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


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.
Penn State Hershey Children's Hospital, Hershey, PA, USA.
Patricia
P
Gordon
Janet A
JA
Thomas
Ruth
R
Suter
Elaina
E
Jurecki
D001708:Biopterins / Q000031:analogs & derivatives* / Q000627:therapeutic use; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D005260:Female; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D009569:Nitric Oxide / Q000378:metabolism; D018579:Patient Selection; D010649:Phenylalanine / Q000097:blood*; D010661:Phenylketonurias / Q000097:blood / Q000175:diagnosis* / Q000188:drug therapy*; D017410:Practice Guidelines as Topic; D011379:Prognosis
D001708:Biopterins; D009569:Nitric Oxide; D010649:Phenylalanine; C003402:sapropterin