Literature DB >> 22847544

Autosomal-dominant chronic mucocutaneous candidiasis with STAT1-mutation can be complicated with chronic active hepatitis and hypothyroidism.

Tomohiro Hori1, Hidenori Ohnishi, Takahide Teramoto, Kohji Tsubouchi, Takafumi Naiki, Yoshinobu Hirose, Osamu Ohara, Mariko Seishima, Hideo Kaneko, Toshiyuki Fukao, Naomi Kondo.   

Abstract

PURPOSE: To describe a case of autosomal-dominant (AD)-chronic mucocutaneous candidiasis (CMC) with a signal transducer and activator of transcription (STAT) 1 gene mutation, and some of the important complications of this disease such as chronic hepatitis.
METHODS: We present a 23-year-old woman with CMC, chronic active hepatitis, and hypothyroidism. Her father also had CMC. We performed several immunological analyses of blood and liver samples, and searched for gene mutations for CMC in the patient and her father.
RESULTS: We identified the heterozygous substitution c.821 G > A (p.Arg274Gln) in the STAT1 gene of both the patient and her father. The level of β-glucan induced interferon (IFN)-γ in her blood cells was significantly low. Immunoblot analysis detected serum anti-interleukin (IL)-17 F autoantibody. She was found to have increased (low-titer) antibodies related to her hypothyroidism and hepatitis. Her serum IL-18 levels fluctuated with her AST and ALT levels. Liver biopsy revealed CD68-positive cell infiltration and IL-18 expression in the sinusoidal regions. These results suggest that the chronic active hepatitis in this patient may be exacerbated by the excessive IL-18 accumulation caused by recurrent mucocutaneous fungal infection, and decreased IFN-γ production.
CONCLUSIONS: AD-CMC is known to be caused by a gain-of-function mutation of the STAT1 gene. Chronic active hepatitis is a rare complication of AD-CMC, with currently unknown pathogenesis. It seems that the clinical phenotype in this patient is modified by autoimmune mechanisms and cytokine dysregulation. AD-CMC can be complicated by various immune disorders including autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy.

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Year:  2012        PMID: 22847544     DOI: 10.1007/s10875-012-9744-6

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


  23 in total

1.  Diagnosis and management of autoimmune hepatitis.

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Review 2.  IFNgamma signaling-does it mean JAK-STAT?

Authors:  Daniel J Gough; David E Levy; Ricky W Johnstone; Christopher J Clarke
Journal:  Cytokine Growth Factor Rev       Date:  2008-10-16       Impact factor: 7.638

Review 3.  Autoimmune polyendocrinopathy syndrome type 1 (APS1) and AIRE gene: new views on molecular basis of autoimmunity.

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Journal:  J Autoimmun       Date:  2005-11-14       Impact factor: 7.094

4.  International Autoimmune Hepatitis Group Report: review of criteria for diagnosis of autoimmune hepatitis.

Authors:  F Alvarez; P A Berg; F B Bianchi; L Bianchi; A K Burroughs; E L Cancado; R W Chapman; W G Cooksley; A J Czaja; V J Desmet; P T Donaldson; A L Eddleston; L Fainboim; J Heathcote; J C Homberg; J H Hoofnagle; S Kakumu; E L Krawitt; I R Mackay; R N MacSween; W C Maddrey; M P Manns; I G McFarlane; K H Meyer zum Büschenfelde; M Zeniya
Journal:  J Hepatol       Date:  1999-11       Impact factor: 25.083

5.  Chronic mucocutaneous candidiasis in humans with inborn errors of interleukin-17 immunity.

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Journal:  Science       Date:  2011-02-24       Impact factor: 47.728

6.  An autoimmune disease, APECED, caused by mutations in a novel gene featuring two PHD-type zinc-finger domains.

Authors: 
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Journal:  Nat Genet       Date:  1997-12       Impact factor: 38.330

8.  A homozygous CARD9 mutation in a family with susceptibility to fungal infections.

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9.  Disappearance of thyrotropin-blocking antibodies and spontaneous recovery from hypothyroidism in autoimmune thyroiditis.

