Evidence- and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria.

ABSTRACT:

Purpose

Phenylketonuria (PKU) is a rare metabolic disorder that requires life-long management to reduce phenylalanine (Phe) concentrations within the recommended range. The availability of pegvaliase (PALYNZIQ™, an enzyme that can metabolize Phe) as a new therapy necessitates the provision of guidance for its use.

Methods

A Steering Committee comprising 17 health-care professionals with experience in using pegvaliase through the clinical development program drafted guidance statements during a series of face-to-face meetings. A modified Delphi methodology was used to demonstrate consensus among a wider group of health-care professionals with experience in using pegvaliase.

Results

Guidance statements were developed for four categories: (1) treatment goals and consider

SUBMITTER: Longo N 

PROVIDER: S-EPMC6752676 | biostudies-literature | 2019 Aug

REPOSITORIES: biostudies-literature