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

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 Harding

Research output: Contribution to journalArticle

4 Citations (Scopus)

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.

Original languageEnglish (US)
JournalGenetics in Medicine
DOIs
StateAccepted/In press - Jan 1 2018

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Phenylketonurias
Politics
Delivery of Health Care
Phenylalanine
Manuscripts
Therapeutics
Guidelines
Enzymes

Keywords

  • anaphylaxis management
  • dietary management
  • hypophenylalaninemia
  • PALYNZIQ, pegvaliase

ASJC Scopus subject areas

  • Genetics(clinical)

Cite this

Evidence- and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria. / Longo, Nicola; Dimmock, David; Levy, Harvey; Viau, Krista; Bausell, Heather; Bilder, Deborah A.; Burton, Barbara; Gross, Christel; Northrup, Hope; Rohr, Fran; Sacharow, Stephanie; Sanchez-Valle, Amarilis; Stuy, Mary; Thomas, Janet; Vockley, Jerry; Zori, Roberto; Harding, Cary.

In: Genetics in Medicine, 01.01.2018.

Research output: Contribution to journalArticle

Longo, N, Dimmock, D, Levy, H, Viau, K, Bausell, H, Bilder, DA, Burton, B, Gross, C, Northrup, H, Rohr, F, Sacharow, S, Sanchez-Valle, A, Stuy, M, Thomas, J, Vockley, J, Zori, R & Harding, C 2018, 'Evidence- and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria', Genetics in Medicine. https://doi.org/10.1038/s41436-018-0403-z
Longo, Nicola ; Dimmock, David ; Levy, Harvey ; Viau, Krista ; Bausell, Heather ; Bilder, Deborah A. ; Burton, Barbara ; Gross, Christel ; Northrup, Hope ; Rohr, Fran ; Sacharow, Stephanie ; Sanchez-Valle, Amarilis ; Stuy, Mary ; Thomas, Janet ; Vockley, Jerry ; Zori, Roberto ; Harding, Cary. / Evidence- and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria. In: Genetics in Medicine. 2018.
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AU - Levy, Harvey

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AU - Bausell, Heather

AU - Bilder, Deborah A.

AU - Burton, Barbara

AU - Gross, Christel

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