Safety and Efficacy of Combined Resveratrol and Sirolimus in Lymphangioleiomyomatosis.

ABSTRACT:

Background

A critical need exists to develop remission-inducing therapies for lymphangioleiomyomatosis.

Research question

Is the addition of resveratrol safe and more efficacious than sirolimus alone in patients with lymphangioleiomyomatosis?

Study design and methods

We conducted a phase 2, dose-escalating, open-label trial of resveratrol in patients with lymphangioleiomyomatosis receiving a stable regimen of sirolimus. Resveratrol was started at 250 mg/d and escalated every 8 weeks to maximum dose of 1,000 mg/d over 24 weeks. The primary outcome was ≥ 42% decline in serum vascular endothelial growth factor D (VEGF-D) levels on combined therapy compared with baseline VEGF-D levels on sirolimus. Secondary objectives included an assessment of the safety profile and the

SUBMITTER: Gupta N 

PROVIDER: S-EPMC10206511 | biostudies-literature | 2023 May

REPOSITORIES: biostudies-literature