uu.seUppsala University Publications
Change search
CiteExportLink to record
Permanent link

Direct link
Cite
Citation style
  • apa
  • ieee
  • modern-language-association
  • vancouver
  • Other style
More styles
Language
  • de-DE
  • en-GB
  • en-US
  • fi-FI
  • nn-NO
  • nn-NB
  • sv-SE
  • Other locale
More languages
Output format
  • html
  • text
  • asciidoc
  • rtf
Growth hormone replacement in hypopituitary adults with growth hormone deficiency evaluated by a utility-weighted quality of life index: a precursor to cost-utility analysis
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.
Uppsala University, Disciplinary Domain of Medicine and Pharmacy, Faculty of Medicine, Department of Women's and Children's Health. (Barnendokrinologisk forskning/Gustafsson)
2007 (English)In: Clinical Endocrinology, ISSN 0300-0664, E-ISSN 1365-2265, Vol. 68, no 1, 122-129 p.Article in journal (Refereed) Published
Abstract [en]

Objectives To examine quality of life (QoL) measured by a utility-weighted index in GH-deficient adults on GH replacement and analyse the impact of demographic and clinical characteristics on changes in utilities during treatment. Design Utilities for items in the QoL-Assessment of Growth Hormone Deficiency in Adults (QoL-AGHDAutility) were estimated based on data obtained from the general population in England and Wales (E&W). These estimates were used to calculate QoL changes in GH-treated patients and compare these with normative population values. Patients A total of 894 KIMS patients (53% women) from E&W were followed for 1 to 6 years. Measurements QoL-AGHDAutility at baseline and at the last reported visit, total QoL-AGHDAutility gain and QoL-AGHDAutility gain per year of follow-up. Results QoL-AGHDAutility in patients before GH treatment differed from the expected population values [0·67 (SD 0·174) vs. 0·85 (SD 0-038),P < 0·0001], constituting a mean deficit of -0·19 (SD 0·168). There was a difference in the mean QoL-AGHDAutility deficit for men [-0·16 (SD 0-170)] and women [-0·21 (SD 0-162)] (P < 0·001). The main improvement occurred during the first year of treatment [reduction of a deficit to -0·07 (SD 0·163) (P < 0·001) in the total cohort]; however, patients' utilities remained lower than those recorded for the general population during subsequent follow-up (P < 0·001). Despite an observed impact of age, primary aetiology, disease onset and comorbidities on QoL-AGHDAutility, all patients showed a similar beneficial response to treatment. Conclusions QoL-AGHDAutility efficiently monitors treatment effects in patients with GHD. The study confirmed the QoL-AGHDAutility deficit before treatment and a similar QoL-AGHDAutility gain observed after commencement of GH replacement in all patients.

Place, publisher, year, edition, pages
2007. Vol. 68, no 1, 122-129 p.
Keyword [en]
Corporal biometry, Pituitary diseases, Adenohypophyseal hormone, Human, Endocrinology, Health economy, Cost analysis, Precursor, Index, Quality of life, Body weight, Deficiency, Endocrinopathy, Adult, Hypophyseal insufficiency, Hormone therapy, Replacement therapy, Somatotropin
National Category
Pharmaceutical Sciences
Identifiers
URN: urn:nbn:se:uu:diva-96562DOI: 10.1111/j.1365-2265.2007.03010.xISI: 000252532800020PubMedID: 17803700OAI: oai:DiVA.org:uu-96562DiVA: diva2:171177
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

Open Access in DiVA

No full text

Other links

Publisher's full textPubMed
By organisation
Department of PharmacyDepartment of Women's and Children's Health
In the same journal
Clinical Endocrinology
Pharmaceutical Sciences

Search outside of DiVA

GoogleGoogle Scholar

doi
pubmed
urn-nbn

Altmetric score

doi
pubmed
urn-nbn
Total: 531 hits
CiteExportLink to record
Permanent link

Direct link
Cite
Citation style
  • apa
  • ieee
  • modern-language-association
  • vancouver
  • Other style
More styles
Language
  • de-DE
  • en-GB
  • en-US
  • fi-FI
  • nn-NO
  • nn-NB
  • sv-SE
  • Other locale
More languages
Output format
  • html
  • text
  • asciidoc
  • rtf