How does a preference-based generic health-related quality of life measure perform in patients with a rare disease? Measurement properties of the EQ-5D-Y proxy version among underage patients with neurofibromatosis type 1
Abstract Background EQ-5D-Y is a pediatric preference-based health-related quality of life (HRQL) measure that is recommended in health economic evaluation according to China’s guidelines. However, there is limited evidence regarding how the EQ-5D-Y perform in patients with rare diseases in the coun...
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| Format: | Article |
| Language: | English |
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BMC
2025-02-01
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| Series: | Orphanet Journal of Rare Diseases |
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| Online Access: | https://doi.org/10.1186/s13023-025-03590-x |
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| author | Wanxian Liang Shihuan Cao Yusi Suo Lining Zhang Lujia Yang Hanfei Wang Han Wang Xuejing Jin |
| author_facet | Wanxian Liang Shihuan Cao Yusi Suo Lining Zhang Lujia Yang Hanfei Wang Han Wang Xuejing Jin |
| author_sort | Wanxian Liang |
| collection | DOAJ |
| description | Abstract Background EQ-5D-Y is a pediatric preference-based health-related quality of life (HRQL) measure that is recommended in health economic evaluation according to China’s guidelines. However, there is limited evidence regarding how the EQ-5D-Y perform in patients with rare diseases in the country. Neurofibromatosis type 1 (NF1) is a rare disease that affects the growth and development of underage patients. This study aimed to examine the performance of EQ-5D-Y proxy version among underage NF1 patients in China. Methods Data from a nationwide cross-sectional survey from Nov 2022 to Jan 2023 was used. A total of 154 caregivers for underage NF1 patients who completed the EQ-5D-Y proxy version, PedsQL 4.0 Generic Core Scales (PedsQL GCS) proxy version, and Zarit Burden Interview (ZBI-22) were included. The performance of the EQ-5D-Y was assessed by response pattern (ceiling and floor effects), convergent validity against the PedsQL GCS, known-groups validity, and Shannon (H’) and Shannon evenness (J’) indices. Result Data from 154 caregivers were analyzed. The mean age of caregivers was 38.23 (6.02) years, and 78.57% of them were mothers of NF1 patients. The mean age of NF1 patients was 8.38 (3.34) years, with 51.30% being females. The ceiling effect of EQ-5D-Y was 30.52%, and floor effect was 0%. Moderate to strong correlations were found between EQ-5D-Y and PedsQL GCS dimensions that share similar constructs (rho − 0.42 to -0.60, all p values < 0.001). The hypotheses of known-groups defined by different PedsQL GCS and ZBI scores were validated. The EQ-5D-Y exhibited the strongest informativity and discriminatory power of the “feeling worried, sad or unhappy” dimension and weakest for the “mobility” dimension (H’(mobility) = 0.60, J’(mobility) = 0.38; H’(feeling worried, sad or unhappy) = 1.23, J’(feeling worried, sad or unhappy) = 0.78). Conclusions EQ-5D-Y is acceptable for measuring HRQL of underage NF1 patients in China. More evidence for using EQ-5D-Y in rare diseases is awaited. |
| format | Article |
| id | doaj-art-12798ee2f22f4b7ca383fcbbcaababea |
| institution | DOAJ |
| issn | 1750-1172 |
| language | English |
| publishDate | 2025-02-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj-art-12798ee2f22f4b7ca383fcbbcaababea2025-08-20T03:01:00ZengBMCOrphanet Journal of Rare Diseases1750-11722025-02-0120111110.1186/s13023-025-03590-xHow does a preference-based generic health-related quality of life measure perform in patients with a rare disease? Measurement properties of the EQ-5D-Y proxy version among underage patients with neurofibromatosis type 1Wanxian Liang0Shihuan Cao1Yusi Suo2Lining Zhang3Lujia Yang4Hanfei Wang5Han Wang6Xuejing Jin7Center for Evidence-Based Chinese Medicine, School of Traditional Chinese Medicine, Beijing University of Chinese MedicineCenter for Evidence-Based Chinese Medicine, School of Traditional Chinese Medicine, Beijing University of Chinese MedicineCenter for Evidence-Based Chinese Medicine, School of Traditional Chinese Medicine, Beijing University of Chinese MedicineCenter for Evidence-Based Chinese Medicine, School of Traditional Chinese Medicine, Beijing University of Chinese MedicineCenter for Evidence-Based Chinese Medicine, School of Traditional Chinese Medicine, Beijing University of Chinese MedicineCenter for Evidence-Based Chinese Medicine, School