Gamma secretase inhibitors (GSI) have been determined to attenuate vascular malformations by inhibiting NOTCH expression in endothelial cells. Vascular malformations are developmental abnormalities of venous, arterial, capillary or lymphatic vessels which have significant morbidities such as pain, infection, pulmonary emboli, bleeding and even death. There is growing interest in medical management of vascular malformations, however existing treatments often do not result in complete lesion resolution and do not produce permanent results, thus there is a significant need for alternative therapies. The use of GSIs can provide an alternative and potent treatment for vascular formations.
- Notch signaling pathway is a potential therapeutic target for extracranial vascular malformations Sci Rep. 2018 Dec 20;8(1):17987