Literature DB >> 8490057

Biochemical diagnosis of a fatal case of Günther's disease in a newborn with hydrops foetalis.

L Verstraeten1, N Van Regemorter, A Pardou, H de Verneuil, V Da Silva, F Rodesch, D Vermeylen, C Donner, J C Noël, Y Nordmann.   

Abstract

The birth of a male baby was induced at 32 weeks. In utero, the child presented, inter alia, signs of hydrops, hepatosplenomegaly and anaemia. Two in utero transfusions for correction of the anaemia were performed at 28 and 29 weeks, respectively. The baby rapidly presented respiratory distress with mixed acidosis. Three hours after birth, pink urine was excreted. Signs of icterus necessitated phototherapy, after which photosensitivity occurred. Erythrocytes were fluorescent under long-wavelength UV light. The baby died 24 hours after birth, displaying severe acidosis, a diffuse haemorrhagic syndrome, and repeated brady-cardia which did not respond to isoprenaline. The analysis of porphyrins in urine, blood and faeces of the baby gave the following results: 1) uroporphyrin (I and III isomeric series) was increased in urine and faeces, with traces in erythrocytes and plasma; 2) heptacarboxyporphyrin I was found mainly in urine and much less in erythrocytes, plasma and faeces; 3) coproporphyrin I was increased in urine, erythrocytes, plasma and faeces, and 4) 5-aminolaevulinic acid and porphobilinogen in urine and plasma were within the reference ranges. Determination of the enzymes of haem biosynthesis in erythrocytes and lymphocytes showed that both parents possessed only 50% of the normal activity of cosynthase. A previously described point mutation in codon 73 was observed in one parent. Fatal cases of neonatal Günther's disease are extremely rare and such an observation, according to our knowledge, is probably one of the first described.

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Year:  1993        PMID: 8490057     DOI: 10.1515/cclm.1993.31.3.121

Source DB:  PubMed          Journal:  Eur J Clin Chem Clin Biochem        ISSN: 0939-4974


  9 in total

1.  A novel point mutation in congenital erythropoietic porphyria in two members of Japanese family.

Authors:  K Tanigawa; M Bensidhoum; N Takamura; H Namba; S Yamashita; H de Verneuil; C Ged
Journal:  Hum Genet       Date:  1996-05       Impact factor: 4.132

2.  Gene transfer of the uroporphyrinogen III synthase cDNA into haematopoietic progenitor cells in view of a future gene therapy in congenital erythropoietic porphyria.

Authors:  F Mazurier; F Moreau-Gaudry; S Salesse; C Barbot; C Ged; J Reiffers; H de Verneuil
Journal:  J Inherit Metab Dis       Date:  1997-06       Impact factor: 4.982

3.  Uroporphyrinogen III synthase erythroid promoter mutations in adjacent GATA1 and CP2 elements cause congenital erythropoietic porphyria.

Authors:  C Solis; G I Aizencang; K H Astrin; D F Bishop; R J Desnick
Journal:  J Clin Invest       Date:  2001-03       Impact factor: 14.808

Review 4.  Porphyria Diagnostics-Part 1: A Brief Overview of the Porphyrias.

Authors:  Vaithamanithi-Mudumbai Sadagopa Ramanujam; Karl Elmo Anderson
Journal:  Curr Protoc Hum Genet       Date:  2015-07-01

Review 5.  Porphyrias: animal models and prospects for cellular and gene therapy.

Authors:  H de Verneuil; C Ged; S Boulechfar; F Moreau-Gaudry
Journal:  J Bioenerg Biomembr       Date:  1995-04       Impact factor: 2.945

Review 6.  Diagnosis of inherited disorders of liver metabolism.

Authors:  P T Clayton
Journal:  J Inherit Metab Dis       Date:  2003       Impact factor: 4.982

Review 7.  Congenital erythropoietic porphyria: Recent advances.

Authors:  Angelika L Erwin; Robert J Desnick
Journal:  Mol Genet Metab       Date:  2018-12-27       Impact factor: 4.797

8.  Congenital erythropoietic porphyria: identification and expression of 10 mutations in the uroporphyrinogen III synthase gene.

Authors:  W Xu; C A Warner; R J Desnick
Journal:  J Clin Invest       Date:  1995-02       Impact factor: 14.808

9.  An Atypical Case of Congenital Erythropoietic Porphyria.

Authors:  Bénédicte Sudrié-Arnaud; Marine Legendre; Sarah Snanoudj; Fanny Pelluard; Soumeya Bekri; Abdellah Tebani
Journal:  Genes (Basel)       Date:  2021-11-19       Impact factor: 4.096

  9 in total

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