The Resource Adjuvant treatment for phenylketonuria (PKU), prepared for Agency for Healthcare Research and Quality, U.S. Department of Health and Human Services ; prepared by Vanderbilt Evidence-based Practice Center ; investigators, Mary Louise Lindegren [and others], (electronic resource)

Adjuvant treatment for phenylketonuria (PKU), prepared for Agency for Healthcare Research and Quality, U.S. Department of Health and Human Services ; prepared by Vanderbilt Evidence-based Practice Center ; investigators, Mary Louise Lindegren [and others], (electronic resource)

Label
Adjuvant treatment for phenylketonuria (PKU)
Title
Adjuvant treatment for phenylketonuria (PKU)
Statement of responsibility
prepared for Agency for Healthcare Research and Quality, U.S. Department of Health and Human Services ; prepared by Vanderbilt Evidence-based Practice Center ; investigators, Mary Louise Lindegren [and others]
Contributor
Subject
Genre
Language
eng
Summary
OBJECTIVES: We systematically reviewed evidence on adjuvant treatment of phenylketonuria (PKU) and evidence for a target phenylalanine (Phe) level to minimize cognitive impairment. DATA SOURCES: We searched MEDLINE, PsycINFO, Embase Drugs and Pharmacology, the Cumulative Index of Nursing and Allied Health Literature (CINAHL), the National Agricultural Library (AGRICOLA), and the reference lists of included studies. We searched the unpublished literature for additional data. REVIEW METHODS: We included studies published in English before August 2011. We excluded studies with fewer than 10 individuals; individual case reports; and studies lacking relevance to PKU treatment or Phe levels and measures of cognition (intelligence quotient [IQ] or core domains of executive function). We meta-analyzed studies addressing Phe level and IQ, and summarized studies of treatment in tabular form. RESULTS: We located 17 studies providing data regarding blood Phe levels and IQ changes, 10 studies addressing sapropterin dihydrochloride (BH4), and 3 addressing the use of large neutral amino acid formulations (LNAAs). Blood Phe level is positively correlated with the probability of having an IQ of less than 85. This predicted probability exceeds the population probability (approximately 15 percent) at 400 omol/L and reaches a maximum of about 80 percent at 2000 omol/L. Currently, findings on the association of Phe levels and measures of executive function are inconsistent, and too few studies have used the same outcome measures to combine data meaningfully. BH4 research to date includes two randomized controlled trials (RCTs) and three uncontrolled open-label trials. Phe levels were reduced by at least 30 percent in up to half of treated participants (32 to 50 percent). In the one RCT that compared the effect of placebo on likelihood of a 30-percent reduction in Phe, only 9 percent of those on placebo achieved this effect after 6 weeks, compared with 44 percent of the treated group. Three very small studies (total number of participants, 47) assessed LNAAs and reported no evidence that Phe levels were reduced to clinically meaningful levels. CONCLUSIONS: The strength of the evidence is moderate for a threshold effect of a Phe level of 400 omol/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
Member of
Cataloging source
NLM
Funding information
Prepared for Agency for Healthcare Research and Quality, U.S. Department of Health and Human Services ; prepared by Vanderbilt Evidence-based Practice Center
Government publication
federal national government publication
Illustrations
illustrations
Index
no index present
Literary form
non fiction
Nature of contents
  • dictionaries
  • bibliography
NLM call number
WD 205.5.A5
http://library.link/vocab/relatedWorkOrContributorName
  • Lindegren, Mary Louise
  • United States
  • Vanderbilt Evidence-based Practice Center
  • Effective Health Care Program (U.S.)
  • Effective Health Care Program (U.S.)
Series statement
  • Comparative effectiveness review
  • AHRQ publication
Series volume
  • no. 56
  • no. 12-EHC035-EF
http://library.link/vocab/subjectName
  • Phenylketonurias
  • Adjuvants, Pharmaceutic
  • Chemotherapy, Adjuvant
  • Cognition Disorders
  • Phenylalanine
  • Phenylalanine
http://bibfra.me/vocab/relation/supportingbody
RzTDSdffmEM
Label
Adjuvant treatment for phenylketonuria (PKU), prepared for Agency for Healthcare Research and Quality, U.S. Department of Health and Human Services ; prepared by Vanderbilt Evidence-based Practice Center ; investigators, Mary Louise Lindegren [and others], (electronic resource)
Instantiates
Publication
Note
"February 2012."
Bibliography note
Includes bibliographical references
Carrier category
online resource
Carrier category code
cr
Carrier MARC source
rdacarrier
Content category
text
Content type code
txt
Content type MARC source
rdacontent
Control code
793411696
Extent
1 online resource (343 pages)
Form of item
online
Media category
computer
Media MARC source
rdamedia
Media type code
c
Other physical details
illustrations
Specific material designation
remote
System control number
(OCoLC)793411696
Label
Adjuvant treatment for phenylketonuria (PKU), prepared for Agency for Healthcare Research and Quality, U.S. Department of Health and Human Services ; prepared by Vanderbilt Evidence-based Practice Center ; investigators, Mary Louise Lindegren [and others], (electronic resource)
Publication
Note
"February 2012."
Bibliography note
Includes bibliographical references
Carrier category
online resource
Carrier category code
cr
Carrier MARC source
rdacarrier
Content category
text
Content type code
txt
Content type MARC source
rdacontent
Control code
793411696
Extent
1 online resource (343 pages)
Form of item
online
Media category
computer
Media MARC source
rdamedia
Media type code
c
Other physical details
illustrations
Specific material designation
remote
System control number
(OCoLC)793411696

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