Literature DB >> 15517377

Brain-Thyroid-Lung syndrome: a patient with a severe multi-system disorder due to a de novo mutation in the thyroid transcription factor 1 gene.

Michel A A P Willemsen1, Guido J Breedveld, Siep Wouda, Barto J Otten, Jan L Yntema, Martin Lammens, Bert B A de Vries.   

Abstract

UNLABELLED: A 23-year-old man was diagnosed with pulmonary alveolar proteinosis at the age of 11 months, and primary hypothyroidism gradually developed during infancy. He had delayed developmental milestones and severe hypotonia that evolved into non-progressive chorea during childhood. He died from large cell lung carcinoma at the age of 23 years. A de novo heterozygous insertion mutation 859-860insC in the TITF-1 gene was demonstrated.
CONCLUSION: TITF-1 gene mutations should be considered in paediatric and adult patients with unexplained (combinations of) chorea, mental retardation, primary hypothyroidism, and chronic lung disease. Introduction of a name for the disorder, e.g. Brain-Thyroid-Lung syndrome, would probably facilitate further recognition. Whether the TITF-1 gene mutation in this patient predisposed to the development of lung cancer remains speculative.

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Year:  2004        PMID: 15517377     DOI: 10.1007/s00431-004-1559-x

Source DB:  PubMed          Journal:  Eur J Pediatr        ISSN: 0340-6199            Impact factor:   3.183


  13 in total

Review 1.  Pulmonary alveolar proteinosis.

Authors:  Bruce C Trapnell; Jeffrey A Whitsett; Koh Nakata
Journal:  N Engl J Med       Date:  2003-12-25       Impact factor: 91.245

2.  Deletion of thyroid transcription factor-1 gene in an infant with neonatal thyroid dysfunction and respiratory failure.

Authors:  K Devriendt; C Vanhole; G Matthijs; F de Zegher
Journal:  N Engl J Med       Date:  1998-04-30       Impact factor: 91.245

3.  Intramolecular control of transcriptional activity by the NK2-specific domain in NK-2 homeodomain proteins.

Authors:  H Watada; R G Mirmira; J Kalamaras; M S German
Journal:  Proc Natl Acad Sci U S A       Date:  2000-08-15       Impact factor: 11.205

4.  Choreoathetosis, hypothyroidism, and pulmonary alterations due to human NKX2-1 haploinsufficiency.

Authors:  Heiko Krude; Barbara Schütz; Heike Biebermann; Arpad von Moers; Dirk Schnabel; Heidi Neitzel; Holger Tönnies; Dagmar Weise; Antony Lafferty; Siegfried Schwarz; Mario DeFelice; Andreas von Deimling; Frank van Landeghem; Roberto DiLauro; Annette Grüters
Journal:  J Clin Invest       Date:  2002-02       Impact factor: 14.808

5.  Mutations in TITF-1 are associated with benign hereditary chorea.

Authors:  Guido J Breedveld; Jeroen W F van Dongen; Cesare Danesino; Andrea Guala; Alan K Percy; Leon S Dure; Peter Harper; Lazarus P Lazarou; Herma van der Linde; Marijke Joosse; Annette Grüters; Marcy E MacDonald; Bert B A de Vries; Willem Frans M Arts; Ben A Oostra; Heiko Krude; Peter Heutink
Journal:  Hum Mol Genet       Date:  2002-04-15       Impact factor: 6.150

6.  Nkx2.1 transcription factor in lung cells and a transforming growth factor-beta1 heterozygous mouse model of lung carcinogenesis.

Authors:  Yang Kang; Haroun Hebron; Laurent Ozbun; Jennifer Mariano; Parviz Minoo; Sonia B Jakowlew
Journal:  Mol Carcinog       Date:  2004-08       Impact factor: 4.784

7.  Autosomal dominant transmission of congenital hypothyroidism, neonatal respiratory distress, and ataxia caused by a mutation of NKX2-1.

Authors:  Daniel A Doyle; Iris Gonzalez; Becky Thomas; Mena Scavina
Journal:  J Pediatr       Date:  2004-08       Impact factor: 4.406

Review 8.  Pulmonary alveolar proteinosis: progress in the first 44 years.

Authors:  John F Seymour; Jeffrey J Presneill
Journal:  Am J Respir Crit Care Med       Date:  2002-07-15       Impact factor: 21.405

Review 9.  Transcriptional regulation of lung development: emergence of specificity.

