The First International Conference on Periodic Fever, Aphthous Stomatitis, Pharyngitis, Adenitis Syndrome

Abstract

Table I. The major autoinflammatory syndromes FMF Mevalonate kinase deficiency, formerly hyperimmunoglobulinemia D and periodic fever syndrome Tumor necrosis factor associated periodic syndrome Cryopyrin-associated periodic syndromes Familial cold autoinflammatory syndrome Muckle-Well syndrome Neonatal onset multisystem inflammatory disease or Chronic infantile neurological cutaneous and articular syndrome Deficiencies in receptor antagonists Deficiency in IL-1 receptor antagonist Deficiency in IL-36 receptor antagonist Interferonopathies Nakajo-Nishimura syndrome Chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature syndrome Joint contractures, muscular atrophy, microcytic anemia, and panniculitis-induced lipodystrophy syndrome Stimulator of interferon genes associated vasculopathy with onset in infancy Aicardi Goutières syndrome Others PFAPA Chronic recurrent multifocal osteomyelitis Pyogenic arthritis, pyoderma gangrenosum, acne syndrome Blau syndrome/early-onset sarcoidosis Deficiency of IL-10 or IL-10 receptor Deficiency of adenosine deaminase 2

Consensus treatment plans for periodic fever, aphthous stomatitis, pharyngitis and adenitis syndrome (PFAPA): a framework to evaluate treatment responses from the childhood arthritis and rheumatology research alliance (CARRA) PFAPA work group

“…Current therapeutic studies in PFAPA are limited by small, heterogeneous patient populations, variable diagnostic criteria, retrospective data collection, and treatment analysis that did not include comparator arms between therapeutic options [30]. Our prior survey showed considerable variation in treatment of PFAPA, both between and within subspecialties [18].…”

“…Evidence for IL-1 blockade, while promising, was anecdotal or based on small case series and uncontrolled trials so was not included as a therapeutic option [8,9,29,30].…”

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

“…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].…”

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

“…Currently, the treatment of PFAPA syndrome is primarily targeted to the resolution of acute inflammatory 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 inflammatory 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 ].…”