Use of Pegvaliase in the Management of Phenylketonuria: Case Series of Early Experience in US Clinics
August 2021
in “
Molecular Genetics and Metabolism Reports
”
Studysummary This case series highlights the real-world challenges and experiences of US healthcare providers using pegvaliase for phenylketonuria treatment, emphasizing patient education on adverse events and dietary changes, and tailored titration schedules, while reporting that most patients achieved treatment efficacy despite initial adverse events.
Our plain-language summary. Not medical advice or a treatment recommendation. Consult a qualified healthcare professional before changing treatment. Full disclaimer
This case series examined the early use of pegvaliase in managing phenylketonuria (PKU) in 15 adult patients across US clinics, highlighting the importance of patient education and individualized treatment plans. Despite challenges such as financial issues, treatment adherence, and neuropsychological comorbidities, 12 patients achieved treatment efficacy, defined by reduced blood phenylalanine levels, with a mean time to efficacy of 20.4 weeks. Adverse events, particularly hypersensitivity reactions, were common but manageable with flexible titration schedules. The study emphasized the need for ongoing communication between patients and providers to manage dietary changes and adverse events effectively. Notably, one patient experienced hair loss due to low blood phenylalanine, which resolved with dietary adjustments. The findings aimed to guide clinicians in managing pegvaliase treatment until more extensive data became available.
