Everolimus long-term use in patients with tuberous sclerosis complex: Four-year update of the EXIST-2 study.

ABSTRACT:

Objectives

We examined the long-term effects of everolimus in patients with renal angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis.

Methods

Following favorable results from the double-blind core phase of EXIST-2 (NCT00790400), patients were allowed to receive open-label everolimus (extension phase). Patients initially randomly assigned to everolimus continued on the same dose; those who were receiving placebo crossed over to everolimus 10 mg/day. Dose modifications were based on tolerability. The primary end point was angiomyolipoma response rate, defined as a ≥50% reduction from baseline in the sum volume of target renal angiomyolipomas in the absence of new target angiomyolipomas, kidney volume increase of >20% from nadir, and

SUBMITTER: Bissler JJ 

PROVIDER: S-EPMC5549893 | biostudies-literature | 2017

REPOSITORIES: biostudies-literature