Authors:  P Minoo
Journal:  Respir Res       Date:  2000-09-01

10.  The transcription factor TTF-1 is expressed at the onset of thyroid and lung morphogenesis and in restricted regions of the foetal brain.

Authors:  D Lazzaro; M Price; M de Felice; R Di Lauro
Journal:  Development       Date:  1991-12       Impact factor: 6.868

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  29 in total

Review 1.  The molecular causes of thyroid dysgenesis: a systematic review.

Authors:  I C Nettore; V Cacace; C De Fusco; A Colao; P E Macchia
Journal:  J Endocrinol Invest       Date:  2013-05-22       Impact factor: 4.256

Review 2.  Surfactant dysfunction.

Authors:  W Adam Gower; Lawrence M Nogee
Journal:  Paediatr Respir Rev       Date:  2011-03-05       Impact factor: 2.726

3.  Heterogeneous pulmonary phenotypes associated with mutations in the thyroid transcription factor gene NKX2-1.

Authors:  Aaron Hamvas; Robin R Deterding; Susan E Wert; Frances V White; Megan K Dishop; Danielle N Alfano; Ann C Halbower; Benjamin Planer; Mark J Stephan; Derek A Uchida; Lee D Williames; Jill A Rosenfeld; Robert Roger Lebel; Lisa R Young; F Sessions Cole; Lawrence M Nogee
Journal:  Chest       Date:  2013-09       Impact factor: 9.410

Review 4.  Defects of Thyroid Hormone Synthesis and Action.

Authors:  Zeina C Hannoush; Roy E Weiss
Journal:  Endocrinol Metab Clin North Am       Date:  2017-03-06       Impact factor: 4.741

5.  Benign hereditary chorea: dopaminergic brain imaging in patients with a novel intronic NKX2.1 gene mutation.

Authors:  Takashi Konishi; Satoshi Kono; Masaya Fujimoto; Tatsuhiro Terada; Kozo Matsushita; Yasuomi Ouchi; Hiroaki Miyajima
Journal:  J Neurol       Date:  2012-07-24       Impact factor: 4.849

6.  Pulmonary pathology in thyroid transcription factor-1 deficiency syndrome.

Authors:  Csaba Galambos; Hara Levy; Carolyn L Cannon; Sara O Vargas; Lynne M Reid; Robert Cleveland; Robert Lindeman; Daphne E deMello; Susan E Wert; Jeffrey A Whitsett; Antonio R Perez-Atayde; Harry Kozakewich
Journal:  Am J Respir Crit Care Med       Date:  2010-03-04       Impact factor: 21.405

7.  Comprehensive genotyping and clinical characterisation reveal 27 novel NKX2-1 mutations and expand the phenotypic spectrum.

Authors:  Anne Thorwarth; Sarah Schnittert-Hübener; Pamela Schrumpf; Ines Müller; Sabine Jyrch; Christof Dame; Heike Biebermann; Gunnar Kleinau; Juri Katchanov; Markus Schuelke; Grit Ebert; Anne Steininger; Carsten Bönnemann; Knut Brockmann; Hans-Jürgen Christen; Patricia Crock; Francis deZegher; Matthias Griese; Jacqueline Hewitt; Sten Ivarsson; Christoph Hübner; Klaus Kapelari; Barbara Plecko; Dietz Rating; Iva Stoeva; Hans-Hilger Ropers; Annette Grüters; Reinhard Ullmann; Heiko Krude
Journal:  J Med Genet       Date:  2014-04-08       Impact factor: 6.318

Review 8.  An official American Thoracic Society clinical practice guideline: classification, evaluation, and management of childhood interstitial lung disease in infancy.

Authors:  Geoffrey Kurland; Robin R Deterding; James S Hagood; Lisa R Young; Alan S Brody; Robert G Castile; Sharon Dell; Leland L Fan; Aaron Hamvas; Bettina C Hilman; Claire Langston; Lawrence M Nogee; Gregory J Redding
Journal:  Am J Respir Crit Care Med       Date:  2013-08-01       Impact factor: 21.405

Review 9.  Interstitial lung diseases in children.

Authors:  Annick Clement; Nadia Nathan; Ralph Epaud; Brigitte Fauroux; Harriet Corvol
Journal:  Orphanet J Rare Dis       Date:  2010-08-20       Impact factor: 4.123

10.  Mutational analysis of thyroid transcription factor-1 gene (TTF-1) in lung carcinomas.

Authors:  Xiao Yan Bai; Hong Shen
Journal:  In Vitro Cell Dev Biol Anim       Date:  2007-12-11       Impact factor: 2.416

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