【摘要】：肝豆?fàn)詈俗冃允且环N常染色體隱性遺傳的銅代謝障礙性疾病,在世界范圍內(nèi)較公認(rèn)的患病率為1/100000~1/30000,致病基因攜帶率為1/90,屬于罕見病范疇。肝豆?fàn)詈俗冃砸彩巧贁?shù)幾種存在有效治療方法的遺傳性疾病之一,但尚無治愈方法。該病一經(jīng)診斷需終身治療,可給患者及其家庭帶來長(zhǎng)期的疾病經(jīng)濟(jì)負(fù)擔(dān)。這一罕見病的經(jīng)濟(jì)背景過去未知,數(shù)據(jù)稀缺。目的:研究肝豆?fàn)詈俗冃曰颊吣昶骄膊〗?jīng)濟(jì)負(fù)擔(dān)及其影響因素。方法:采用回顧性問卷調(diào)查研究設(shè)計(jì),對(duì)上海交通大學(xué)附屬第一人民醫(yī)院神經(jīng)內(nèi)科2012年之后收治的肝豆?fàn)詈俗冃曰颊哌M(jìn)行問卷調(diào)查收集并計(jì)算2013年10月至2014年10月1年期間疾病經(jīng)濟(jì)負(fù)擔(dān)情況。并采用多元線性回歸法分析疾病經(jīng)濟(jì)負(fù)擔(dān)的主要影響因素。結(jié)果:研究總共調(diào)查了73名肝豆?fàn)詈俗冃缘幕颊?年平均疾病經(jīng)濟(jì)負(fù)擔(dān)總計(jì)為14879.2元(范圍349.3元-67458.2元)。其中直接醫(yī)療相關(guān)疾病經(jīng)濟(jì)負(fù)擔(dān)為11793.1元,占總開支的79.3%;直接非醫(yī)療相關(guān)疾病經(jīng)濟(jì)負(fù)擔(dān)為1204元;間接疾病經(jīng)濟(jì)負(fù)擔(dān)為1882.2元。多元線性回歸分析結(jié)果顯示對(duì)于年總費(fèi)用,住院天數(shù)的標(biāo)準(zhǔn)系數(shù)為0.674(P=0.000),排銅藥物數(shù)量的標(biāo)準(zhǔn)系數(shù)是0.188(P=0.019);對(duì)于直接醫(yī)療相關(guān)費(fèi)用,住院天數(shù)的標(biāo)準(zhǔn)系數(shù)為0.650(P=0.000),排銅藥物數(shù)量的標(biāo)準(zhǔn)系數(shù)是0.227(P=0.006);對(duì)于間接疾病經(jīng)濟(jì)負(fù)擔(dān),住院天數(shù)的標(biāo)準(zhǔn)系數(shù)為0.346(P=0.005)。結(jié)論:肝豆?fàn)詈俗冃曰颊呒膊〗?jīng)濟(jì)負(fù)擔(dān)較重,主要來源于直接醫(yī)療相關(guān)支出。年總疾病經(jīng)濟(jì)負(fù)擔(dān)及直接醫(yī)療經(jīng)濟(jì)負(fù)擔(dān)與住院天數(shù)及排銅藥物數(shù)量相關(guān),疾病間接經(jīng)濟(jì)負(fù)擔(dān)與住院天數(shù)相關(guān)。本研究在初步了解我國WD患者經(jīng)濟(jì)負(fù)擔(dān)現(xiàn)狀的基礎(chǔ)上,為制定相關(guān)疾病防治策略提供基線數(shù)據(jù),為今后干預(yù)項(xiàng)目的衛(wèi)生政策評(píng)估、新治療方案、對(duì)病人及家屬的資助計(jì)劃以及今后費(fèi)用研究設(shè)計(jì)的提供信息來源。
[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.
【學(xué)位授予單位】：上海交通大學(xué)
【學(xué)位級(jí)別】：博士
【學(xué)位授予年份】：2015
【分類號(hào)】：R742.4

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本文編號(hào)：2206611