Richard Huan Xu1, Dong Dong2,3,4,5, Nan Luo6, Eliza Lai-Yi Wong7,8, Renchi Yang9, Junshuai Liu10, Huiqin Yuan11, Shuyang Zhang12. 1. Department of Rehabilitation Sciences, The Hong Kong Polytechnic University, Hong Kong SAR, China. 2. JC School of Public Health and Primary Care, The Chinese University of Hong Kong, Hong Kong SAR, China. dongdong@cuhk.edu.hk. 3. Centre for Health Systems and Policy Research, JC School of Public Health and Primary Care, The Chinese University of Hong Kong, Hong Kong SAR, China. dongdong@cuhk.edu.hk. 4. Shenzhen Research Institute, The Chinese University of Hong Kong, Shenzhen, Guangdong, China. dongdong@cuhk.edu.hk. 5. 4/F School of Public Health, The Chinese University of Hong Kong, Prince of Wales Hospital, Shatin, NT, Hong Kong SAR, China. dongdong@cuhk.edu.hk. 6. Saw Swee Hock School of Public Health, National University of Singapore, Singapore, Singapore. 7. JC School of Public Health and Primary Care, The Chinese University of Hong Kong, Hong Kong SAR, China. 8. Centre for Health Systems and Policy Research, JC School of Public Health and Primary Care, The Chinese University of Hong Kong, Hong Kong SAR, China. 9. Thrombosis and Hemostasis Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China. 10. Beijing Society of Rare Disease Clinical Care and Accessibility, Beijing, China. 11. School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing, Jiangsu, China. 12. Department of Cardiology, Peking Union Medical College Hospital, Peking Union Medical College and Chinese Academy of Medical Science, Beijing, 100730, China. shuyangzhang103@nrdrs.org.
Abstract
OBJECTIVE: This study's objective was to develop an algorithm that mapping the Haem-A-QoL scores to EQ-5D-5L utility scores in patients with hemophilia in China. METHODS: A national sample of 862 patients with hemophilia completed both the EQ-5D-5L and Haem-A-QoL instruments. Eight regression models were selected to develop the mapping algorithm, they were: the ordinary least squares, general linear regression, Tobit regression, censored least absolute deviation, mixture beta regression, adjusted limited dependent variable mixture, the two-part, and robust MM-estimator model. Root mean squared error (RMSE), mean absolute error (MAE), and R-square (R2) calculated using the tenfold cross-validation and random sample validation methods were used to assess the predictive ability of the models. RESULTS: Based on RMSE, MAE, and R2, the mixture beta regression model with selected Haem-A-QoL subscale scores as the predicted variables showed the best performance. CONCLUSIONS: Our mapping algorithm bolsters the calculation of QALYs while conducting an economic evaluation of hemophilia-related interventions when only Haem-A-QoL data are available. The external validity of the algorithm should be further assessed in the other populations.
OBJECTIVE: This study's objective was to develop an algorithm that mapping the Haem-A-QoL scores to EQ-5D-5L utility scores in patients with hemophilia in China. METHODS: A national sample of 862 patients with hemophilia completed both the EQ-5D-5L and Haem-A-QoL instruments. Eight regression models were selected to develop the mapping algorithm, they were: the ordinary least squares, general linear regression, Tobit regression, censored least absolute deviation, mixture beta regression, adjusted limited dependent variable mixture, the two-part, and robust MM-estimator model. Root mean squared error (RMSE), mean absolute error (MAE), and R-square (R2) calculated using the tenfold cross-validation and random sample validation methods were used to assess the predictive ability of the models. RESULTS: Based on RMSE, MAE, and R2, the mixture beta regression model with selected Haem-A-QoL subscale scores as the predicted variables showed the best performance. CONCLUSIONS: Our mapping algorithm bolsters the calculation of QALYs while conducting an economic evaluation of hemophilia-related interventions when only Haem-A-QoL data are available. The external validity of the algorithm should be further assessed in the other populations.
Authors: Jamie O'Hara; Shaun Walsh; Charlotte Camp; Giuseppe Mazza; Liz Carroll; Christina Hoxer; Lars Wilkinson Journal: Health Qual Life Outcomes Date: 2018-05-02 Impact factor: 3.186