Literature DB >> 29932457

Epidermolysis bullosa simplex generalized severe induces a T helper 17 response and is improved by apremilast treatment.

E Castela1,2, M K Tulic3, A Rozières2, E Bourrat4, J-F Nicolas2,5, J Kanitakis6,7, P Vabres8, D Bessis9, J Mazereeuw10, F Morice-Picard11, D Baty12, F Berard5, J-P Lacour1,13, T Passeron1,3, C Chiaverini1,13.   

Abstract

BACKGROUND: Epidermolysis bullosa simplex generalized severe (EBS-gen sev) is a genetic disorder caused by mutation in the KRT5 or KRT14 genes. Although it is usually considered a mechanical disease, recent data argue for additional inflammatory mechanisms.
OBJECTIVES: To assess the inflammation in the skin of patients with EBS-gen sev.
METHODS: A first immunohistochemical retrospective study was performed on frozen skin samples from 17 patients with EBS-gen sev. A second multicentre prospective study was conducted on 10 patients with severe EBS-gen sev. Blister fluid and epidermis were processed for immunochemical analysis and quantitative real-time polymerase chain reaction. Cytokine expression was analysed in blister fluid and compared with that in controls.
RESULTS: Histological analysis showed a constant dermal perivascular CD4+ lymphocyte infiltrate in skin biopsies of both blister (n = 17) and rubbed skin (n = 5), an epidermal infiltration of neutrophils and eosinophils in 70% of cases, and increased immunostaining for CXCL9 and CXCL10 in blistering skin. High levels of T helper 17 cytokines were detected in lesional skin. Three adult patients with EBS-gen sev were treated with apremilast, with a dramatic improvement of skin blistering and good tolerance.
CONCLUSIONS: Our study demonstrates the importance of inflammation in patients with EBS-gen sev and underlines the key role for T helper 17 cells in its pathogenesis. In addition, this study provides promising new therapeutic approaches for this disabling disorder.
© 2018 British Association of Dermatologists.

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Year:  2018        PMID: 29932457     DOI: 10.1111/bjd.16897

Source DB:  PubMed          Journal:  Br J Dermatol        ISSN: 0007-0963            Impact factor:   9.302


  15 in total

1.  Off-label studies on apremilast in dermatology: a review.

Authors:  Nolan J Maloney; Jeffrey Zhao; Kyle Tegtmeyer; Ernest Y Lee; Kyle Cheng
Journal:  J Dermatolog Treat       Date:  2019-04-02       Impact factor: 3.359

Review 2.  Skin Fragility: Perspectives on Evidence-based Therapies.

Authors:  Leena Bruckner-Tuderman
Journal:  Acta Derm Venereol       Date:  2020-02-12       Impact factor: 3.875

Review 3.  Small molecule drug development for rare genodermatoses - evaluation of the current status in epidermolysis bullosa.

Authors:  Verena Wally; Manuela Reisenberger; Sophie Kitzmüller; Martin Laimer
Journal:  Orphanet J Rare Dis       Date:  2020-10-19       Impact factor: 4.123

4.  A Novel Mutation p.L461P in KRT5 Causing Localized Epidermolysis Bullosa Simplex.

Authors:  Xin Jiang; Yingyu Zhu; Huihui Sun; Feng Gu
Journal:  Ann Dermatol       Date:  2020-12-30       Impact factor: 1.444

Review 5.  Keratins as an Inflammation Trigger Point in Epidermolysis Bullosa Simplex.

Authors:  Nadezhda A Evtushenko; Arkadii K Beilin; Anastasiya V Kosykh; Ekaterina A Vorotelyak; Nadya G Gurskaya
Journal:  Int J Mol Sci       Date:  2021-11-18       Impact factor: 5.923

Review 6.  Investigational Treatments for Epidermolysis Bullosa.

Authors:  Ping-Chen Hou; Han-Tang Wang; Stasha Abhee; Wei-Ting Tu; John A McGrath; Chao-Kai Hsu
Journal:  Am J Clin Dermatol       Date:  2021-07-22       Impact factor: 7.403

7.  Epidermolysis bullosa simplex clearance after nasopharyngeal carcinoma treatment.

Authors:  Nour Marwan AlKhawajah; Marwan Mohammed AlKhawajah; Nouf Abdulaziz Aljomah; Abeer Adeeb Alabduljabbar; Salim Alkeraye
Journal:  JAAD Case Rep       Date:  2021-03-27

Review 8.  Challenges in Treating Genodermatoses: New Therapies at the Horizon.

Authors:  Marie-Anne Morren; Eric Legius; Fabienne Giuliano; Smail Hadj-Rabia; Daniel Hohl; Christine Bodemer
Journal:  Front Pharmacol       Date:  2022-01-05       Impact factor: 5.810

9.  Translational perspectives to treat Epidermolysis bullosa-Where do we stand?

Authors:  Christine Prodinger; Johann W Bauer; Martin Laimer
Journal:  Exp Dermatol       Date:  2020-11       Impact factor: 4.511

Review 10.  Clinical Perspectives of Gene-Targeted Therapies for Epidermolysis Bullosa.

Authors:  Tobias Welponer; Christine Prodinger; Josefina Pinon-Hofbauer; Arno Hintersteininger; Hannelore Breitenbach-Koller; Johann W Bauer; Martin Laimer
Journal:  Dermatol Ther (Heidelb)       Date:  2021-06-10
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