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Thalidomide stimulates vessel maturation and reduces epistaxis in individuals with hereditary hemorrhagic telangiectasia

Lookup NU author(s): Professor Helen ArthurORCiD


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Hereditary hemorrhagic telangiectasia (HHT) is an inherited disorder characterized by vascular malformations. Many affected individuals develop recurrent nosebleeds, which can severely affect their quality of life and are clinically difficult to treat. We report here that treatment with thalidomide reduced the severity and frequency of nosebleeds (epistaxis) in the majority of a small group of subjects with HHT tested. The blood hemoglobin levels of the treated individuals rose as a result of reduced hemorrhage and enhanced blood vessel stabilization. In mice heterozygous for a null mutation in the Eng gene (encoding endoglin), an experimental model of HHT, thalidomide treatment stimulated mural cell coverage and thus rescued vessel wall defects. Thalidomide treatment increased platelet-derived growth factor-B (PDGF-B) expression in endothelial cells and stimulated mural cell activation. The effects of thalidomide treatment were partially reversed by pharmacological or genetic interference with PDGF signaling from endothelial cells to pericytes. Biopsies of nasal epithelium from individuals with HHT treated or not with thalidomide showed that similar mechanisms may explain the effects of thalidomide treatment in humans. Our findings demonstrate the ability of thalidomide to induce vessel maturation, which may be useful as a therapeutic strategy for the treatment of vascular malformations.

Publication metadata

Author(s): Lebrin F, Srun S, Raymond K, Martin S, van den Brink S, Freitas C, Breant C, Mathivet T, Larrivee B, Thomas JL, Arthur HM, Westermann CJJ, Disch F, Mager JJ, Snijder RJ, Eichmann A, Mummery CL

Publication type: Article

Publication status: Published

Journal: Nature Medicine

Year: 2010

Volume: 16

Issue: 4

Pages: 420-428

Print publication date: 01/04/2010

ISSN (print): 1078-8956

ISSN (electronic): 1546-170X

Publisher: Nature Publishing Group


DOI: 10.1038/nm.2131


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Funder referenceFunder name
Besluit subsidies investeringen kennisinfrastructuur
British Heart Foundation
Fondation Bettencourt
Agence Nationale de la Recherche (Agence Nationale de la Recherche blanc, Neuroscience)
Fondation pour la Recherche Medicale
HHT Foundation International
Institut National de la Sante et de la Recherche Medicale
Stichting Wetenschappelijk Onderzoek Rendu Osler
2008B106Netherlands Heart Foundation
3980Association pour la Recherche sur le Cancer