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

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 anxiety, including self-injection, side effects, the titration schedule, prescribed dietary changes, and variable time to efficacy. Overall, managing anxiety in those considering or receiving pegvaliase involves patient-centered communication, shared decision-making, and personalized treatment plans.
Conclusions: The best practice recommendations described herein can guide healthcare providers in proactively addressing anxiety during the different stages of pegvaliase treatment, and support providers with initiating and managing pegvaliase in individuals who may experience baseline and treatment-related anxiety.
Competing Interests: Declaration of Competing Interest The expert panel was compensated for their participation in the in-person advisory board. In addition, KJB received consulting payments from BioMarin, Denali, Chiesi, and Sanofi and speaker fees as well as travel support from BioMarin, Chiesi, and Sanofi; CE received speaker fees from BioMarin; EE received consulting payments and speaker fees from BioMarin. SH received consulting payments from BioMarin, Cycle Therapeutics, PTC Therapeutics, Sanofi, and Vitaflo, speaker fees from Vitaflo, and participated as clinical trial investigator for BioMarin and PTC Therapeutics; BMH received consulting payments from BioMarin, Horizon Therapeutics, and Synlogic; MM received consulting payments from Horizon Therapeutics, BioMarin, Eton Pharmaceuticals, Acer Therapeutics, Ultragenyx, Applied Therapeutics, Jnana Therapeutics, BridgeBio, and Alexion, and participated as clinical trial investigator for Aeglea Biotherapeutics, Reneo Pharmaceuticals, PTC Therapeutics, Homology Medicines, Horizon Therapeutics, Arcturus Therapeutics, Jnana Therapeutics, Synlogic Therapeutics, and BioMarin; SM received consulting payments from Horizon Therapeutics; HN is a clinical trial investigator for BioMarin, Synlogic Therapeutics, Jnana Therapeutics, Sanofi, and PTC Therapeutics and received consulting fees from BioMarin, Synlogic Therapeutics, Jnana Therapeutics, and PTC as well as speakers fees from BioMarin; SS received consulting payments from BioMarin and travel support from BioMarin and NPKUA; LW received consulting payments from BioMarin and Cycle Pharmaceuticals; DAB received consulting payments from Synlogic Therapeutics, Encoded Therapeutics, and Taysha Gene Therapies as well as consulting payments and travel support from BioMarin. MR and MS have no conflicts of interest to declare. KL, SR, and BW are employees and shareholders of BioMarin Pharmaceutical Inc.
(Copyright © 2023 The Authors. Published by Elsevier Inc. All rights reserved.)