The impact on quality of life of diet restrictions and disease symptoms associated with phenylketonuria: a time trade-off and discrete choice experiment study

Sara Olofsson (), Katarina Gralén, Christina Hoxer, Paul Okhuoya and Ulf Persson
Additional contact information
Sara Olofsson: The Swedish Institute for Health Economics, IHE
Katarina Gralén: The Swedish Institute for Health Economics, IHE
Christina Hoxer: BioMarin Pharmaceutical Inc
Paul Okhuoya: BioMarin Pharmaceutical Inc
Ulf Persson: The Swedish Institute for Health Economics, IHE

The European Journal of Health Economics, 2022, vol. 23, issue 6, No 7, 993-1005

Abstract: Abstract Phenylketonuria (PKU) is a metabolic disorder leading to a deficiency in the metabolism of phenylalanine (Phe). Elevated Phe levels in the blood, tissue and brain may lead to emotional, cognitive, and physical symptoms in adults. To control blood Phe levels, most individuals with PKU need to follow a strict, life-long Phe-restricted diet. The main objective of this study was to estimate the impact of PKU-related disease symptoms and dietary restrictions on health-related quality of life (HRQoL). This study was designed as a web-based survey using the methods of Time Trade-Off (TTO) and Discrete Choice Experiment with duration (DCETTO) in a survey of the Swedish general population and population with PKU. Data were collected using questionnaires for TTO diet (n = 509), TTO symptoms (n = 507), and DCETTO (n = 1117). The disutility of diet restrictions ranged from 0.050 for a partially restricted diet without medical food to 0.193 for a fully restricted diet with medical food in the TTO (DCE: 0.043–0.108). The disutility of experiencing symptoms (emotional, cognitive and physical) ranged from 0.148 for mild symptoms to 0.593 for severe symptoms in the TTO (DCE: 0.122–1.522). The study shows that both diet and symptoms have a significant negative impact on HRQoL in PKU and that utility estimates are largely consistent across methods and samples.

Keywords: Phenylketonuria; Phenylalanine hydroxylase deficiency; Quality of life; Burden of illness; Time trade-off; Discrete choice (search for similar items in EconPapers)
JEL-codes: I1 I31 (search for similar items in EconPapers)
Date: 2022
References: View references in EconPapers View complete reference list from CitEc
Citations:

Downloads: (external link)
http://link.springer.com/10.1007/s10198-021-01415-9 Abstract (text/html)
Access to the full text of the articles in this series is restricted.

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:spr:eujhec:v:23:y:2022:i:6:d:10.1007_s10198-021-01415-9

Ordering information: This journal article can be ordered from
http://www.springer. ... cs/journal/10198/PS2

DOI: 10.1007/s10198-021-01415-9

Access Statistics for this article

The European Journal of Health Economics is currently edited by J.-M.G.v.d. Schulenburg

More articles in The European Journal of Health Economics from Springer, Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ) Contact information at EDIRC.
Bibliographic data for series maintained by Sonal Shukla () and Springer Nature Abstracting and Indexing ().