[A new treatment for vascular anomalies: Six cases treated with rapamycin].

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[A new treatment for vascular anomalies: Six cases treated with rapamycin].

Arch Pediatr. 2017 Jul;24(7):600-606

Authors: Thirion S, Jamblin P, Demarche M, Boon L, Thiry A, Hoyoux C

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.

PMID: 28595829 [PubMed - indexed for MEDLINE]

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