肝豆状核变性患者疾病经济负担的调查与分析

发布时间：2018-08-27 07:55

【摘要】：肝豆状核变性是一种常染色体隐性遗传的铜代谢障碍性疾病,在世界范围内较公认的患病率为1/100000~1/30000,致病基因携带率为1/90,属于罕见病范畴。肝豆状核变性也是少数几种存在有效治疗方法的遗传性疾病之一,但尚无治愈方法。该病一经诊断需终身治疗,可给患者及其家庭带来长期的疾病经济负担。这一罕见病的经济背景过去未知,数据稀缺。目的:研究肝豆状核变性患者年平均疾病经济负担及其影响因素。方法:采用回顾性问卷调查研究设计,对上海交通大学附属第一人民医院神经内科2012年之后收治的肝豆状核变性患者进行问卷调查收集并计算2013年10月至2014年10月1年期间疾病经济负担情况。并采用多元线性回归法分析疾病经济负担的主要影响因素。结果:研究总共调查了73名肝豆状核变性的患者,年平均疾病经济负担总计为14879.2元(范围349.3元-67458.2元)。其中直接医疗相关疾病经济负担为11793.1元,占总开支的79.3%;直接非医疗相关疾病经济负担为1204元;间接疾病经济负担为1882.2元。多元线性回归分析结果显示对于年总费用,住院天数的标准系数为0.674(P=0.000),排铜药物数量的标准系数是0.188(P=0.019);对于直接医疗相关费用,住院天数的标准系数为0.650(P=0.000),排铜药物数量的标准系数是0.227(P=0.006);对于间接疾病经济负担,住院天数的标准系数为0.346(P=0.005)。结论:肝豆状核变性患者疾病经济负担较重,主要来源于直接医疗相关支出。年总疾病经济负担及直接医疗经济负担与住院天数及排铜药物数量相关,疾病间接经济负担与住院天数相关。本研究在初步了解我国WD患者经济负担现状的基础上,为制定相关疾病防治策略提供基线数据,为今后干预项目的卫生政策评估、新治疗方案、对病人及家属的资助计划以及今后费用研究设计的提供信息来源。
[Abstract]:Hepatolenticular degeneration (HLD) is an autosomal recessive copper metabolic disorder. The worldwide prevalence rate is 1 / 100000U / 1 / 300000.The carrying rate of disease-causing genes is 1 / 90, which belongs to the category of rare diseases. Hepatolenticular degeneration is one of the few genetic diseases with effective treatment, but there is no cure. Once diagnosed, the disease needs life-long treatment, which can bring long-term economic burden to patients and their families. The economic background of this rare disease is unknown and data is scarce. Objective: to study the annual average economic burden of disease in patients with hepatolenticular degeneration and its influencing factors. Methods: a retrospective questionnaire survey was designed. A questionnaire survey was conducted on the patients with hepatolenticular degeneration admitted after 2012 in the Department of Neurology, the first people's Hospital affiliated to Shanghai Jiaotong University, and the economic burden of the disease was calculated from October 2013 to October 2014. The main influencing factors of economic burden of disease were analyzed by multivariate linear regression method. Results: a total of 73 patients with hepatolenticular degeneration were investigated. The average annual economic burden of disease was 14879.2 yuan (349.3 yuan-67458.2 yuan). The economic burden of direct medical care related diseases is 11793.1 yuan, accounting for 79.3% of the total expenditure; direct non-medical related diseases economic burden is 1204 yuan; indirect disease economic burden is 1882.2 yuan. The results of multiple linear regression analysis showed that the standard coefficient of hospitalization days was 0.674 (P0. 000) for the total annual cost, the standard coefficient for the quantity of copper excretion was 0.188 (P0. 019). The standard coefficient of hospitalization days was 0.650 (P0. 000), the standard coefficient of the amount of copper excretion was 0.227 (P0. 006), the standard coefficient of hospitalization days was 0.346 (P0. 005) for the economic burden of indirect diseases. Conclusion: the burden of disease in patients with hepatolenticular degeneration is heavy and comes from direct medical expenditure. The total disease economic burden and direct medical economic burden were related to the days of hospitalization and the quantity of copper excretion drugs, while the indirect economic burden of disease was related to the days of hospitalization. On the basis of preliminary understanding of the current economic burden of WD patients in China, this study provides baseline data for the formulation of relevant disease prevention and control strategies, and provides a new treatment plan for the future health policy evaluation of intervention projects. Funding programs for patients and their families and sources of information for future cost research and design.
【学位授予单位】：上海交通大学
【学位级别】：博士
【学位授予年份】：2015
【分类号】：R742.4

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