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Using EQ-5D to derive general population-based utilities for the Quality of Life-Assessment of Growth Hormone Deficiency in Adults (QoL-AGHDA)
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Pharmacy, Department of Pharmacy.
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Women's and Children's Health. (Barnendokrinologisk forskning/Gustafsson)
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Pharmacy, Department of Pharmacy.
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Pharmacy, Department of Pharmacy.
2007 (English)In: Value in Health, ISSN 1098-3015, E-ISSN 1524-4733, Vol. 10, no 1, 73-81 p.Article in journal (Refereed) Published
Abstract [en]

Objective: Disease-oriented quality of life (QoL) measures that are not preference-based lack legitimacy for direct use in cost-utility analyses. This has prompted the search for other methods for deriving utilities. The QoL Assessment of Growth Hormone Deficiency in Adults questionnaire (QoL-AGHDA) is a disease-oriented measure used to assess impairment in QoL in adults with growth hormone deficiency. The present study was designed to generate a model for deriving utilities from the QoL-AGHDA. Methods: The EQ-5D, the QoL-AGHDA, and demographic questions were mailed to a random sample (n = 3005) of the Swedish population (response rate 65%). Multiple regression analysis was used to obtain cross-validated parameters of QoL-AGHDA-based utilities. Two models were developed (simple and full versions). The simple version used the EQ-5Dindex (derived from European values) as the dependent variable, and age, sex, and QoL-AGHDA score as independent variables in a regression analysis. The full model utilized all available demographic information. The QoL-AGHDA scores were thus transformed into a single score (0-1), corresponding to the QoL-AGHDA-based utility. Results: The simple transformation algorithm was U (QoL-AGHDA-based utilities) = 1.05 - 0.0189 x QoL-AGHDA score - 0.00238 x age - 0.0127 x sex (male = 0; female = 1). The mean of the weighted estimate for the population (n =1752) was 0.85 (SD 0.10). The estimate for men (n = 861; mean 0.86; SD 0.10) was higher (P < 0.001) than for women (n = 891; mean 0.84; SD 0.10). Conclusion: For practical reasons, the simple model can be recommended for deriving utilities directly from the QoL-AGHDA for the Swedish population.

Place, publisher, year, edition, pages
2007. Vol. 10, no 1, 73-81 p.
Keyword [en]
EQ-5D, growth hormone deficiency in adults, pharmacoeconomic evaluations, QoL-AGHDA, quality of life, utilities
National Category
Medical and Health Sciences
Identifiers
URN: urn:nbn:se:uu:diva-96561DOI: 10.1111/j.1524-4733.2006.00146.xISI: 000243756600009PubMedID: 17261118OAI: oai:DiVA.org:uu-96561DiVA: diva2:171176
Available from: 2007-12-17 Created: 2007-12-17 Last updated: 2017-12-14Bibliographically approved
In thesis
1. Quality of Life in Adult Patients with Growth Hormone Deficiency: Bridging the gap between clinical evaluation and health economic assessment
Open this publication in new window or tab >>Quality of Life in Adult Patients with Growth Hormone Deficiency: Bridging the gap between clinical evaluation and health economic assessment
2007 (English)Doctoral thesis, comprehensive summary (Other academic)
Abstract [en]

The goals of this thesis are to evaluate quality of life (QoL) in adult patients with growth hormone deficiency (GHD) in relation to population normative data, to construct a preference-weighted index (utility) from a disease-specific QoL measure and to assess it in a clinical context.

The study included samples from the general population and patients with GHD from four European populations: England & Wales, the Netherlands, Spain and Sweden. The country-specific patient cohorts were retrieved from KIMS (Pfizer International Metabolic Database).

A questionnaire was developed that contained items from existing QoL questionnaires including, among others, Quality of Life Assessment in Growth Hormone Deficiency in Adults (QoL-AGHDA) and the EQ-5D. The QoL-AGHDA is a disease-specific measure for use in adults with GHD. The EQ-5D is a generic instrument which describes health states for which country-specific preference-based weights are available. Thus, it was possible to generate preference-weighted indices (utilities) based on data generated by both instruments.

This thesis reports QoL-AGHDA normative values for the populations of England & Wales, the Netherlands, Spain and Sweden, and confirms the extent of QoL impairment in patients with GHD in comparison with the general population. Long-term GH replacement resulted in sustained improvements in overall QoL towards normative country-specific values, as well in most of the dimensions that were impaired before treatment.

For use in health economic evaluations, models for generating utilities (QoL-AGHDAutility) from QoL-AGHDA were developed. It is believed that these models may facilitate medical decision making, given that they provide a tool for obtaining utilities in the absence of directly collected preference-weighted indices.

QoL-AGHDAutility effectively monitored treatment effects in patients with GHD. Moreover, this study confirmed a QoL-AGHDAutility deficit before treatment and a gain after starting GH replacement.

The novel aspect of the present approach was to apply preference-weighted indices derived from a disease-specific measure to assess QoL in the clinical context, together with patient demographic and clinical characteristics. The robustness of this analysis is reinforced by the fact that utilities in both general and patient populations were generated using the same methodology.

Place, publisher, year, edition, pages
Uppsala: Acta Universitatis Upsaliensis, 2007. 123 p.
Series
Digital Comprehensive Summaries of Uppsala Dissertations from the Faculty of Pharmacy, ISSN 1651-6192 ; 66
Keyword
Health policy and health economics, growth hormone deficiency in adults, quality of life, cost-utility analysis, growth hormone replacement, normative data, QoL-AGHDA, Hälsopolitik och hälsoekonomi
Identifiers
urn:nbn:se:uu:diva-8353 (URN)978-91-554-7052-4 (ISBN)
Public defence
2008-01-18, B21, BMC, Husarg 3, Uppsala, 13:15
Opponent
Supervisors
Available from: 2007-12-17 Created: 2007-12-17Bibliographically approved

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