Best practice recommendations for the management of anxiety during the pegvaliase journey

作者：Kendra Bjoraker, Caroline Eggerding, Elisheva Ellenberg, Suzanne Hollander, B. Holmes, Kristin Lindstrom, Markey McNutt, Suzanne M. Miller, Hope Northrup, Meaghan Rogers, Sarah Rose, Mia Scott, Soo In Shim, Bridget Wardley, Leah Wessenberg, Deborah A. Bilder · 发表于：Molecular Genetics and Metabolism · 年份：2023 · DOI：10.1016/j.ymgme.2023.107737 · 被引用次数：5 · 研究领域：Metabolism and Genetic Disorders、Pharmacogenetics and Drug Metabolism、Genomics and Rare Diseases

BACKGROUND: Pegvaliase, an enzyme substitution therapy, is a treatment option for phenylketonuria (PKU). Due to the neuropathophysiology and disease burden of PKU, individuals can experience baseline anxiety unrelated to pegvaliase therapy. In addition, there are aspects of pegvaliase therapy that may be anxiety-inducing for those considering or receiving treatment. The aim of this manuscript is to present best practice recommendations for the identification and management of anxiety symptoms that can occur along the pegvaliase journey. METHODS: A modified Delphi approach was used to seek consensus among a multidisciplinary panel of experts. To this end, an in-person meeting was held that was preceded by a medical specialist- and patient-specific survey to develop preliminary recommendations on ways to address anxiety along the pegvaliase journey. After the meeting, an additional survey was conducted to rank the proposed solutions and mitigation strategies from which a set of recommendations was developed. All recommendations were voted on with the aim of consensus generation, defined as achieving ≥75% agreement among experts. RESULTS: The panel reached consensus on a total of 28 best practice recommendations for the management of anxiety during the pre-treatment, induction and titration, early maintenance (pre-efficacy), and late maintenance (post-efficacy) stages. The recommendations offer strategies to identify and address the most common causes of pegvaliase-related anxie...