First 1.5 years of pegvaliase clinic: Experiences and outcomes

Stephanie Sacharow; Cassandra Papaleo; Kyla Almeida; Benjamin Goodlett; Amy Kritzer; Harvey Levy; Leslie Martell; Ann Wessel; Krista Viau

doi:10.1016/j.ymgmr.2020.100603

First 1.5 years of pegvaliase clinic: Experiences and outcomes

Mol Genet Metab Rep. 2020 May 25:24:100603. doi: 10.1016/j.ymgmr.2020.100603. eCollection 2020 Sep.

Authors

Stephanie Sacharow¹, Cassandra Papaleo², Kyla Almeida², Benjamin Goodlett¹, Amy Kritzer¹, Harvey Levy¹, Leslie Martell², Ann Wessel², Krista Viau²

Affiliations

¹ Department of Pediatrics, Harvard Medical School, Boston, MA, United States of America.
² Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, United States of America.

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.