PDF(Pubmed)
Abstract：
BACKGROUND: The IMPACT survey aimed to elucidate the humanistic, clinical and economic burden of osteogenesis imperfecta (OI) on individuals with OI, their families, caregivers and wider society. Research methodology, demographics and initial insights from the survey have been previously reported. The cost of illness (healthcare resource use, productivity loss, out-of-pocket spending) and drivers of the economic impact of OI are reported here.
METHODS: IMPACT was an international mixed-methods online survey in eight languages (fielded July-September 2021) targeting adults (aged ≥ 18 years) or adolescents (aged ≥ 12-17 years) with OI, caregivers with or without OI and other close relatives. Survey domains included demographics, socioeconomic factors, clinical characteristics, treatment patterns, quality of life and health economics. The health economic domain for adults, which included questions on healthcare resource use, productivity loss and out-of-pocket spending, was summarised. Regression and pairwise analyses were conducted to identify independent drivers and associations with respondent characteristics.
RESULTS: Overall, 1,440 adults with OI responded to the survey. Respondents were mostly female (70%) and from Europe (63%) with a median age of 43 years. Within a 12-month period, adults with OI reported visiting a wide range of healthcare professionals. Two-thirds (66%) of adults visited a hospital, and one-third (33%) visited the emergency department. The mean total number of diagnostic tests undergone by adults within these 12 months was 8.0. Adults had undergone a mean total of 11.8 surgeries up to the time point of the survey. The proportions of adults using queried consumables or services over 12 months ranged from 18-82%, depending on the type of consumable or service. Most adults (58%) were in paid employment, of which nearly one-third (29%) reported missing a workday. Of the queried expenses, the mean total out-of-pocket spending in 4 weeks was €191. Respondent characteristics such as female sex, more severe self-reported OI and the experience of fractures were often associated with increased economic burden.
CONCLUSIONS: IMPACT provides novel insights into the substantial cost of illness associated with OI on individuals, healthcare systems and society at large. Future analyses will provide insights into country-specific economic impact, humanistic impact and the healthcare journey of individuals with OI.
METHODS: IMPACT was an international mixed-methods online survey in eight languages (fielded July-September 2021) targeting adults (aged ≥ 18 years) or adolescents (aged ≥ 12-17 years) with OI, caregivers with or without OI and other close relatives. Survey domains included demographics, socioeconomic factors, clinical characteristics, treatment patterns, quality of life and health economics. The health economic domain for adults, which included questions on healthcare resource use, productivity loss and out-of-pocket spending, was summarised. Regression and pairwise analyses were conducted to identify independent drivers and associations with respondent characteristics.
RESULTS: Overall, 1,440 adults with OI responded to the survey. Respondents were mostly female (70%) and from Europe (63%) with a median age of 43 years. Within a 12-month period, adults with OI reported visiting a wide range of healthcare professionals. Two-thirds (66%) of adults visited a hospital, and one-third (33%) visited the emergency department. The mean total number of diagnostic tests undergone by adults within these 12 months was 8.0. Adults had undergone a mean total of 11.8 surgeries up to the time point of the survey. The proportions of adults using queried consumables or services over 12 months ranged from 18-82%, depending on the type of consumable or service. Most adults (58%) were in paid employment, of which nearly one-third (29%) reported missing a workday. Of the queried expenses, the mean total out-of-pocket spending in 4 weeks was €191. Respondent characteristics such as female sex, more severe self-reported OI and the experience of fractures were often associated with increased economic burden.
CONCLUSIONS: IMPACT provides novel insights into the substantial cost of illness associated with OI on individuals, healthcare systems and society at large. Future analyses will provide insights into country-specific economic impact, humanistic impact and the healthcare journey of individuals with OI.
摘要：
背景：影响调查旨在阐明人文主义，成骨不全症（OI）对OI患者的临床和经济负担，他们的家人,照顾者和更广泛的社会。研究方法，先前已经报道了调查的人口统计学和初步见解。疾病成本(医疗保健资源使用，生产力损失，自付支出)和OI经济影响的驱动因素在这里报告。
方法：IMPACT是一项以八种语言进行的国际混合方法在线调查（2021年7月至9月），针对成人（≥18岁）或青少年（≥12-17岁）患有OI，有或没有OI和其他近亲的看护人。调查领域包括人口统计，社会经济因素,临床特征,治疗模式,生活质量和卫生经济学。成人的健康经济领域，其中包括有关医疗资源使用的问题，生产力损失和自付支出，被总结了。进行回归和成对分析以确定独立的驱动因素和与应答者特征的关联。
结果：总体而言，1,440名患有OI的成年人对调查做出了回应。受访者大多是女性（70％），来自欧洲（63％），平均年龄为43岁。在12个月内,患有OI的成年人报告拜访了广泛的医疗保健专业人员。三分之二（66％）的成年人去了医院，三分之一（33％）访问了急诊科。在这12个月内，成年人接受的诊断测试的平均总数为8.0。截至调查时间点，成年人平均总共进行了11.8次手术。超过12个月使用查询消耗品或服务的成年人比例为18-82%，取决于消耗品或服务的类型。大多数成年人（58％）从事有偿工作，其中近三分之一(29%)报告错过了一个工作日。在查询的费用中，4周内的平均自付总支出为191欧元。受访者的特征，如女性性别，更严重的自我报告OI和骨折的经历通常与经济负担增加相关.
结论：IMPACT提供了与OI相关的疾病对个体的巨大成本的新见解，医疗保健系统和整个社会。未来的分析将提供对特定国家经济影响的见解，人文影响和OI患者的医疗旅程。
方法：IMPACT是一项以八种语言进行的国际混合方法在线调查（2021年7月至9月），针对成人（≥18岁）或青少年（≥12-17岁）患有OI，有或没有OI和其他近亲的看护人。调查领域包括人口统计，社会经济因素,临床特征,治疗模式,生活质量和卫生经济学。成人的健康经济领域，其中包括有关医疗资源使用的问题，生产力损失和自付支出，被总结了。进行回归和成对分析以确定独立的驱动因素和与应答者特征的关联。
结果：总体而言，1,440名患有OI的成年人对调查做出了回应。受访者大多是女性（70％），来自欧洲（63％），平均年龄为43岁。在12个月内,患有OI的成年人报告拜访了广泛的医疗保健专业人员。三分之二（66％）的成年人去了医院，三分之一（33％）访问了急诊科。在这12个月内，成年人接受的诊断测试的平均总数为8.0。截至调查时间点，成年人平均总共进行了11.8次手术。超过12个月使用查询消耗品或服务的成年人比例为18-82%，取决于消耗品或服务的类型。大多数成年人（58％）从事有偿工作，其中近三分之一(29%)报告错过了一个工作日。在查询的费用中，4周内的平均自付总支出为191欧元。受访者的特征，如女性性别，更严重的自我报告OI和骨折的经历通常与经济负担增加相关.
结论：IMPACT提供了与OI相关的疾病对个体的巨大成本的新见解，医疗保健系统和整个社会。未来的分析将提供对特定国家经济影响的见解，人文影响和OI患者的医疗旅程。
