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Journal article
Improved attention linked to sustained phenylalanine reduction in adults with early-treated phenylketonuria
American journal of medical genetics. Part A, v 188(3), pp 768-778
Mar 2022
PMID: 34826353
Featured in Collection : UN Sustainable Development Goals @ Drexel
Abstract
Pegvaliase is approved to reduce phenylalanine (Phe) levels for people with phenylketonuria (PKU). PRISM-1 (NCT01819727) and PRISM-2 (NCT01889862) data were analyzed to evaluate the relationship between Phe and inattention in adult participants with PKU. In the modified-intent-to-treat population (N = 156), baseline mean (SE) plasma Phe was 1263 (29) μmol/L and the Attention Deficit Hyperactivity Disorder Rating Scale-IV Inattentive (IA) symptoms score was 9.8 (0.5). Mean (SE) IA scores fell 9.0 (1.1) in Quartile 1 (Phe reduction between 1166 and 2229 μmol/L) versus 4.3 (0.7) in Quartile 4 (Phe reduction of 139 μmol/L to increase of 934 μmol/L), p = 0.004. Least squares mean (SE) change from baseline IA score was -7.9 (0.7) for participants with final Phe ≤ 360 μmol/L and -4.5 (0.7) for final Phe > 360 μmol/L, p < 0.001. In the inattention subgroup, IA scores fell 13.3 (1.5) in Quartile 1 (Phe reduction between 1288 and 2229 μmol/L) versus 6.2 (1.3) in Quartile 4 (Phe reduction of 247 to increase of 934 μmol/L), p = 0.009. Inattention symptoms improved among those whose Phe levels decreased, particularly those with high baseline IA scores. IA improvements were larger among participants with the greatest plasma Phe reductions, supporting this value as a therapeutic goal.
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Details
- Title
- Improved attention linked to sustained phenylalanine reduction in adults with early-treated phenylketonuria
- Creators
- Deborah A Bilder - Huntsman Cancer InstituteGeorgianne L Arnold - Children's Hospital of PittsburghDavid Dimmock - Children’s InstituteMitzie L Grant - St. Christopher's Hospital for ChildrenDarren Janzen - Oregon Health & Science UniversityNicola Longo - University of UtahMina Nguyen-Driver - Oregon Health & Science UniversityElaina Jurecki - BioMarinMarkus Merilainen - BioMarinGianni Amato - BioMarinSusan Waisbren - Boston Children's Hospital
- Publication Details
- American journal of medical genetics. Part A, v 188(3), pp 768-778
- Publisher
- Wiley
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Psychiatry
- Web of Science ID
- WOS:000722708900001
- Scopus ID
- 2-s2.0-85119839333
- Other Identifier
- 991019167983904721
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- Collaboration types
- Industry collaboration
- Domestic collaboration
- Web of Science research areas
- Genetics & Heredity