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

@article{Lebrin2010ThalidomideSV,
  title={Thalidomide stimulates vessel maturation and reduces epistaxis in individuals with hereditary hemorrhagic telangiectasia},
  author={Franck Lebrin and Samly Srun and Karine Raymond and Sabrina Martin and Stieneke van den Brink and Catarina Freitas and Ch. Br{\'e}ant and Thomas Mathivet and Bruno Larriv{\'e}e and Jean-L{\'e}on Thomas and Helen M. Arthur and Cornelis J. J. Westermann and F. J. M. Disch and Johannes Jurgen Mager and Repke J. Snijder and Anne Eichmann and Christine L. Mummery},
  journal={Nature Medicine},
  year={2010},
  volume={16},
  pages={420-428}
}
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… 

Pericytes in Hereditary Hemorrhagic Telangiectasia.

How disturbed TGF-β and VEGF signaling relates to blood vessel destabilization and HHT development is reviewed and therapeutic opportunities based on the concept of vessel normalization to treat HHT are discussed.

Anti-angiogenic therapeutic strategies in hereditary hemorrhagic telangiectasia

The experimental evidence for dysregulated angiogenesis in HHT, the anti-angiogenic therapeutic strategies used in animal models and some patients with HHT and the potential benefit of theAnti-Angiogenic treatment for ameliorating this severe, progressive vascular disease are reviewed.

Thalidomide Effects in Patients with Hereditary Hemorrhagic Telangiectasia During Therapeutic Treatment and in Fli-EGFP Transgenic Zebrafish Model

Thalidomide reverses telangiectasia and controls nosebleeds by down-regulating the expression of TGF-&bgr;3 and VEGF in HHT patients and it also leads to vascular remodeling in the zebrafish model.

Functional Alterations Involved in Increased Bleeding in Hereditary Hemorrhagic Telangiectasia Mouse Models

The results demonstrate that HHT patients with high bleeding, as determined by a high Epistaxis Severity Score (ESS), do not have prolonged clotting times or alterations in clotting factors, and support the idea that endoglin and Alk1 haploinsufficiency leads to a common phenotype of impaired hemostasis, but through different mechanisms.

Propanolol and angiogenesis inhibition in hereditary haemorrhagic telangiectasia.

  • G. Breier
  • Medicine, Biology
    Thrombosis and haemostasis
  • 2012
The development of the vascular lesions in HHT has not only been attributed to decreased TGF-β signalling but also to increased activity of the pro-angiogenic VEGF pathway, which suggests that raloxifene counteracts the haploinsufficiency of ENG and ALK1 and may be beneficial for epistaxis treatment in H HT menopausal women.

Increase of circulating endothelial cells in patients with Hereditary Hemorrhagic Telangiectasia

Hereditary Hemorrhagic Telangiectasia patients show an increase of circulating endothelial cells and a decrease of HPCs, suggesting that their active turnover characterizes the initial phase of the disease.

Thalidomide for Hereditary Hemorrhagic Telangiectasia With Pulmonary Arterial Hypertension.

Thalidomide was beneficial against mucocutaneous bleeding,1–7 but careful consideration is required with regard to its initiation in HHT patients with PAH, as reflected by serial changes in B-type natriuretic peptide and tricuspid regurgitation pressure gradient.

Thresholds of Endoglin Expression in Endothelial Cells Explains Vascular Etiology in Hereditary Hemorrhagic Telangiectasia Type 1

A positive correlation between low basal levels of endoglin and the general prevalence of clinical manifestations in selected organs is found and the development of drugs promotingendoglin expression as potentially protective is supported.

Mononuclear cells and vascular repair in HHT

A review summarizes recent studies regarding the role of MNCs in the etiology of HHT and vascular repair, and evaluates the efficacy of DPP4 inhibition in tissue integrity and repair.
...

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