Literature DB >> 36161784

Expert consensus on diagnosis and treatment of very long-chain acyl-CoA dehydrogenase deficiency.

Division of Biochemistry and Metabolism Medical Genetics Branch, Chinese Medical Association, Division of Genetics and Metabolism, Child Diseases and Health Care Branch Chinese Association for Maternal and Child Health.   

Abstract

Very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency is a metabolic disease of long chain fatty acid oxidation. The clinical manifestations are heterogeneous, mainly with heart, liver, skeletal muscle and brain damage, and the onset of which can be from newborn to adult. Cardiomyopathy type is more serious with high mortality. The liver failure type and myopathy type would be potentially lethal, but generally the prognosis is relatively good. Recurrent hypoglycemia, energy metabolism disorder, liver dysfunction, cardiomyopathy and serious arrhythmia are the main causes of death. Most patients can be identified through neonatal screening, and the prognosis is usually good in patients with early diagnosis and treatment. The purpose of this consensus is to standardize the diagnosis, treatment and management of VLCAD deficiency, so as to improve the prognosis of patients and reduce death and disability.

Entities:  

Keywords:  Autosomal recessive disease; Expert consensus; Fatty acid β-oxidation; Neonatal screening; Very long-chain acyl-CoA dehydrogenase deficiency

Mesh:

Substances:

Year:  2022        PMID: 36161784      PMCID: PMC9109756          DOI: 10.3724/zdxbyxb-2022-0107

Source DB:  PubMed          Journal:  Zhejiang Da Xue Xue Bao Yi Xue Ban        ISSN: 1008-9292


  30 in total

1.  Bezafibrate in skeletal muscle fatty acid oxidation disorders: a randomized clinical trial.

Authors:  Mette Cathrine Ørngreen; Karen Lindhardt Madsen; Nicolai Preisler; Grete Andersen; John Vissing; Pascal Laforêt
Journal:  Neurology       Date:  2014-01-22       Impact factor: 9.910

Review 2.  Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment--A retrospective chart review.

Authors:  Jerry Vockley; Deborah Marsden; Elizabeth McCracken; Stephanie DeWard; Amanda Barone; Kristen Hsu; Emil Kakkis
Journal:  Mol Genet Metab       Date:  2015-06-18       Impact factor: 4.797

3.  Infants suspected to have very-long chain acyl-CoA dehydrogenase deficiency from newborn screening.

Authors:  J Lawrence Merritt; Sverre Vedal; Jose E Abdenur; Sylvia M Au; Bruce A Barshop; Lisa Feuchtbaum; Cary O Harding; Cheryl Hermerath; Fred Lorey; David E Sesser; John D Thompson; Arthur Yu
Journal:  Mol Genet Metab       Date:  2014-01-23       Impact factor: 4.797

4.  Clinical and biochemical outcome of patients with very long-chain acyl-CoA dehydrogenase deficiency.

Authors:  Valentina Rovelli; Francesca Manzoni; Krista Viau; Marzia Pasquali; Nicola Longo
Journal:  Mol Genet Metab       Date:  2019-04-16       Impact factor: 4.797

5.  Triheptanoin versus trioctanoin for long-chain fatty acid oxidation disorders: a double blinded, randomized controlled trial.

Authors:  Melanie B Gillingham; Stephen B Heitner; Julie Martin; Sarah Rose; Amy Goldstein; Areeg Hassan El-Gharbawy; Stephanie Deward; Michael R Lasarev; Jim Pollaro; James P DeLany; Luke J Burchill; Bret Goodpaster; James Shoemaker; Dietrich Matern; Cary O Harding; Jerry Vockley
Journal:  J Inherit Metab Dis       Date:  2017-09-04       Impact factor: 4.982

6.  Development of a new enzymatic diagnosis method for very-long-chain Acyl-CoA dehydrogenase deficiency by detecting 2-hexadecenoyl-CoA production and its application in tandem mass spectrometry-based selective screening and newborn screening in Japan.

Authors:  Go Tajima; Nobuo Sakura; Kenichiro Shirao; Satoshi Okada; Miyuki Tsumura; Yutaka Nishimura; Hiroaki Ono; Yuki Hasegawa; Ikue Hata; Etsuo Naito; Seiji Yamaguchi; Yosuke Shigematsu; Masao Kobayashi
Journal:  Pediatr Res       Date:  2008-12       Impact factor: 3.756

7.  A novel disease with deficiency of mitochondrial very-long-chain acyl-CoA dehydrogenase.

Authors:  T Aoyama; Y Uchida; R I Kelley; M Marble; K Hofman; J H Tonsgard; W J Rhead; T Hashimoto
Journal:  Biochem Biophys Res Commun       Date:  1993-03-31       Impact factor: 3.575

8.  A Delphi clinical practice protocol for the management of very long chain acyl-CoA dehydrogenase deficiency.

Authors:  Georgianne L Arnold; Johan Van Hove; Debra Freedenberg; Arnold Strauss; Nicola Longo; Barbara Burton; Cheryl Garganta; Can Ficicioglu; Stephen Cederbaum; Cary Harding; Richard G Boles; Dietrich Matern; Pranesh Chakraborty; Annette Feigenbaum
Journal:  Mol Genet Metab       Date:  2009-01-20       Impact factor: 4.797

9.  Serum C14:1/C12:1 ratio is a useful marker for differentiating affected patients with very long-chain acyl-CoA dehydrogenase deficiency from heterozygous carriers.

Authors:  Kenji Yamada; Yoshimitsu Osawa; Hironori Kobayashi; Yuki Hasegawa; Seiji Fukuda; Seiji Yamaguchi; Takeshi Taketani
Journal:  Mol Genet Metab Rep       Date:  2019-11-05

10.  Open-label clinical trial of bezafibrate treatment in patients with fatty acid oxidation disorders in Japan.

Authors:  Kenji Yamada; Hideaki Shiraishi; Eishin Oki; Mika Ishige; Toshiyuki Fukao; Yusuke Hamada; Norio Sakai; Fumihiro Ochi; Asami Watanabe; Sanae Kawakami; Kazuyo Kuzume; Kenji Watanabe; Koji Sameshima; Kiyotaka Nakamagoe; Akira Tamaoka; Naoko Asahina; Saki Yokoshiki; Takashi Miyakoshi; Kota Ono; Koji Oba; Toshiyuki Isoe; Hiroshi Hayashi; Seiji Yamaguchi; Norihiro Sato
Journal:  Mol Genet Metab Rep       Date:  2018-02-22
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