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Adjuvant treatment for phenylketonuria (PKU) |
Lindegren ML, Krishnaswami S, Fonnesbeck C, Reimschisel T, Fisher J, Jackson K, Shields T, Sathe NA, McPheeters ML |
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Record Status This is a bibliographic record of a published health technology assessment from a member of INAHTA. No evaluation of the quality of this assessment has been made for the HTA database. Citation Lindegren ML, Krishnaswami S, Fonnesbeck C, Reimschisel T, Fisher J, Jackson K, Shields T, Sathe NA, McPheeters ML. Adjuvant treatment for phenylketonuria (PKU) Rockville: Agency for Healthcare Research and Quality (AHRQ). Comparative Effectiveness Review No. 56. 2012 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' conclusions 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. Indexing Status Subject indexing assigned by CRD MeSH Adjuvants, Pharmaceutic; Phenylketonuriass Language Published English Country of organisation United States English summary An English language summary is available. Address for correspondence AHRQ, Center for Outcomes and Evidence Technology Assessment Program, 540 Gaither Road, Rockville, MD 20850, USA Email: AHRQTAP@ahrq.hhs.gov AccessionNumber 32013000184 Date abstract record published 20/03/2013 |
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