TY - JOUR
T1 - Best practice recommendations for the management of anxiety during the pegvaliase journey
AU - Bjoraker, Kendra J.
AU - Eggerding, Caroline
AU - Ellenberg, Elisheva
AU - Hollander, Suzanne
AU - Holmes, Brittany M.
AU - Lindstrom, Kristin
AU - McNutt, Markey
AU - Miller, Suzanne
AU - Northrup, Hope
AU - Rogers, Meaghan
AU - Rose, Sarah
AU - Scott, Mia
AU - Shim, Soo
AU - Wardley, Bridget
AU - Wessenberg, Leah
AU - Bilder, Deborah A.
N1 - Publisher Copyright:
© 2023 The Authors
PY - 2024/1
Y1 - 2024/1
N2 - 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.
AB - 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.
KW - Anxiety
KW - Modified Delphi
KW - PKU
KW - Pegvaliase
KW - Phenylketonuria
KW - Recommendations
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UR - http://www.scopus.com/inward/citedby.url?scp=85178637870&partnerID=8YFLogxK
U2 - 10.1016/j.ymgme.2023.107737
DO - 10.1016/j.ymgme.2023.107737
M3 - Article
C2 - 38043481
AN - SCOPUS:85178637870
SN - 1096-7192
VL - 141
JO - Molecular genetics and metabolism
JF - Molecular genetics and metabolism
IS - 1
M1 - 107737
ER -