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 considerations prior to initiating therapy, (2) dosing considerations, (3) considerations for dietary management, and (4) best approaches to optimize medical management. A total of 34 guidance statements were included in the modified Delphi voting and consensus was reached on all after two rounds of voting.

CONCLUSION:

Here we describe evidence- and consensus-based recommendations for the use of pegvaliase in adults with PKU. The manuscript was evaluated against the Appraisal of Guidelines for Research and Evaluation (AGREE II) instrument and is intended for use by health-care professionals who will prescribe pegvaliase and those who will treat patients receiving pegvaliase.

• Authors: Nicola Longo, David Dimmock, Harvey Levy, Krista Viau, Heather Bausell, Deborah A Bilder, Barbara Burton, Christel Gross, Hope Northrup, Fran Rohr, Stephanie Sacharow, Amarilis Sanchez-Valle, Mary Stuy, Janet Thomas, Jerry Vockley, Roberto Zori, Cary O Harding
• Journal: Genetics in medicine : official journal of the American College of Medical Genetics (Genet Med) Vol. 21 Issue 8 Pg. 1851-1867 (08 2019) ISSN: 1530-0366 [Electronic] United States
• PMID: 30546086 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Recombinant Proteins
• Phenylalanine
• Phenylalanine Ammonia-Lyase
• pegvaliase

Topics

• Adolescent
• Adult
• Child
• Dose-Response Relationship, Drug
• Humans
• Middle Aged
• Phenylalanine (genetics, metabolism)
• Phenylalanine Ammonia-Lyase (blood, genetics, therapeutic use)
• Phenylketonurias (blood, drug therapy, genetics, pathology)
• Recombinant Proteins (blood, genetics, therapeutic use)
• Young Adult