Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1 With MEK Inhibitor Selumetinib: A Nonrandomized Clinical Trial.

ABSTRACT:

Importance

Cutaneous neurofibromas (cNFs) can cause itching, disfigurement, pain, and emotional difficulties in people with neurofibromatosis type 1 (NF1).

Objective

To determine the impact of the mitogen-activated protein kinase kinase inhibitor selumetinib on cNF including change in tumor volume and patient-reported outcome measures.

Design, setting, and participants

Adults with NF1 and 9 or more measurable cNFs were enrolled in this nonrandomized pilot trial at the National Cancer Institute and the University of Alabama at Birmingham, which spanned from August 26, 2017, to August 21, 2023.

Intervention

Participants received selumetinib for up to 24 cycles (1 cycle = 28 days) with restaging visits after every 4 cycles.

Main outcomes and measures

Photog

SUBMITTER: Gross AM 

PROVIDER: S-EPMC11866071 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature