First 1.5 years of pegvaliase clinic: Experiences and outcomes
Stephanie Sacharow, Cassandra Papaleo, Kyla Almeida, Benjamin D. Goodlett, Amy Kritzer, Harvey L. Levy, Leslie Martell, Ann Wessel, Krista Viau
Abstract
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.
Topics & Concepts
MedicinePediatricsPhenylalanineInternal medicineAmino acidBiochemistryChemistryMetabolism and Genetic DisordersNeonatal Health and BiochemistryDiet and metabolism studies