Literature DB >> 24233262

Clinical and genetic profile of children with periodic fever syndromes from a single medical center in South East Michigan.

Shanmuganathan Chandrakasan, Saurabh Chiwane, Matthew Adams, Basil M Fathalla.   

Abstract

OBJECTIVE: To report a cohort of children with periodic fever syndromes (PFS) from Southeast Michigan.
METHODS: A retrospective review of medical records for patients referred for periodic fever over 5 years.
RESULTS: Sixty-six patients including 21 FMF, 15 PFAPA, four TRAPS and one patient with combined HIDS and FMF were included. In addition, 25 patients were categorized as clinical PFS (cPFS) based on their clinical features however their genetic workup was either negative or inconclusive. Majority of the patients with FMF were from Middle Eastern background (88 %), but positive family history was noted in only 55 % of cases. Mean age at diagnosis was 40.8 months with a mean delay in diagnosis of 24 months. Most common MEFV mutations were p.M694V and p.M694I. Four patients with TRAPS were from mixed European descent and age at onset of symptoms was 6, 12, 12, and 84 months respectively. TNFRSF1A sequence variants in the TRAPS patients included p.R121Q (R92Q) and p.C99G (C70G); one patient had a rare occurrence of a concurrent p.V726A/-MEFV mutation. One patient with HIDS and FMF presented with atypical overlapping PFS clinical manifestations and genetic evaluation showed a unique combination of p.I268T/p.V377I MVK mutations and p.E230K/-MEFV variant. All patients with PFAPA group were from mixed European descent, symptoms started at a mean age of 34.6 months with a mean delay in diagnosis of 23.3 months. Symptoms started during infancy in six patients. All patients fulfilled the diagnostic criteria for PFAPA. The mean age of onset of symptoms in cPFS group was 17.2 months. Empiric colchicine and glucocorticosteroids controlled flares in majority of patients with cPFS. No evidence of amyloidosis was found in this entire cohort of 66 patients after a mean of 29.2 months of follow-up.
CONCLUSION: PFS can present with atypical manifestations and should not be excluded based on a negative family history. Concomitant mutations in different autoinflammatory disorders genes can be present and possibly explain atypical manifestations. Various therapies may be considered even if genetic testing is inconclusive or negative.

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Year:  2014        PMID: 24233262     DOI: 10.1007/s10875-013-9960-8

Source DB:  PubMed          Journal:  J Clin Immunol        ISSN: 0271-9142            Impact factor:   8.317


  26 in total

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Authors:  M F McDermott; I Aksentijevich; J Galon; E M McDermott; B W Ogunkolade; M Centola; E Mansfield; M Gadina; L Karenko; T Pettersson; J McCarthy; D M Frucht; M Aringer; Y Torosyan; A M Teppo; M Wilson; H M Karaarslan; Y Wan; I Todd; G Wood; R Schlimgen; T R Kumarajeewa; S M Cooper; J P Vella; C I Amos; J Mulley; K A Quane; M G Molloy; A Ranki; R J Powell; G A Hitman; J J O'Shea; D L Kastner
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2.  Sacroiliitis in familial Mediterranean fever and seronegative spondyloarthropathy: importance of differential diagnosis.

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3.  The E148Q mutation in the MEFV gene: is it a disease-causing mutation or a sequence variant?

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7.  The frequency of sacroiliitis in familial Mediterranean fever and the role of HLA-B27 and MEFV mutations in the development of sacroiliitis.

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8.  Long-term follow-up, clinical features, and quality of life in a series of 103 patients with hyperimmunoglobulinemia D syndrome.

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Journal:  Eur J Pediatr       Date:  2007-05-23       Impact factor: 3.183

10.  Genetic variation in the familial Mediterranean fever gene (MEFV) and risk for Crohn's disease and ulcerative colitis.

Authors:  Alexandra-Chloé Villani; Mathieu Lemire; Edouard Louis; Mark S Silverberg; Catherine Collette; Geneviève Fortin; Elaine R Nimmo; Yannick Renaud; Sébastien Brunet; Cécile Libioulle; Jacques Belaiche; Alain Bitton; Daniel Gaudet; Albert Cohen; Diane Langelier; John D Rioux; Ian D R Arnott; Gary E Wild; Paul Rutgeerts; Jack Satsangi; Séverine Vermeire; Thomas J Hudson; Denis Franchimont
Journal:  PLoS One       Date:  2009-09-28       Impact factor: 3.240

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1.  Physicians' perspectives on the diagnosis and management of periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome.

Authors:  Kalpana Manthiram; Suzanne C Li; Jonathan S Hausmann; Gil Amarilyo; Karyl Barron; Hanna Kim; Simona Nativ; Geraldina Lionetti; Andrew Zeft; Donald Goldsmith; David Kimberlin; Kathryn Edwards; Fatma Dedeoglu; Sivia Lapidus
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2.  Overlap of familial Mediterranean fever and hyper-IgD syndrome in an Arabic kindred.

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4.  Systematic literature review of efficacy/effectiveness and safety of current therapies for the treatment of cryopyrin-associated periodic syndrome, hyperimmunoglobulin D syndrome and tumour necrosis factor receptor-associated periodic syndrome.

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6.  Reasons for canakinumab initiation among patients with periodic fever syndromes: a retrospective medical chart review from the United States.

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7.  Colchicine - an effective treatment for children with a clinical diagnosis of autoinflammatory diseases without pathogenic gene variants.

Authors:  Susanne M Benseler; Jasmin B Kuemmerle-Deschner; Tatjana Welzel; Anna L Wildermuth; Norbert Deschner
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  7 in total

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