Abstract
Objective
This study aimed to develop a mapping function that links the acromegaly quality of life (AcroQoL) questionnaire to EQ-5D-5L to obtain a preference-based utility value to inform economic evaluation.
Methods
A nationwide cross-sectional questionnaire survey among patients with acromegaly was conducted online in China during 17 December 2019 to 6 January 2020. The study sample was randomly divided into a training set and a validation set. Ordinary least squares (OLS), Tobit, beta-based mixture, and adjusted limited dependent variable mixture models were tested for development of the function in the training set. Total and subscale scores and individual items of AcroQoL were included as predictors in the models along with their squared terms and demographic and clinical characteristics, and selected by backward stepwise selection. The root mean square error, mean absolute error, Akaike’s information criterion, Bayesian information criterion and adjusted R-square were used to assess goodness of fit and predictive ability of the models.
Results
There were 424 adult patients with acromegaly eligible for this analysis. Average EQ-5D-5L index score and AcroQoL score for them was 0.82 (SD = 0.15) and 44.3 (SD = 22.9), respectively. A total of 60 candidate models were tested. Considering model simplicity and predictive ability in both training and validation set, the best model was the OLS model using scores of physical dimension and its square term as predictors.
Conclusion
A validated mapping function was developed in this study for estimating EQ-5D scores using AcroQoL outcomes. Its external validity can be further tested in other population with Acromegaly.
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Acknowledgements
The authors would like to thank all the patients with acromegaly from the China Acromegaly Patient Association (CAPA) for participating in this survey and sharing their points of view on quality of life. We thank the doctors and nurses in CAPA for giving routine online lectures and providing help and care to the patients and their families. We thank the Illness Challenge Foundation and China Alliance of Rare Diseases for their support in the survey.
Funding
This work was supported by the National Key Research and Development Program of China (Grant no. 2016YFC0901500) and the Center for Rare Diseases Research, Chinese Academy of Medical Sciences, Beijing, China (Grant no. 2016ZX310174-4).
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KW, XG, SY, BX, SZ, and DD designed the study; KW, SY, HZ, and DD performed the survey and collected responses; BX, HZ, SZ and DD supervised the implementation and quality control of the survey; KW performed the statistical analysis; KW and XG prepared the manuscript; BX, SZ, and DD revised the manuscript; All authors approved the final version of the manuscript.
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Ethics approval has been obtained from Survey and Behavioral Research Ethics committee of the Chinese University of Hong Kong (SBRE-18-268) and the Ethics Committee of Peking Union Medical College Hospital (SK-814) prior to commencement of the study. Informed consents were obtained from the participants before the survey.
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Wang, K., Guo, X., Yu, S. et al. Mapping of the acromegaly quality of life questionnaire to ED-5D-5L index score among patients with acromegaly. Eur J Health Econ 22, 1381–1391 (2021). https://doi.org/10.1007/s10198-021-01318-9
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DOI: https://doi.org/10.1007/s10198-021-01318-9