Literature DB >> 16531551

Hearing improvement in a patient with variant Muckle-Wells syndrome in response to interleukin 1 receptor antagonism.

M Rynne1, C Maclean, A Bybee, M F McDermott, P Emery.   

Abstract

BACKGROUND: Muckle-Wells syndrome (MWS), familial cold autoinflammatory syndrome, and neonatal onset multisystem inflammatory disease, also called chronic, infantile, neurological, cutaneous, and articular syndrome, are three hereditary autoinflammatory syndromes caused by mutations affecting the CIAS1/NALP3 gene on chromosome 1q44. The proinflammatory cytokine, interleukin 1beta, is believed to have a fundamental role in their pathogenesis. CASE REPORT: The case is described of a 59 year old white woman who presented with increasingly severe MWS-type features over a 15 year period. The response to interleukin 1beta inhibition with anakinra was dramatic, including a reduction in intracranial pressure with associated auditory improvement, as demonstrated by serial audiometry.
CONCLUSIONS: The confirmed improvement in hearing after initiation of interleukin 1 receptor antagonism corroborates previous reports that specific blockade of this single cytokine reverses most of the symptoms of this group of CIAS1/NALP3 related autoinflammatory conditions, including the sensorineural deafness, which has not been previously reported.

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Year:  2006        PMID: 16531551      PMCID: PMC1798106          DOI: 10.1136/ard.2005.038091

Source DB:  PubMed          Journal:  Ann Rheum Dis        ISSN: 0003-4967            Impact factor:   19.103


  10 in total

1.  New mutations of CIAS1 that are responsible for Muckle-Wells syndrome and familial cold urticaria: a novel mutation underlies both syndromes.

Authors:  Catherine Dodé; Nathalie Le Dû; Laurence Cuisset; Frank Letourneur; Jean-Marie Berthelot; Gérard Vaudour; Alain Meyrier; Richard A Watts; David G I Scott; Anne Nicholls; Brigitte Granel; Camille Frances; François Garcier; Patrick Edery; Serge Boulinguez; Jean-Paul Domergues; Marc Delpech; Gilles Grateau
Journal:  Am J Hum Genet       Date:  2002-04-25       Impact factor: 11.025

2.  Interleukin-1-receptor antagonist in the Muckle-Wells syndrome.

Authors:  Philip N Hawkins; Helen J Lachmann; Michael F McDermott
Journal:  N Engl J Med       Date:  2003-06-19       Impact factor: 91.245

3.  Response to anakinra in a de novo case of neonatal-onset multisystem inflammatory disease.

Authors:  Philip N Hawkins; Alison Bybee; Ebun Aganna; Michael F McDermott
Journal:  Arthritis Rheum       Date:  2004-08

4.  Genomic-based therapy: targeting interleukin-1 for autoinflammatory diseases.

Authors:  Hal M Hoffman; Dhavalkumar D Patel
Journal:  Arthritis Rheum       Date:  2004-02

5.  Anakinra in mutation-negative NOMID/CINCA syndrome: comment on the articles by Hawkins et al and Hoffman and Patel.

Authors:  Joost Frenkel; Nico M Wulffraat; Wietse Kuis
Journal:  Arthritis Rheum       Date:  2004-11

6.  Molecular basis of the spectral expression of CIAS1 mutations associated with phagocytic cell-mediated autoinflammatory disorders CINCA/NOMID, MWS, and FCU.

Authors:  Bénédicte Neven; Isabelle Callebaut; Anne-Marie Prieur; Jérôme Feldmann; Christine Bodemer; Loredana Lepore; Beata Derfalvi; Suata Benjaponpitak; Richard Vesely; Marie Jose Sauvain; Stefan Oertle; Roger Allen; Gareth Morgan; Arndt Borkhardt; Clare Hill; Janet Gardner-Medwin; Alain Fischer; Geneviève de Saint Basile
Journal:  Blood       Date:  2003-11-20       Impact factor: 22.113

Review 7.  The expanding spectrum of systemic autoinflammatory disorders and their rheumatic manifestations.

