title: The therapeutic response in Gorham's syndrome to the beta-blocking agent propranolol is correlated to VEGF-A, but not to VEGF-C or FLT1 expression.
abstract: BACKGROUND
  Gorham's syndrome is a rare illness of unknown etiology. It is characterized by a local proliferation of blood or lymphatic vessels that in bones leads to progressive resorption and destruction. The cause of the disease is not elucidated, and therapeutic options remain limited.
  
  
  METHODS
  We report herein the case of a young female Caucasian patient aged 18 years with diffuse Gorham syndrome. In tissue specimens angiogenesis and massive lymphangiogenesis as well as the expression of vascular endothelial growth factor-A (VEGF-A) and neuropilins was observed. Lymphangiogenesis is a prominent feature of the disease and a number of lymphatic markers were found to be expressed, however only VEGF-A, but not vascular endothelial growth factor-C (VEGF-C) was found to be elevated in the circulation. Circulating levels of soluble VEGF receptor-1 were also not elevated. Furthermore, the patient responded favorably and the disease was stabilized following treatment with the beta-blocking agent Propranolol alone which acts on VEGF-A alone, but not on soluble VEGF receptor-1 levels.
  
  
  CONCLUSIONS
  This suggests that the disease is dependent on VEGF-A, but on neither VEGF-C, the major driver of lymphangiogenesis, nor FLT1. Furthermore, Propranolol acts on VEGF-A but not FLT1 expression.
authors.affiliation: INSERM U1029, Allée Geoffroy St. Hilaire, 33 615, Pessac, France. jessica.massiere@u-bordeaux.fr.|Université Bordeaux, Allée Geoffroy St. Hilaire, 33 615, Pessac, France. jessica.massiere@u-bordeaux.fr.
authors.forename: Jessica
authors.initials: J
authors.lastname: Baud
authors.affiliation: INSERM U1029, Allée Geoffroy St. Hilaire, 33 615, Pessac, France. rahim.lomri@inserm.fr.|Université Bordeaux, Allée Geoffroy St. Hilaire, 33 615, Pessac, France. rahim.lomri@inserm.fr.
authors.forename: Abderrahim
authors.initials: A
authors.lastname: Lomri
authors.affiliation: Service de Pédiatrie, Hôpital Saint Louis, Rue du Dr Schweitzer, 17019, La Rochelle Cedex, France. Denis.GRABER@ch-larochelle.fr.
authors.forename: Denis
authors.initials: D
authors.lastname: Graber
authors.affiliation: INSERM U1029, Allée Geoffroy St. Hilaire, 33 615, Pessac, France. andreas.bikfalvi@u-bordeaux.fr.|Université Bordeaux, Allée Geoffroy St. Hilaire, 33 615, Pessac, France. andreas.bikfalvi@u-bordeaux.fr.
authors.forename: Andreas
authors.initials: A
authors.lastname: Bikfalvi
mesh_terms: D000293:Adolescent; D000319:Adrenergic beta-Antagonists / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D042583:Lymphangiogenesis; D009389:Neovascularization, Pathologic; D010015:Osteolysis, Essential / Q000097:blood* / Q000188:drug therapy*; D011433:Propranolol / Q000627:therapeutic use*; D016896:Treatment Outcome; D042461:Vascular Endothelial Growth Factor A / Q000097:blood*; D042582:Vascular Endothelial Growth Factor C / Q000097:blood*; D040281:Vascular Endothelial Growth Factor Receptor-1 / Q000097:blood*
chemical_list: D000319:Adrenergic beta-Antagonists; C467484:VEGFA protein, human; C467486:VEGFC protein, human; D042461:Vascular Endothelial Growth Factor A; D042582:Vascular Endothelial Growth Factor C; D011433:Propranolol; C501162:FLT1 protein, human; D040281:Vascular Endothelial Growth Factor Receptor-1