Literature DB >> 17034542

Poikiloderma, tendon contracture and pulmonary fibrosis: a new autosomal dominant syndrome?

N P Khumalo1, K Pillay, P Beighton, H Wainwright, B Walker, N Saxe, B M Mayosi, E D Bateman.   

Abstract

Members of two generations of a South African family have a unique syndrome comprising poikiloderma, tendon contractures and progressive pulmonary fibrosis. The condition is clinically important as the skin changes, which involve the face, have considerable cosmetic impact, while lung involvement is potentially lethal in adulthood. Skin manifestations which facilitate diagnosis include facial telangiectasia, mottled hypo- and hyperpigmentation, papules and epidermal atrophy. The scalp, facial and body hair are fine and scanty. The tendon contractures lead to progressive digital flexion deformities and abnormalities of the ankles and feet, with disturbance of gait. Pulmonary involvement manifests as progressive dyspnoea. Pedigree data are compatible with an autosomal dominant mode of transmission. Poikiloderma of Weary is characterized by linear sclerotic and fibrous bands and not tendon contractures and is not associated with potentially lethal pulmonary fibrosis. Rather than name this disorder a variant of Weary syndrome, it might be prudent to use as an umbrella title one composed by Weary himself: 'hereditary sclerosing poikiloderma' (HSP), under which variants such as HSP Weary type, HSP with cardiac involvement (aortic stenosis described as inconsistently associated with Weary syndrome) and HSP with tendon/pulmonary involvement (current family) may be classified. The manifestations in this family differ from other poikilodermata and, to the best of our knowledge, have not been previously documented.

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Year:  2006        PMID: 17034542     DOI: 10.1111/j.1365-2133.2006.07473.x

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


  9 in total

1.  CUGC for hereditary fibrosing poikiloderma with tendon contractures, myopathy, and pulmonary fibrosis (POIKTMP).

Authors:  Sébastien Küry; Sandra Mercier; Gasnat Shaboodien; Thomas Besnard; Sébastien Barbarot; Nonhlanhla P Khumalo; Bongani M Mayosi; Stéphane Bézieau
Journal:  Eur J Hum Genet       Date:  2015-10-07       Impact factor: 4.246

2.  Hereditary sclerosing poikiloderma.

Authors:  Hyo Jin Lee; Dong Hoon Shin; Jong Soo Choi; Ki Hong Kim
Journal:  J Korean Med Sci       Date:  2012-01-27       Impact factor: 2.153

3.  Case Report: Hereditary Fibrosing Poikiloderma With Tendon Contractures, Myopathy, and Pulmonary Fibrosis (POIKTMP) Presenting With Liver Cirrhosis and Steroid-Responsive Interstitial Pneumonia.

Authors:  Michiko Takimoto-Sato; Toshinari Miyauchi; Masaru Suzuki; Hideyuki Ujiie; Toshifumi Nomura; Tomoo Ikari; Tomohiko Nakamura; Kei Takahashi; Machiko Matsumoto-Sasaki; Hirokazu Kimura; Hiroki Kimura; Yuichiro Matsui; Takashi Kitagataya; Ren Yamada; Kazuharu Suzuki; Akihisa Nakamura; Masato Nakai; Takuya Sho; Koji Ogawa; Naoya Sakamoto; Naoko Yamaguchi; Noriyuki Otsuka; Utano Tomaru; Satoshi Konno
Journal:  Front Genet       Date:  2022-05-05       Impact factor: 4.772

4.  Expanding the clinical spectrum of hereditary fibrosing poikiloderma with tendon contractures, myopathy and pulmonary fibrosis due to FAM111B mutations.

