Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis-1 with Everolimus: A Phase II, Open-Label, Single-Arm Trial.

ABSTRACT:

Background

Cutaneous neurofibromas cause disfigurement and discomfort in individuals with neurofibromatosis type 1 (NF-1).

Methods

The primary objective of this phase II, open-label, single-arm trial was to assess whether orally administered everolimus reduced the surface volume of cutaneous neurofibromas in patients with NF-1.

Results

Of 22 patients who took the study drug, 17 completed the trial; 5 patients withdrew due to adverse events. Sixteen patients had photographs of sufficient quality for assessment of the primary outcome. A significant reduction in lesion surface volume, defined as an end of trial volume > 2 standard errors (SE) less than baseline volume, was observed for 4/31 lesions (13%) from 3/16 patients (19%). Additionally, a statistically significant

SUBMITTER: Slopis JM 

PROVIDER: S-EPMC6277319 | biostudies-literature | 2018 Dec

REPOSITORIES: biostudies-literature