Evolving patient selection and clinical benefit criteria for sapropterin dihydrochloride (Kuvan®) treatment of PKU patients

Patricia Gordon, Janet A. Thomas, Ruth Suter, Elaina Jurecki

Research output: Contribution to journalArticlepeer-review

9 Scopus citations

Abstract

Purpose: To understand current patient selection, dosing, and response criteria used for sapropterin dihydrochloride (sapropterin, Kuvan®) to treat phenylketonuria (PKU). Methods: Results of a 2010 survey of twenty-nine academic medical centers are reported to describe practice patterns in comparison to results of a survey done in 2008 and to what is reported in the literature. Results/conclusions: In addition to reduction in blood phenylalanine (Phe) levels, clinicians report using broader disease-management approaches when evaluating clinical benefit of sapropterin, including consideration of increased Phe tolerance and behavioral changes. Similar approaches are reported in the literature.

Original languageEnglish (US)
Pages (from-to)672-676
Number of pages5
JournalMolecular Genetics and Metabolism
Volume105
Issue number4
DOIs
StatePublished - Apr 2012

All Science Journal Classification (ASJC) codes

  • Endocrinology, Diabetes and Metabolism
  • Biochemistry
  • Molecular Biology
  • Genetics
  • Endocrinology

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