Literature DB >> 28105570

Primary Carnitine Deficiency: Is Foetal Development Affected and Can Newborn Screening Be Improved?

Jan Rasmussen1, David M Hougaard2, Noreen Sandhu2, Katrine Fjællegaard3, Poula R Petersen3, Ulrike Steuerwald4,5, Allan M Lund6.   

Abstract

Primary carnitine deficiency (PCD) causes low levels of carnitine in patients potentially leading to metabolic and cardiac symptoms. Newborn screening for PCD is now routine in many countries by measuring carnitine levels in infants. In this study we report Apgar scores, length and weight in newborns with PCD and newborns born to mothers with PCD compared to controls. Furthermore we report how effective different screening algorithms have been to detect newborns with PCD in the Faroe Islands.
RESULTS: Newborns with PCD and newborns born to mothers with PCD did not differ with regard to Apgar scores, length and weight compared to controls. Newborns with PCD and newborns born to mothers with PCD had significantly lower levels of free carnitine (fC0) than controls. Screening algorithms focusing only on fC0 had a high rate of detection of newborns with PCD. Sample collection 4-9 days after birth seems to result in a higher detection rate than the current 2-3 days.
CONCLUSION: The clinical status at birth in infants with PCD and infants born to mothers with PCD does not differ compared to control infants. Screening algorithms for PCD should focus on fC0, and blood samples should be taken when the maternal influence on fC0 has diminished.

Entities:  

Keywords:  Apgar; Faroe Islands; Newborn screening; Primary carnitine deficiency

Year:  2017        PMID: 28105570      PMCID: PMC5680280          DOI: 10.1007/8904_2016_30

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


  25 in total

1.  Carnitine levels in 26,462 individuals from the nationwide screening program for primary carnitine deficiency in the Faroe Islands.

Authors:  Jan Rasmussen; Olav W Nielsen; Nils Janzen; Morten Duno; Hannes Gislason; Lars Køber; Ulrike Steuerwald; Allan M Lund
Journal:  J Inherit Metab Dis       Date:  2013-05-08       Impact factor: 4.982

Review 2.  Carnitine deficiency disorders in children.

Authors:  Charles A Stanley
Journal:  Ann N Y Acad Sci       Date:  2004-11       Impact factor: 5.691

3.  The Apgar score has survived the test of time.

Authors:  Mieczyslaw Finster; Margaret Wood
Journal:  Anesthesiology       Date:  2005-04       Impact factor: 7.892

Review 4.  Disorders of carnitine transport and the carnitine cycle.

Authors:  Nicola Longo; Cristina Amat di San Filippo; Marzia Pasquali
Journal:  Am J Med Genet C Semin Med Genet       Date:  2006-05-15       Impact factor: 3.908

Review 5.  Carnitine transport: pathophysiology and metabolism of known molecular defects.

Authors:  I Tein
Journal:  J Inherit Metab Dis       Date:  2003       Impact factor: 4.982

6.  Screening newborns for inborn errors of metabolism by tandem mass spectrometry.

Authors:  Bridget Wilcken; Veronica Wiley; Judith Hammond; Kevin Carpenter
Journal:  N Engl J Med       Date:  2003-06-05       Impact factor: 91.245

7.  Storage policies and use of the Danish Newborn Screening Biobank.

Authors:  B Nørgaard-Pedersen; D M Hougaard
Journal:  J Inherit Metab Dis       Date:  2007-07-12       Impact factor: 4.982

8.  Primary carnitine deficiency and pivalic acid exposure causing encephalopathy and fatal cardiac events.

Authors:  Jan Rasmussen; Olav W Nielsen; Allan M Lund; Lars Køber; Høgni Djurhuus
Journal:  J Inherit Metab Dis       Date:  2012-05-08       Impact factor: 4.982

9.  Biochemical screening of 504,049 newborns in Denmark, the Faroe Islands and Greenland--experience and development of a routine program for expanded newborn screening.

Authors:  Allan Meldgaard Lund; David Michael Hougaard; Henrik Simonsen; Brage Storstein Andresen; Mette Christensen; Morten Dunø; Kristin Skogstrand; Rikke K J Olsen; Ulrich Glümer Jensen; Arieh Cohen; Nanna Larsen; Peter Saugmann-Jensen; Niels Gregersen; Niels Jacob Brandt; Ernst Christensen; Flemming Skovby; Bent Nørgaard-Pedersen
Journal:  Mol Genet Metab       Date:  2012-06-21       Impact factor: 4.797

10.  Defective urinary carnitine transport in heterozygotes for primary carnitine deficiency.

Authors:  F Scaglia; Y Wang; R H Singh; P P Dembure; M Pasquali; P M Fernhoff; N Longo
Journal:  Genet Med       Date:  1998 Nov-Dec       Impact factor: 8.822

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  5 in total

1.  Functional and molecular studies in primary carnitine deficiency.

Authors:  Marta Frigeni; Bijina Balakrishnan; Xue Yin; Fernanda R O Calderon; Rong Mao; Marzia Pasquali; Nicola Longo
Journal:  Hum Mutat       Date:  2017-09-14       Impact factor: 4.878

Review 2.  Mass Spectrometric Analysis of L-carnitine and its Esters: Potential Biomarkers of Disturbances in Carnitine Homeostasis.

Authors:  Judit Bene; Andras Szabo; Katalin Komlósi; Bela Melegh
Journal:  Curr Mol Med       Date:  2020       Impact factor: 2.222

3.  Biochemical, Molecular, and Clinical Characterization of Patients With Primary Carnitine Deficiency via Large-Scale Newborn Screening in Xuzhou Area.

Authors:  Wei Zhou; Huizhong Li; Ting Huang; Yan Zhang; Chuanxia Wang; Maosheng Gu
Journal:  Front Pediatr       Date:  2019-02-26       Impact factor: 3.418

4.  Increased detection of primary carnitine deficiency through second-tier newborn genetic screening.

Authors:  Yiming Lin; Weifeng Zhang; Chenggang Huang; Chunmei Lin; Weihua Lin; Weilin Peng; Qingliu Fu; Dongmei Chen
Journal:  Orphanet J Rare Dis       Date:  2021-03-23       Impact factor: 4.123

5.  Newborn screening for carnitine transporter defect in Bavaria and the long-term follow-up of the identified newborns and mothers: Assessing the benefit and possible harm based on 19 ½ years of experience.

Authors:  Katharina A Schiergens; Katharina J Weiss; Wulf Röschinger; Amelie S Lotz-Havla; Joachim Schmitt; Robert Dalla Pozza; Sarah Ulrich; Birgit Odenwald; Joachim Kreuder; Esther M Maier
Journal:  Mol Genet Metab Rep       Date:  2021-06-12
  5 in total

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