Sirolimus Gel Treatment vs Placebo for Facial Angiofibromas in Patients With Tuberous Sclerosis Complex: A Randomized Clinical Trial.

ABSTRACT:

Importance

Most patients with tuberous sclerosis complex (TSC), an autosomal-dominant disorder that is caused by the constitutive activation of mammalian target of rapamycin, experience disfigurement caused by skin lesions involving facial angiofibromas. Many have been left untreated because of a lack of therapeutic options that are less invasive than surgery or laser treatment.

Objective

To confirm the efficacy and safety of sirolimus gel, 0.2%, for treatment of patients with angiofibromas and/or skin lesions.

Design, setting, and patients

Multicenter, randomized clinical trial at 9 centers in Japan from December 2015 to October 2016 including 62 children and adults with TSC.

Interventions

Patients who developed angiofibromas were randomly assigned, in a 1:1 r

SUBMITTER: Wataya-Kaneda M 

PROVIDER: S-EPMC6128500 | biostudies-literature | 2018 Jul

REPOSITORIES: biostudies-literature