Related Experiment Video
Updated: Mar 1, 2026
A Patient-Derived Xenograft Model for Venous Malformation
Published on: June 15, 2020
[A new treatment for vascular anomalies: Six cases treated with rapamycin]
S Thirion1, P Jamblin2, M Demarche3
1Faculté de médecine, université de Liège (ULg), CHR de la Citadelle, 1, boulevard du 12(e)-de-Ligne, 4000 Liège, Belgique.
Insights
Rapamycin, an mTOR inhibitor, shows promise for treating complicated vascular anomalies (VAs) that resist standard therapies. This treatment offers hope for improving patients' quality of life by managing these lifelong, progressive vascular conditions.
Area of Science:
- Vascular biology and developmental abnormalities.
- Pharmacological interventions for congenital disorders.
Background:
- Vascular anomalies (VAs) arise from embryonic vascular system defects, presenting as malformed vessels.
- These anomalies are non-regressing, grow with the patient, and can worsen without treatment.
Observation:
- Complicated VAs often cause chronic pain, swelling, bleeding, and functional deficits.
- Patient quality of life is significantly impaired due to the persistent and recurring nature of VAs.
Findings:
- Six cases of complicated VAs, unresponsive to conventional treatments, were treated with rapamycin.
- Rapamycin, an mTOR inhibitor, demonstrated potential in managing these refractory vascular anomalies.
Implications:
- Rapamycin offers a potential new therapeutic avenue for complex vascular anomalies.
- Further research into mTOR inhibition could improve management strategies and patient outcomes for VAs.
Abstract:
Vascular anomalies (VAs) result from the defective development of the embryonic vascular system and feature dysplastic malformed vessels, which are not always apparent at birth. They do not regress over the patient's lifetime; they usually have commensurate growth during childhood and may worsen over time if not treated. VAs may cause chronic painful swelling, bleeding, functional deficits or vital structure obstruction. These patients' quality of life is usually impaired because of the chronicity and recurrence of the disease. We report on six cases of complicated VAs, refractory to current treatments, treated with rapamycin, an mTor inhibitor recently used in VAs.
