Literature DB >> 29292490

Two Uneventful Pregnancies in a Woman with Glutaric Aciduria Type 1.

Karolina M Stepien1, Gregory M Pastores2,3, Una Hendroff2, Ciara McCormick4, Patricia Fitzimons5, Naveed Khawaja4, Ingrid Borovickova5, Eileen P Treacy2,3.   

Abstract

Glutaric aciduria type 1 (GA1) is an autosomal recessive rare disorder caused by mutations in the GCDH gene resulting in deficiency of glutaryl-CoA dehydrogenase, leading to accumulation of the amino acids lysine, hydroxylysine and tryptophan and other metabolites. The phenotypic spectrum of disease is broad. Stress caused by infection and fever and possibly pregnancy may lead to worsening of the signs and symptoms, often with uncertain recovery.We describe a case of a female patient with GA1 who had two clinically uneventful pregnancies.At the age of 11 she was diagnosed with GA1 by family screening. The cultured skin fibroblast showed reduced glutaryl-CoA dehydrogenase activity (0.16 mg protein per min).The initial diagnostic urine glutaric acid level for this patient was 1,784 μmol/mmol creatinine. Mutation analysis showed compound heterozygosity for the p.(Gly185Arg), c.553G>A in exon 7 and p.(Arg402Trp), c.1204C.T in exon 11 mutations of the GCDH.Her pregnancy at the age of 23 was complicated by pre-eclampsia and required treatment with beta-blockers. Four years later the second pregnancy was uncomplicated. The management plan during the caesarean section included intravenous dextrose and lipid infusions. The patient rapidly recovered from both surgeries.Both babies have had normal development to date. On newborn screening, plasma acylcarnitine showed a transient increase in glutarylcarnitine, and the urine organic acid analysis showed a trace of 3-hydroxyglutarylcarnitine, likely to be of maternal transfer.The multidisciplinary team, consisting of metabolic, dietetic and obstetric care providers, have responsibility to ensure the risk of acute decompensation in pregnant GA1 women is minimal.

Entities:  

Keywords:  Glutaric aciduria type 1; Maternal transfer; Pregnancy

Year:  2018        PMID: 29292490      PMCID: PMC6122047          DOI: 10.1007/8904_2017_81

Source DB:  PubMed          Journal:  JIMD Rep        ISSN: 2192-8304


  31 in total

1.  Clinical and mutational spectra of 23 Chinese patients with glutaric aciduria type 1.

Authors:  Qiao Wang; Xiyuan Li; Yuan Ding; Yupeng Liu; Jinqing Song; Yanling Yang
Journal:  Brain Dev       Date:  2013-12-09       Impact factor: 1.961

2.  Natural history, outcome, and treatment efficacy in children and adults with glutaryl-CoA dehydrogenase deficiency.

Authors:  Stefan Kölker; Sven F Garbade; Cheryl R Greenberg; James V Leonard; Jean-Marie Saudubray; Antonia Ribes; H Serap Kalkanoglu; Allan M Lund; Begoña Merinero; Moacir Wajner; Mónica Troncoso; Monique Williams; John H Walter; Jaume Campistol; Milagros Martí-Herrero; Melissa Caswill; Alberto B Burlina; Florian Lagler; Esther M Maier; Bernd Schwahn; Aysegul Tokatli; Ali Dursun; Turgay Coskun; Ronald A Chalmers; David M Koeller; Johannes Zschocke; Ernst Christensen; Peter Burgard; Georg F Hoffmann
Journal:  Pediatr Res       Date:  2006-04-26       Impact factor: 3.756

Review 3.  Proposed recommendations for diagnosing and managing individuals with glutaric aciduria type I: second revision.

Authors:  Nikolas Boy; Chris Mühlhausen; Esther M Maier; Jana Heringer; Birgit Assmann; Peter Burgard; Marjorie Dixon; Sandra Fleissner; Cheryl R Greenberg; Inga Harting; Georg F Hoffmann; Daniela Karall; David M Koeller; Michael B Krawinkel; Jürgen G Okun; Thomas Opladen; Roland Posset; Katja Sahm; Johannes Zschocke; Stefan Kölker
Journal:  J Inherit Metab Dis       Date:  2016-11-16       Impact factor: 4.982

4.  Correlation of genotype and phenotype in glutaryl-CoA dehydrogenase deficiency.

Authors:  E Christensen; A Ribes; B Merinero; J Zschocke
Journal:  J Inherit Metab Dis       Date:  2004       Impact factor: 4.982

Review 5.  Biological roles of L-carnitine in perinatal metabolism.

Authors:  J Arenas; J C Rubio; M A Martín; Y Campos
Journal:  Early Hum Dev       Date:  1998-12       Impact factor: 2.079

6.  Mutation analysis in glutaric aciduria type I.

Authors:  J Zschocke; E Quak; P Guldberg; G F Hoffmann
Journal:  J Med Genet       Date:  2000-03       Impact factor: 6.318

7.  Maternal glutaric acidemia, type I identified by newborn screening.

Authors:  Eric A Crombez; Stephen D Cederbaum; Elaine Spector; Erica Chan; Denise Salazar; Julie Neidich; Stephen Goodman
Journal:  Mol Genet Metab       Date:  2008-03-04       Impact factor: 4.797

8.  Carnitine deficiency presenting as familial cardiomyopathy: a treatable defect in carnitine transport.

Authors:  L J Waber; D Valle; C Neill; S DiMauro; A Shug
Journal:  J Pediatr       Date:  1982-11       Impact factor: 4.406

Review 9.  Diagnosis and management of glutaric aciduria type I--revised recommendations.

Authors:  Stefan Kölker; Ernst Christensen; James V Leonard; Cheryl R Greenberg; Avihu Boneh; Alberto B Burlina; Alessandro P Burlina; Marjorie Dixon; Marinus Duran; Angels García Cazorla; Stephen I Goodman; David M Koeller; Mårten Kyllerman; Chris Mühlhausen; Edith Müller; Jürgen G Okun; Bridget Wilcken; Georg F Hoffmann; Peter Burgard
Journal:  J Inherit Metab Dis       Date:  2011-03-23       Impact factor: 4.982

10.  Screening of a healthy newborn identifies three adult family members with symptomatic glutaric aciduria type I.

Authors:  McH Janssen; Laj Kluijtmans; S B Wortmann
Journal:  BBA Clin       Date:  2014-06-02
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  2 in total

Review 1.  Impact of pregnancy on inborn errors of metabolism.

Authors:  Gisela Wilcox
Journal:  Rev Endocr Metab Disord       Date:  2018-03       Impact factor: 6.514

2.  Metabolic Serendipities of Expanded Newborn Screening.

Authors:  Raquel Yahyaoui; Javier Blasco-Alonso; Montserrat Gonzalo-Marín; Carmen Benito; Juliana Serrano-Nieto; Inmaculada González-Gallego; Pedro Ruiz-Sala; Belén Pérez; Domingo González-Lamuño
Journal:  Genes (Basel)       Date:  2020-08-29       Impact factor: 4.096

  2 in total

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