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 journalArticle

9 Citations (Scopus)

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 1 2012

Fingerprint

Phenylketonurias
Patient Selection
Phenylalanine
Therapeutics
Disease Management
Blood
sapropterin
Surveys and Questionnaires

All Science Journal Classification (ASJC) codes

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

Cite this

@article{49e6991501b543908d96d24ad45832c8,
title = "Evolving patient selection and clinical benefit criteria for sapropterin dihydrochloride (Kuvan{\circledR}) treatment of PKU patients",
abstract = "Purpose: To understand current patient selection, dosing, and response criteria used for sapropterin dihydrochloride (sapropterin, Kuvan{\circledR}) 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.",
author = "Patricia Gordon and Thomas, {Janet A.} and Ruth Suter and Elaina Jurecki",
year = "2012",
month = "4",
day = "1",
doi = "10.1016/j.ymgme.2011.12.023",
language = "English (US)",
volume = "105",
pages = "672--676",
journal = "Molecular Genetics and Metabolism",
issn = "1096-7192",
publisher = "Academic Press Inc.",
number = "4",

}

Evolving patient selection and clinical benefit criteria for sapropterin dihydrochloride (Kuvan®) treatment of PKU patients. / Gordon, Patricia; Thomas, Janet A.; Suter, Ruth; Jurecki, Elaina.

In: Molecular Genetics and Metabolism, Vol. 105, No. 4, 01.04.2012, p. 672-676.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Evolving patient selection and clinical benefit criteria for sapropterin dihydrochloride (Kuvan®) treatment of PKU patients

AU - Gordon, Patricia

AU - Thomas, Janet A.

AU - Suter, Ruth

AU - Jurecki, Elaina

PY - 2012/4/1

Y1 - 2012/4/1

N2 - 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.

AB - 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.

UR - http://www.scopus.com/inward/record.url?scp=84863393087&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84863393087&partnerID=8YFLogxK

U2 - 10.1016/j.ymgme.2011.12.023

DO - 10.1016/j.ymgme.2011.12.023

M3 - Article

C2 - 22310224

AN - SCOPUS:84863393087

VL - 105

SP - 672

EP - 676

JO - Molecular Genetics and Metabolism

JF - Molecular Genetics and Metabolism

SN - 1096-7192

IS - 4

ER -