PMID- 32711565 OWN - NLM STAT- MEDLINE DCOM- 20210708 LR - 20210708 IS - 1824-7288 (Electronic) IS - 1720-8424 (Linking) VI - 46 IP - 1 DP - 2020 Jul 25 TI - Proposal for a new therapeutic high dosage of Pidotimod in children with periodic fever, aphthous stomatitis, pharyngitis, adenitis (PFAPA) syndrome: a randomized controlled study. PG - 106 LID - 10.1186/s13052-020-00871-y [doi] LID - 106 AB - BACKGROUND: 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. METHODS: 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. RESULTS: 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. CONCLUSIONS: We firstly showed that high dosage of Pidotimod could be an effective and safe to reduce the PFAPA attacks in children. FAU - Manti, Sara AU - Manti S AD - Pediatric Respiratory Unit - San Marco Hospital, Department of Clinical and Experimental Medicine, University of Catania, Viale Carlo Azeglio Ciampi, 95121, Catania, Italy. FAU - Filosco, Federica AU - Filosco F AD - Pediatric Respiratory Unit - San Marco Hospital, Department of Clinical and Experimental Medicine, University of Catania, Viale Carlo Azeglio Ciampi, 95121, Catania, Italy. FAU - Parisi, Giuseppe Fabio AU - Parisi GF AUID- ORCID: 0000-0003-4291-0195 AD - Pediatric Respiratory Unit - San Marco Hospital, Department of Clinical and Experimental Medicine, University of Catania, Viale Carlo Azeglio Ciampi, 95121, Catania, Italy. gf.parisi@policlinico.unict.it. FAU - Finocchiaro, Giuseppe Germano AU - Finocchiaro GG AD - Pediatric Respiratory Unit - San Marco Hospital, Department of Clinical and Experimental Medicine, University of Catania, Viale Carlo Azeglio Ciampi, 95121, Catania, Italy. FAU - Papale, Maria AU - Papale M AD - Pediatric Respiratory Unit - San Marco Hospital, Department of Clinical and Experimental Medicine, University of Catania, Viale Carlo Azeglio Ciampi, 95121, Catania, Italy. FAU - Giugno, Andrea AU - Giugno A AD - Pediatric Respiratory Unit - San Marco Hospital, Department of Clinical and Experimental Medicine, University of Catania, Viale Carlo Azeglio Ciampi, 95121, Catania, Italy. FAU - Barone, Patrizia AU - Barone P AD - Pediatric Respiratory Unit - San Marco Hospital, Department of Clinical and Experimental Medicine, University of Catania, Viale Carlo Azeglio Ciampi, 95121, Catania, Italy. FAU - Leonardi, Salvatore AU - Leonardi S AD - Pediatric Respiratory Unit - San Marco Hospital, Department of Clinical and Experimental Medicine, University of Catania, Viale Carlo Azeglio Ciampi, 95121, Catania, Italy. LA - eng PT - Journal Article PT - Randomized Controlled Trial DEP - 20200725 PL - England TA - Ital J Pediatr JT - Italian journal of pediatrics JID - 101510759 RN - 0 (Glucocorticoids) RN - 0 (Immunologic Factors) RN - 0 (Thiazolidines) RN - 785363R681 (pidotimod) RN - 9842X06Q6M (Betamethasone) RN - SZB83O1W42 (Pyrrolidonecarboxylic Acid) SB - IM MH - Betamethasone/administration & dosage MH - Child MH - Child, Preschool MH - Cross-Over Studies MH - Familial Mediterranean Fever/complications/*drug therapy MH - Female MH - Glucocorticoids/administration & dosage MH - Humans MH - Immunologic Factors/*administration & dosage MH - Lymphadenitis/complications/*drug therapy MH - Male MH - Pharyngitis/complications/*drug therapy MH - Prospective Studies MH - Pyrrolidonecarboxylic Acid/administration & dosage/*analogs & derivatives MH - Stomatitis, Aphthous/complications/*drug therapy MH - Syndrome MH - Thiazolidines/*administration & dosage MH - Treatment Outcome PMC - PMC7382793 OTO - NOTNLM OT - Betamethasone OT - Children OT - High dosage OT - Immunomodulation OT - PFAPA OT - Pidotimod OT - Treatment COIS- The authors have no conflicts of interest to disclose that could be perceived as prejudicing the impartiality of the research reported. EDAT- 2020/07/28 06:00 MHDA- 2021/07/09 06:00 PMCR- 2020/07/25 CRDT- 2020/07/27 06:00 PHST- 2020/04/23 00:00 [received] PHST- 2020/07/16 00:00 [accepted] PHST- 2020/07/27 06:00 [entrez] PHST- 2020/07/28 06:00 [pubmed] PHST- 2021/07/09 06:00 [medline] PHST- 2020/07/25 00:00 [pmc-release] AID - 10.1186/s13052-020-00871-y [pii] AID - 871 [pii] AID - 10.1186/s13052-020-00871-y [doi] PST - epublish SO - Ital J Pediatr. 2020 Jul 25;46(1):106. doi: 10.1186/s13052-020-00871-y.