Treatment options for periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome in children and adults: a narrative review

Gaggiano, Carla; Rigante, Donato; Sota, Jurgen; Grosso, Salvatore; Cantarini, Luca

doi:10.1007/s10067-018-4361-2

Cited by 57 publications

(63 citation statements)

References 60 publications

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“…The periodic fever, aphthous stomatitis, pharyngitis, and adenitis (PFAPA) syndrome is an autoinflammatory condition characterized by the clinical features described by the acronym itself. Other various manifestations may occur in addition to cardinal signs, including skin rash, abdominal pain, arthralgia, and conjunctivitis [136][137][138]. This syndrome is currently the most frequent autoinflammatory cause of noninfective recurrent fever among children in the European population.…”

Section: Multifactorial Autoinflammatory Diseases: Working For a Propmentioning

confidence: 99%

“…Noteworthy, the enlargement of the cervical lymph node represents a diagnostic clue for patients with the PFAPA syndrome and should be taken into account for patients presenting with laterocervical lymphadenopathy solely during fever attacks. Cervical lymphadenitis is part of diagnostic criteria drafted for both PFAPA children and adults [136][137][138][144][145][146].…”

Section: Visceral Involvementmentioning

confidence: 99%

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Hints for Genetic and Clinical Differentiation of Adult-Onset Monogenic Autoinflammatory Diseases

Gaggiano

Rigante

Vitale

et al. 2019

Mediators of Inflammation

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Monogenic autoinflammatory diseases (mAIDs) are inherited errors of innate immunity characterized by systemic inflammation recurring with variable frequency and involving the skin, serosal membranes, synovial membranes, joints, the gastrointestinal tube, and/or the central nervous system, with reactive amyloidosis as a potential severe long-term consequence. Although individually uncommon, all mAIDs set up an emerging chapter of internal medicine: recent findings have modified our knowledge regarding mAID pathophysiology and clarified that protean inflammatory symptoms can be variably associated with periodic fevers, depicting multiple specific conditions which usually start in childhood, such as familial Mediterranean fever, tumor necrosis factor receptor-associated periodic syndrome, cryopyrin-associated periodic syndrome, and mevalonate kinase deficiency. There are no evidence-based studies to establish which potential genotype analysis is the most appropriate in adult patients with clinical phenotypes suggestive of mAIDs. This review discusses genetic and clinical hints for an ideal diagnostic approach to mAIDs in adult patients, as their early identification is essential to prompt effective treatment and improve quality of life, and also highlights the most recent developments in the diagnostic work-up for the most frequent hereditary periodic febrile syndromes worldwide.

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Section: Multifactorial Autoinflammatory Diseases: Working For a Propmentioning

confidence: 99%

Section: Visceral Involvementmentioning

confidence: 99%

Hints for Genetic and Clinical Differentiation of Adult-Onset Monogenic Autoinflammatory Diseases

Gaggiano

Rigante

Vitale

et al. 2019

Mediators of Inflammation

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“…We aimed to investigate the e cacy and safety of Pidotimod, administered at high doses, in treating patients affected by PFAPA syndrome. To achieve this goal a preliminary, prospective, controlled, open, cross-over study was performed.Currently, the treatment of PFAPA syndrome is primarily targeted to the resolution of acute in ammatory episodes, although there is no consensus concerning the best remedy for this disease [16,17]. Due to the limitation of the treatment as well as the nature of an acute in ammatory response occurring in PFAPA syndrome, authors aimed at assessing immunological mechanisms behind this condition to identify immune mediators that, associated pathogenetically with PFAPA, can be new potential therapeutic target [17].…”

Section: Discussionmentioning

confidence: 99%

“…To achieve this goal a preliminary, prospective, controlled, open, cross-over study was performed.Currently, the treatment of PFAPA syndrome is primarily targeted to the resolution of acute in ammatory episodes, although there is no consensus concerning the best remedy for this disease [16,17]. Due to the limitation of the treatment as well as the nature of an acute in ammatory response occurring in PFAPA syndrome, authors aimed at assessing immunological mechanisms behind this condition to identify immune mediators that, associated pathogenetically with PFAPA, can be new potential therapeutic target [17]. Among the molecules capable of interfering in the innate and adaptive immune response, Pidotimod, an immunomodulatory agent able in increasing antigen presentation and promoting adaptive Th1-mediated immunity, showed good e cacy and safety when administered in, addition to a bacterial lysate, in children aged 3-12 years with PFAPA syndrome [13].…”

Section: Discussionmentioning

confidence: 99%

Proposal for a new therapeutic high dosage of Pidotimod in children with Periodic fever, aphthous stomatitis, pharyngitis, adenitis (PFAPA) syndrome: a randomized controlled study.

Manti

Filosco

Parisi

et al. 2020

Preprint

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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 400mg daily) in combination with betamethasone 0.5-1 mg on need, based on parents/caregivers' decision (group A) or betamethasone 0.5-1mg 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.

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“…Even so, the concept that “if the corticosteroid works, it must be PFAPA syndrome” is hazardous, as clinical signs to support PFAPA diagnosis are nonspecific, and suggesting to use corticosteroids as antipyretics liberally should be frankly discouraged in children. A complete understanding of the syndrome is probably not far to come, as both interleukin‐1‐related genes and interferon‐induced genes are overexpressed during PFAPA flares 44‐47 …”

mentioning

confidence: 99%

When, how, and why do fevers hold children hostage?

Rigante

2020

J Evidence Based Medicine

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Treatment options for periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome in children and adults: a narrative review

Cited by 57 publications

References 60 publications

Hints for Genetic and Clinical Differentiation of Adult-Onset Monogenic Autoinflammatory Diseases

Hints for Genetic and Clinical Differentiation of Adult-Onset Monogenic Autoinflammatory Diseases

Proposal for a new therapeutic high dosage of Pidotimod in children with Periodic fever, aphthous stomatitis, pharyngitis, adenitis (PFAPA) syndrome: a randomized controlled study.

When, how, and why do fevers hold children hostage?

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