Consensus recommendations on management of selumetinib-associated adverse events in pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas.

ABSTRACT:

Background

Selumetinib is the first approved treatment for pediatric patients with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN) in the EU and US, as well as in multiple other countries. Evidence for the management of selumetinib-associated adverse events (AEs) is mostly limited to clinical trials and expanded-access programs. We gathered a panel of European healthcare practitioners with clinical experience prescribing selumetinib and/or managing pediatric patients with NF1-PN to provide recommendations on the prevention and management of AEs.

Methods

A modified Delphi approach was used to develop the recommendations among the group of experts. Initial statements were developed from a literature review of current management recommend

SUBMITTER: Azizi AA 

PROVIDER: S-EPMC11398946 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature