Document Detail


Co-occurrence and contribution of Fabry disease and Klippel-Trénaunay-Weber syndrome to a patient with atypical skin lesions.
MedLine Citation:
PMID:  11531972     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
Fabry disease (FD) is an X-linked recessive inborn error of glycosphingolipid metabolism. Among clinical symptoms, maculopapular skin lesions, known as angiokeratoma, most often appear on the lower abdomen, scrotum, and thighs, with a tendency toward bilateral symmetry. A 30-year-old male patient was referred to us for evaluation of a complex vascular and cutaneous malformation. Skin examination showed numerous angiokeratoma, which had developed only on the right part of the body, with a sharp delineation in the midline of the trunk. The diagnosis of FD was confirmed by demonstration of a decreased alpha-galactosidase A activity, and the patient was shown to be hemizygote for a missense mutation (R342Q) in the alpha-galactosidase A gene (GLA). This mutation was also demonstrated in DNA extracted from fibroblast cultures established from both affected and unaffected skin areas, thus excluding the hypothesis of somatic mosaicism or revertant mosaicism. Interestingly, the diagnosis of Klippel-Trénaunay-Weber syndrome (KTWS) was also made, through clinical and radiological investigations. This is the first report on the association between FD and KTWS. Karyotype analysis was normal. It is likely that the mixed vascular malformations of KTWS affecting capillary and venous systems have contributed to the unusual angiokeratoma distribution pattern observed in the patient.
Authors:
D P Germain
Publication Detail:
Type:  Case Reports; Journal Article    
Journal Detail:
Title:  Clinical genetics     Volume:  60     ISSN:  0009-9163     ISO Abbreviation:  Clin. Genet.     Publication Date:  2001 Jul 
Date Detail:
Created Date:  2001-09-04     Completed Date:  2001-09-27     Revised Date:  2004-11-17    
Medline Journal Info:
Nlm Unique ID:  0253664     Medline TA:  Clin Genet     Country:  Denmark    
Other Details:
Languages:  eng     Pagination:  63-7     Citation Subset:  IM    
Affiliation:
Department of Genetics, Hopital Europeen Georges Pompidou, 20, rue Leblanc, F-75015 Paris, France. dominique.germain@hop.egp.ap-hop-paris.fr
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MeSH Terms
Descriptor/Qualifier:
Adult
Base Sequence
Fabry Disease / complications,  genetics,  pathology*
Humans
Karyotyping
Klippel-Trenaunay-Weber Syndrome / complications,  genetics,  pathology*
Male
Mutation, Missense
Skin Diseases / complications*
alpha-Galactosidase / genetics
Chemical
Reg. No./Substance:
EC 3.2.1.22/alpha-Galactosidase

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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