Authors:  N Takasu; T Yamada; M Takasu; I Komiya; Y Nagasawa; T Asawa; T Shinoda; T Aizawa; Y Koizumi
Journal:  N Engl J Med       Date:  1992-02-20       Impact factor: 91.245

10.  Changes of TSH-Stimulation Blocking Antibody (TSBAb) and Thyroid Stimulating Antibody (TSAb) Over 10 Years in 34 TSBAb-Positive Patients with Hypothyroidism and in 98 TSAb-Positive Graves' Patients with Hyperthyroidism: Reevaluation of TSBAb and TSAb in TSH-Receptor-Antibody (TRAb)-Positive Patients.

Authors:  Nobuyuki Takasu; Mina Matsushita
Journal:  J Thyroid Res       Date:  2012-05-10
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  28 in total

1.  IL-17 T cells' defective differentiation in vitro despite normal range ex vivo in chronic mucocutaneous candidiasis due to STAT1 mutation.

Authors:  Najla Mekki; Imen Ben-Mustapha; Luyan Liu; Lobna Boussofara; Satoshi Okada; Sophie Cypowyj; Najet Ghariani; Wafa Saidi; Mohamed Denguezli; Jean-Laurent Casanova; Anne Puel; Mohamed-Ridha Barbouche
Journal:  J Invest Dermatol       Date:  2013-11-11       Impact factor: 8.551

Review 2.  A 1-year-old girl with a gain-of-function STAT1 mutation treated with hematopoietic stem cell transplantation.

Authors:  Juan Carlos Aldave; Enrique Cachay; Luis Núñez; Ausberto Chunga; Sergio Murillo; Sophie Cypowyj; Jacinta Bustamante; Anne Puel; Jean-Laurent Casanova; Armando Koo
Journal:  J Clin Immunol       Date:  2013-10-09       Impact factor: 8.317

Review 3.  Immunological loss-of-function due to genetic gain-of-function in humans: autosomal dominance of the third kind.

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Review 4.  IL-17-Mediated Immunity to the Opportunistic Fungal Pathogen Candida albicans.

Authors:  Heather R Conti; Sarah L Gaffen
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5.  Ruxolitinib reverses dysregulated T helper cell responses and controls autoimmunity caused by a novel signal transducer and activator of transcription 1 (STAT1) gain-of-function mutation.

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Journal:  J Allergy Clin Immunol       Date:  2017-01-27       Impact factor: 10.793

6.  Alanine-scanning mutagenesis of human signal transducer and activator of transcription 1 to estimate loss- or gain-of-function variants.

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Journal:  J Allergy Clin Immunol       Date:  2016-12-20       Impact factor: 10.793

Review 7.  Mendelian susceptibility to mycobacterial disease: genetic, immunological, and clinical features of inborn errors of IFN-γ immunity.

Authors:  Jacinta Bustamante; Stéphanie Boisson-Dupuis; Laurent Abel; Jean-Laurent Casanova
Journal:  Semin Immunol       Date:  2014-10-26       Impact factor: 11.130

8.  A STAT1-gain-of-function mutation causing Th17 deficiency with chronic mucocutaneous candidiasis, psoriasiform hyperkeratosis and dermatophytosis.

Authors:  Jakob Nielsen; Emil Kofod-Olsen; Eva Spaun; Carsten S Larsen; Mette Christiansen; Trine Hyrup Mogensen
Journal:  BMJ Case Rep       Date:  2015-10-22

Review 9.  Human STAT1 Gain-of-Function Heterozygous Mutations: Chronic Mucocutaneous Candidiasis and Type I Interferonopathy.

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Journal:  J Clin Immunol       Date:  2020-08-27       Impact factor: 8.317

10.  Simple diagnosis of STAT1 gain-of-function alleles in patients with chronic mucocutaneous candidiasis.

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Journal:  J Leukoc Biol       Date:  2013-12-16       Impact factor: 4.962

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