色素性视网膜炎对日本患者生活质量和经济影响的非介入性横断面研究。
Quality of Life and Economic Impacts of Retinitis Pigmentosa on Japanese Patients: A Non-interventional Cross-sectional Study.
机构信息
Ophthalmology Medical Franchise Department, Novartis Pharma K.K., Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo, 105-6333, Japan.
HEOR Group, Novartis Pharma K.K., Tokyo, Japan.
出版信息
Adv Ther. 2023 May;40(5):2375-2393. doi: 10.1007/s12325-023-02446-9. Epub 2023 Mar 22.
INTRODUCTION
Retinitis pigmentosa (RP) is an inherited progressive disease, characterized by a loss of photoreceptors, and is the second leading cause of visual impairment in Japan. RP is currently incurable and can result in complete blindness, with affected patients typically experiencing a gradual loss of light sensitivity, visual field, and visual acuity. Identification of any unmet medical needs of patients with this condition requires an understanding of the impacts of RP; in this study, we surveyed Japanese patients with RP to investigate the quality of life and economic impacts of visual impairment.
METHODS
This non-interventional, cross-sectional study surveyed Japanese patients with RP. Economic impact was measured using an original questionnaire that assessed out-of-pocket cost (e.g., vision aids and medical services), salary gap with the general public, and the cost of depression and anxiety. Worker productivity was assessed using the Work Productivity and Activity Impairment Questionnaire (WPAI). Quality of life was evaluated using the Health Utilities Index Mark 3 (HUI3), the National Eye Institute Visual Function Questionnaire-25 (VFQ-25), and the 5-level EQ-5D version (EQ-5D-5L). The primary outcome was direct and indirect costs of visual impairment or blindness during the lifetime of patients with RP.
RESULTS
Among 122 surveyed patients with RP, the estimated annual cost per patient was 218,520 yen (2176 USD), and the estimated lifetime cost per patient was 18,523,909 yen (184,501 USD). Additional robustness testing increased the estimated annual cost and lifetime cost per patient to 783,176 yen (7801 USD) and 66,389,827 yen (661,253 USD), respectively. In working patients, work productivity loss was 26.2% per person and impairment of daily activities was 31.6% per person. The mean VFQ-25, HUI3, and EQ-5D-5L scores were 42.0, 0.393, and 0.833, respectively.
CONCLUSION
RP imposed a heavy economic burden and negative quality of life impacts in Japanese patients.
简介
色素性视网膜炎（RP）是一种遗传性进行性疾病，其特征是感光细胞丧失，是日本第二大致盲原因。RP 目前无法治愈，可导致完全失明，受影响的患者通常会逐渐丧失对光的敏感性、视野和视力。为了确定患有这种疾病的患者的任何未满足的医疗需求，需要了解 RP 的影响；在这项研究中，我们调查了日本 RP 患者，以调查视力障碍对他们的生活质量和经济的影响。
方法
这项非干预性、横断面研究调查了日本 RP 患者。经济影响通过使用原始问卷进行评估，该问卷评估了自付费用（例如视力辅助器具和医疗服务）、与公众的工资差距以及抑郁和焦虑的成本。通过工作生产力问卷（WPAI）评估工人生产力。使用健康效用指数标记 3 （HUI3）、国家眼科研究所视觉功能问卷-25（VFQ-25）和 5 级 EQ-5D 版本（EQ-5D-5L）评估生活质量。主要结局是 RP 患者一生中视力障碍或失明的直接和间接成本。
结果
在 122 名接受调查的 RP 患者中，每位患者的年估计费用为 218,520 日元（2176 美元），每位患者的终身估计费用为 18,523,909 日元（184,501 美元）。额外的稳健性测试将每位患者的年估计费用和终身估计费用分别提高到 783,176 日元（7801 美元）和 66,389,827 日元（661,253 美元）。在工作患者中，每个人的工作生产力损失为 26.2%，日常活动受损为 31.6%。VFQ-25、HUI3 和 EQ-5D-5L 的平均得分分别为 42.0、0.393 和 0.833。
结论
RP 给日本患者带来了沉重的经济负担和负面的生活质量影响。
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