First 1.5 years of pegvaliase clinic: Experiences and outcomes

We present Boston Children's Hospital's clinic model for pegvaliase therapy in adults with phenylketonuria (PKU) and clinical outcomes in 46 patients over the first 1.5 years of commercial therapy. Approximately 70% (18/26) of patients starting pegvaliase achieved blood phenylalanine (Phe)...

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Detalles Bibliográficos
Autores principales: Sacharow, Stephanie, Papaleo, Cassandra, Almeida, Kyla, Goodlett, Benjamin, Kritzer, Amy, Levy, Harvey, Martell, Leslie, Wessel, Ann, Viau, Krista
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
Materias:
Short Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7256053/
https://www.ncbi.nlm.nih.gov/pubmed/32489881
http://dx.doi.org/10.1016/j.ymgmr.2020.100603
Descripción
Sumario:We present Boston Children's Hospital's clinic model for pegvaliase therapy in adults with phenylketonuria (PKU) and clinical outcomes in 46 patients over the first 1.5 years of commercial therapy. Approximately 70% (18/26) of patients starting pegvaliase achieved blood phenylalanine (Phe) <360 μmol/L, with an average of a 68 ± 24% decrease in blood Phe from baseline. All patients experienced at least minor side effects, but in most, management of the side effects allowed for treatment to continue.

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