of Traditional Chinese Medicine, Beijing University of Chinese MedicineCenter for Evidence-Based Chinese Medicine, School of Traditional Chinese Medicine, Beijing University of Chinese MedicineCenter for Evidence-Based Chinese Medicine, School of Traditional Chinese Medicine, Beijing University of Chinese MedicineAbstract Background EQ-5D-Y is a pediatric preference-based health-related quality of life (HRQL) measure that is recommended in health economic evaluation according to China’s guidelines. However, there is limited evidence regarding how the EQ-5D-Y perform in patients with rare diseases in the country. Neurofibromatosis type 1 (NF1) is a rare disease that affects the growth and development of underage patients. This study aimed to examine the performance of EQ-5D-Y proxy version among underage NF1 patients in China. Methods Data from a nationwide cross-sectional survey from Nov 2022 to Jan 2023 was used. A total of 154 caregivers for underage NF1 patients who completed the EQ-5D-Y proxy version, PedsQL 4.0 Generic Core Scales (PedsQL GCS) proxy version, and Zarit Burden Interview (ZBI-22) were included. The performance of the EQ-5D-Y was assessed by response pattern (ceiling and floor effects), convergent validity against the PedsQL GCS, known-groups validity, and Shannon (H’) and Shannon evenness (J’) indices. Result Data from 154 caregivers were analyzed. The mean age of caregivers was 38.23 (6.02) years, and 78.57% of them were mothers of NF1 patients. The mean age of NF1 patients was 8.38 (3.34) years, with 51.30% being females. The ceiling effect of EQ-5D-Y was 30.52%, and floor effect was 0%. Moderate to strong correlations were found between EQ-5D-Y and PedsQL GCS dimensions that share similar constructs (rho − 0.42 to -0.60, all p values < 0.001). The hypotheses of known-groups defined by different PedsQL GCS and ZBI scores were validated. The EQ-5D-Y exhibited the strongest informativity and discriminatory power of the “feeling worried, sad or unhappy” dimension and weakest for the “mobility” dimension (H’(mobility) = 0.60, J’(mobility) = 0.38; H’(feeling worried, sad or unhappy) = 1.23, J’(feeling worried, sad or unhappy) = 0.78). Conclusions EQ-5D-Y is acceptable for measuring HRQL of underage NF1 patients in China. More evidence for using EQ-5D-Y in rare diseases is awaited.https://doi.org/10.1186/s13023-025-03590-xEQ-5D-YMeasurement propertiesNeurofibromatosis type 1Health-related quality of lifeRare diseases |
| spellingShingle | Wanxian Liang Shihuan Cao Yusi Suo Lining Zhang Lujia Yang Hanfei Wang Han Wang Xuejing Jin How does a preference-based generic health-related quality of life measure perform in patients with a rare disease? Measurement properties of the EQ-5D-Y proxy version among underage patients with neurofibromatosis type 1 Orphanet Journal of Rare Diseases EQ-5D-Y Measurement properties Neurofibromatosis type 1 Health-related quality of life Rare diseases |
| title | How does a preference-based generic health-related quality of life measure perform in patients with a rare disease? Measurement properties of the EQ-5D-Y proxy version among underage patients with neurofibromatosis type 1 |
| title_full | How does a preference-based generic health-related quality of life measure perform in patients with a rare disease? Measurement properties of the EQ-5D-Y proxy version among underage patients with neurofibromatosis type 1 |
| title_fullStr | How does a preference-based generic health-related quality of life measure perform in patients with a rare disease? Measurement properties of the EQ-5D-Y proxy version among underage patients with neurofibromatosis type 1 |
| title_full_unstemmed | How does a preference-based generic health-related quality of life measure perform in patients with a rare disease? Measurement properties of the EQ-5D-Y proxy version among underage patients with neurofibromatosis type 1 |
| title_short | How does a preference-based generic health-related quality of life measure perform in patients with a rare disease? Measurement properties of the EQ-5D-Y proxy version among underage patients with neurofibromatosis type 1 |
| title_sort | how does a preference based generic health related quality of life measure perform in patients with a rare disease measurement properties of the eq 5d y proxy version among underage patients with neurofibromatosis type 1 |
| topic | EQ-5D-Y Measurement properties Neurofibromatosis type 1 Health-related quality of life Rare diseases |
| url | https://doi.org/10.1186/s13023-025-03590-x |
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