Adjuvant treatment for phenylketonuria (PKU)
Lindegren ML, Krishnaswami S, Fonnesbeck C, Reimschisel T, Fisher J, Jackson K, Shields T, Sathe NA, McPheeters ML
Record ID 32013000184
English
Authors' objectives:
We systematically reviewed evidence on adjuvant treatment of phenylketonuria (PKU) and evidence for a target phenylalanine (Phe) level to minimize cognitive impairment.
Authors' recommendations:
The strength of the evidence is moderate for a threshold effect of a Phe level of 400 ìmol/L associated with IQ <85. Evidence on the association of Phe and measures of executive function is insufficient. The use of adjuvant therapy in PKU is novel; the strength of the evidence is currently moderate for short-term effects on reducing Phe in a subset of initially responsive individuals and low for longer term effects on cognition.
Details
Project Status:
Completed
Year Published:
2012
URL for published report:
https://effectivehealthcare.ahrq.gov/sites/default/files/pdf/phenylketonuria_research.pdf
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
United States
MeSH Terms
- Phenylketonurias
- Phenylalanine
- Drug Therapy
Contact
Organisation Name:
Agency for Healthcare Research and Quality
Contact Address:
Center for Outcomes and Evidence Technology Assessment Program, 540 Gaither Road, Rockville, MD 20850, USA. Tel: +1 301 427 1610; Fax: +1 301 427 1639;
Contact Name:
martin.erlichman@ahrq.hhs.gov
Contact Email:
martin.erlichman@ahrq.hhs.gov
Copyright:
Agency for Healthcare Research and Quality (AHRQ)
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.