Efficacy and Safety of Topical Rapamycin in Patients With Facial Angiofibromas Secondary to Tuberous Sclerosis Complex: The TREATMENT Randomized Clinical Trial.

ABSTRACT:

Importance

Facial angiofibromas occur in approximately 75% of individuals with tuberous sclerosis complex (TSC), causing substantial morbidity and disfigurement. Current therapies are partially effective, uncomfortable, produce scarring, and need repeating to treat recurrence.

Objective

To evaluate the efficacy and safety of topical rapamycin for TSC-related facial angiofibromas.

Design, setting, and participants

This prospective, multicenter, randomized, double-blind, vehicle-controlled trial with 6 monthly clinic visits enrolled 179 patients with TSC-related facial angiofibromas not treated within 6 months from May 2012 to March 2014 in 9 clinical sites in the United States and 1 in Australia.

Interventions

Patients were randomized (1:1:1) to topical formulat

SUBMITTER: Koenig MK 

PROVIDER: S-EPMC6128508 | biostudies-literature | 2018 Jul

REPOSITORIES: biostudies-literature