Authors:  Keith M Hull; Nitza Shoham; Jae Jin Chae; Ivona Aksentijevich; Daniel L Kastner
Journal:  Curr Opin Rheumatol       Date:  2003-01       Impact factor: 5.006

8.  Spectrum of clinical features in Muckle-Wells syndrome and response to anakinra.

Authors:  Philip N Hawkins; Helen J Lachmann; Ebun Aganna; Michael F McDermott
Journal:  Arthritis Rheum       Date:  2004-02

9.  Association of mutations in the NALP3/CIAS1/PYPAF1 gene with a broad phenotype including recurrent fever, cold sensitivity, sensorineural deafness, and AA amyloidosis.

Authors:  Ebun Aganna; Fabio Martinon; Philip N Hawkins; John B Ross; Daniel C Swan; David R Booth; Helen J Lachmann; Alison Bybee; Roxanne Gaudet; Patricia Woo; Conleth Feighery; Finbarr E Cotter; Margot Thome; Graham A Hitman; Jürg Tschopp; Michael F McDermott
Journal:  Arthritis Rheum       Date:  2002-09

10.  Infevers: an evolving mutation database for auto-inflammatory syndromes.

Authors:  Isabelle Touitou; Suzanne Lesage; Michael McDermott; Laurence Cuisset; Hal Hoffman; Catherine Dode; Nitza Shoham; Ebun Aganna; Jean-Pierre Hugot; Carol Wise; Hans Waterham; Denis Pugnere; Jacques Demaille; Cyril Sarrauste de Menthiere
Journal:  Hum Mutat       Date:  2004-09       Impact factor: 4.878

  10 in total
  31 in total

Review 1.  Updated consensus statement on biological agents for the treatment of rheumatic diseases, 2007.

Authors:  D E Furst; F C Breedveld; J R Kalden; J S Smolen; G R Burmester; J Sieper; P Emery; E C Keystone; M H Schiff; P Mease; P L C M van Riel; R Fleischmann; M H Weisman; M E Weinblatt
Journal:  Ann Rheum Dis       Date:  2007-11       Impact factor: 19.103

Review 2.  Unleashing the therapeutic potential of NOD-like receptors.

Authors:  Kaoru Geddes; João G Magalhães; Stephen E Girardin
Journal:  Nat Rev Drug Discov       Date:  2009-06       Impact factor: 84.694

3.  The NOD2 defect in Blau syndrome does not result in excess interleukin-1 activity.

Authors:  Tammy M Martin; Zili Zhang; Paul Kurz; Carlos D Rosé; Hong Chen; Huiying Lu; Stephen R Planck; Michael P Davey; James T Rosenbaum
Journal:  Arthritis Rheum       Date:  2009-02

4.  [Recommendations on therapy using interleukin-1beta-blocking agents].

Authors:  B Manger; M Gaubitz; H Michels
Journal:  Z Rheumatol       Date:  2009-11       Impact factor: 1.372

Review 5.  Therapy of autoinflammatory syndromes.

Authors:  Hal M Hoffman
Journal:  J Allergy Clin Immunol       Date:  2009-12       Impact factor: 10.793

Review 6.  Interleukin-1 function and role in rheumatic disease.

Authors:  Georg Schett; Jean-Michel Dayer; Bernhard Manger
Journal:  Nat Rev Rheumatol       Date:  2015-12-10       Impact factor: 20.543

7.  Critical appraisal of canakinumab in the treatment of adults and children with cryopyrin-associated periodic syndrome (CAPS).

Authors:  Ori Toker; Philip J Hashkes
Journal:  Biologics       Date:  2010-05-25

8.  The evaluation of cochlear functions in Familial Mediterranean Fever.

Authors:  Mehmet Akif Eryilmaz; Abitter Yucel; Erkan Cure; Davut Sakiz; Ahmet Koder; Adem Kucuk; Recep Tunc
Journal:  Eur Arch Otorhinolaryngol       Date:  2016-04-29       Impact factor: 2.503

Review 9.  NOD-like receptors and inflammation.

Authors:  Rebeccah J Mathews; Michael B Sprakes; Michael F McDermott
Journal:  Arthritis Res Ther       Date:  2008-11-25       Impact factor: 5.156

10.  Long term management of patients with cryopyrin-associated periodic syndromes (CAPS): focus on rilonacept (IL-1 Trap).

Authors:  Leigh D Church; Sinisa Savic; Michael F McDermott
Journal:  Biologics       Date:  2008-12
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