Molecular genetics and metabolism · 2026
Management of pegvaliase-related skin concerns: best practice recommendations using a modified Delphi approach.
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Ameijeiras Alvaro Hermida, Vucko Erika, Harding Cary O, Lah Melissa, Muntau Ania Carolina, Rovelli Valentina, Thomas Janet A, Rose Sarah, Lindstrom Kristin, Sacharow Stephanie
Аннотация
INTRODUCTION: The approval of pegvaliase (PALYNZIQ®) represented a paradigm shift in the management of phenylketonuria (PKU) by enabling sustained reductions in blood phenylalanine levels and permitting an unrestricted diet. However, as it is a bacterially derived injectable therapy, individuals receiving pegvaliase may experience skin-related adverse events (AEs). Anticipating these AEs and other pegvaliase-related skin concerns and having best-practice strategies to manage them may be crucial to achieving optimal patient outcomes.
METHODS: An expert panel comprising eight clinicians from the USA and Europe convened to develop best practice recommendations for managing pegvaliase-associated skin concerns. Employing a modified Delphi process, the panel generated 39 initial statements based on their clinical insights and results of a targeted literature review. These statements were refined over two phases of anonymous voting using a 10-point scale, with consensus predefined as ≥75% of panellists rating a statement ≥7.
RESULTS: The Delphi process resulted in 18 consensus statements. These were organized into three themes: patient-centric recommendations/considerations; considerations for premedications and concomitant medications; and recommendations for monitoring. Key recommendations included comprehensive patient education to overcome needle phobia and promote self-injection; training on injection technique and rotation to reduce scar tissue formation; and the proactive use of premedications and as-needed concomitant medications to mitigate skin reactions. Recommendations also emphasize the importance of regular in-person follow-up to assess injection sites and monitor for scar tissue. Mean agreement scores ranged from 8.5 to 10, reflecting robust consensus among panel members.
CONCLUSIONS: These internationally relevant, evidence-based recommendations provide a structured framework for managing pegvaliase-related skin concerns. Adoption of this guidance may enhance treatment adherence, mitigate adverse events, and ultimately improve patient outcomes.
METHODS: An expert panel comprising eight clinicians from the USA and Europe convened to develop best practice recommendations for managing pegvaliase-associated skin concerns. Employing a modified Delphi process, the panel generated 39 initial statements based on their clinical insights and results of a targeted literature review. These statements were refined over two phases of anonymous voting using a 10-point scale, with consensus predefined as ≥75% of panellists rating a statement ≥7.
RESULTS: The Delphi process resulted in 18 consensus statements. These were organized into three themes: patient-centric recommendations/considerations; considerations for premedications and concomitant medications; and recommendations for monitoring. Key recommendations included comprehensive patient education to overcome needle phobia and promote self-injection; training on injection technique and rotation to reduce scar tissue formation; and the proactive use of premedications and as-needed concomitant medications to mitigate skin reactions. Recommendations also emphasize the importance of regular in-person follow-up to assess injection sites and monitor for scar tissue. Mean agreement scores ranged from 8.5 to 10, reflecting robust consensus among panel members.
CONCLUSIONS: These internationally relevant, evidence-based recommendations provide a structured framework for managing pegvaliase-related skin concerns. Adoption of this guidance may enhance treatment adherence, mitigate adverse events, and ultimately improve patient outcomes.