Patients with Congenital Low-Flow Vascular Malformation Treated with Low Dose Sirolimus.

ABSTRACT:

Introduction

Patients with congenital vascular malformations often suffer from an impaired quality of life (QoL) because of pain and functional disabilities. Previous studies have shown that the mTOR inhibitor sirolimus can reduce complaints and improve QoL in some patients. High target levels of sirolimus of 10-15 ng/ml were well tolerated; however, in a relative high percentage of patients sirolimus caused serious adverse events (AEs).

Methods

A case series of 12 patients with therapy-resistant low-flow vascular malformations was treated with sirolimus, using low target levels of 4-10 ng/ml. Efficacy of sirolimus was evaluated in regard to pain symptoms using the visual analogue scale/numeric rating scale and patients reported QoL. To rule out a placebo effect of sirolimus

SUBMITTER: Harbers VEM 

PROVIDER: S-EPMC8190005 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature