneous group of the “periodic diseases” and characterized by febrile episodes lasting for 3–6 days with recurrence every 3–8 weeks, associated with at least one of three main symptoms: aphthous stomatitis, pharyngitis and cervical adenitis [2]
Even though 40 years have passed since its first description, the therapeutic options for managing are non-specific and no consensus exists about the best treatment choice to use in children who receive a new diagnosis as well as in refractory patients
Summary
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. Pidotimod has been suggested as a new potential treatment in PFAPA syndrome for its immunodulatory effects. The diagnosis of PFAPA is based on clinical criteria; these criteria have not been validated in a large cohort of patients and show poor specificity [5, 6]. In a recent survey, Authors highlighted the poor adherence of most physicians to these criteria in their clinical practice [7]. By using these new criteria in clinical practice to diagnose PFAPA, a significant number of patients would not be diagnosed, and Author consider them as not useful as diagnostic criteria [6]
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