Authors:  Sandra Mercier; Sébastien Küry; Emmanuelle Salort-Campana; Armelle Magot; Uchenna Agbim; Thomas Besnard; Nathalie Bodak; Chantal Bou-Hanna; Flora Bréhéret; Perrine Brunelle; Florence Caillon; Brigitte Chabrol; Valérie Cormier-Daire; Albert David; Bruno Eymard; Laurence Faivre; Dominique Figarella-Branger; Emmanuelle Fleurence; Mythily Ganapathi; Romain Gherardi; Alice Goldenberg; Antoine Hamel; Jeanine Igual; Alan D Irvine; Dominique Israël-Biet; Caroline Kannengiesser; Christian Laboisse; Cédric Le Caignec; Jean-Yves Mahé; Stéphanie Mallet; Stuart MacGowan; Maeve A McAleer; Irwin McLean; Cécile Méni; Arnold Munnich; Jean-Marie Mussini; Peter L Nagy; Jeffrey Odel; Grainne M O'Regan; Yann Péréon; Julie Perrier; Juliette Piard; Eve Puzenat; Jacinda B Sampson; Frances Smith; Nadem Soufir; Kurenai Tanji; Christel Thauvin; Christina Ulane; Rosemarie M Watson; Nonhlanhla P Khumalo; Bongani M Mayosi; Sébastien Barbarot; Stéphane Bézieau
Journal:  Orphanet J Rare Dis       Date:  2015-10-15       Impact factor: 4.123

5.  Expanding phenotype of hereditary fibrosing poikiloderma with tendon contractures, myopathy, and pulmonary fibrosis caused by FAM111B mutations: Report of an additional family raising the question of cancer predisposition and a short review of early-onset poikiloderma.

Authors:  Raphaëlle Goussot; Megana Prasad; Corinne Stoetzel; Cédric Lenormand; Hélène Dollfus; Dan Lipsker
Journal:  JAAD Case Rep       Date:  2017-03-19

Review 6.  Mutations within the putative protease domain of the human FAM111B gene may predict disease severity and poor prognosis: A review of POIKTMP cases.

Authors:  Afolake Arowolo; Cenza Rhoda; Nonhlanhla Khumalo
Journal:  Exp Dermatol       Date:  2022-02-13       Impact factor: 4.511

Review 7.  Expanding phenotype of FAM111B-related disease focusing on liver involvement: Literature review, report of a case with end-stage liver disease and proposal for a new acronym.

Authors:  Marina Macchiaiolo; Filippo M Panfili; Davide Vecchio; Fabiana Cortellessa; Michaela V Gonfiantini; Paola S Buonuomo; Andrea Pietrobattista; Paola Francalanci; Lorena Travaglini; Enrico S Bertini; Maya El Hachem; Andrea Bartuli
Journal:  Am J Med Genet A       Date:  2022-07-23       Impact factor: 2.578

8.  Mutations in FAM111B cause hereditary fibrosing poikiloderma with tendon contracture, myopathy, and pulmonary fibrosis.

Authors:  Sandra Mercier; Sébastien Küry; Gasnat Shaboodien; Darren T Houniet; Nonhlanhla P Khumalo; Chantal Bou-Hanna; Nathalie Bodak; Valérie Cormier-Daire; Albert David; Laurence Faivre; Dominique Figarella-Branger; Romain K Gherardi; Elise Glen; Antoine Hamel; Christian Laboisse; Cédric Le Caignec; Pierre Lindenbaum; Armelle Magot; Arnold Munnich; Jean-Marie Mussini; Komala Pillay; Thahira Rahman; Richard Redon; Emmanuelle Salort-Campana; Mauro Santibanez-Koref; Christel Thauvin; Sébastien Barbarot; Bernard Keavney; Stéphane Bézieau; Bongani M Mayosi
Journal:  Am J Hum Genet       Date:  2013-11-21       Impact factor: 11.025

9.  FAM111 protease activity undermines cellular fitness and is amplified by gain-of-function mutations in human disease.

Authors:  Saskia Hoffmann; Satyakrishna Pentakota; Andreas Mund; Peter Haahr; Fabian Coscia; Marta Gallo; Matthias Mann; Nicholas Mi Taylor; Niels Mailand
Journal:  EMBO Rep       Date:  2020-08-09       Impact factor: 8.807

  9 in total

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