First 1.5 Years of Pegvaliase Clinic: Experiences and Outcomes

Overview

Journal Mol Genet Metab Rep

Specialty Endocrinology

Date 2020 Jun 4

PMID 32489881

Citations 7

Authors

Stephanie Sacharow

Cassandra Papaleo

Kyla Almeida

Benjamin Goodlett

Amy Kritzer

Harvey Levy

Leslie Martell

Ann Wessel

Krista Viau

Affiliations

Soon will be listed here.

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.

Citing Articles

Nutritional status of adults with phenylketonuria on pegvaliase: A 15-month prospective study.

Viau K, Martell L, Wessel A, Rohr F, Hollander S, Putman M Mol Genet Metab Rep. 2023; 37:101015.

PMID: 38053931 PMC: 10694747. DOI: 10.1016/j.ymgmr.2023.101015.

Expert Consensus on the Long-Term Effectiveness of Medical Nutrition Therapy and Its Impact on the Outcomes of Adults with Phenylketonuria.

Rocha J, Ahring K, Bausell H, Bilder D, Harding C, Inwood A Nutrients. 2023; 15(18).

PMID: 37764724 PMC: 10536918. DOI: 10.3390/nu15183940.

Nutrition management of PKU with pegvaliase therapy: update of the web-based PKU nutrition management guideline recommendations.

Cunningham A, Rohr F, Splett P, Mofidi S, Bausell H, Stembridge A Orphanet J Rare Dis. 2023; 18(1):155.

PMID: 37349772 PMC: 10288765. DOI: 10.1186/s13023-023-02751-0.

Phenylalanine hydroxylase mRNA rescues the phenylketonuria phenotype in mice.

Cacicedo M, Weinl-Tenbruck C, Frank D, Limeres M, Wirsching S, Hilbert K Front Bioeng Biotechnol. 2022; 10:993298.

PMID: 36277393 PMC: 9585315. DOI: 10.3389/fbioe.2022.993298.

Real-world treatment, dosing, and discontinuation patterns among patients treated with pegvaliase for phenylketonuria: Evidence from dispensing data.

Lah M, Cook K, Gomes D, Liu S, Tabatabaeepour N, Kirson N Mol Genet Metab Rep. 2022; 33:100918.

PMID: 36176956 PMC: 9513695. DOI: 10.1016/j.ymgmr.2022.100918.

References

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