我国复发套细胞淋巴瘤年均经济负担分析
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摘要
目的:通过对我国复发套细胞淋巴瘤(Mantle Cell Lymphoma, MCL)患者的年均直接医疗成本、直接非医疗成本和间接成本进行定量研究,评估该疾病目前临床常规治疗方案(以化疗为基础的治疗方案,包括常规化疗和免疫化疗)的年均经济负担,为临床优选合理治疗方案和促进卫生资源的合理配置提供科学依据和政策建议。方法 :通过系统性文献综述以确定已公开发表的MCL疾病的流行病学数据、 MCL患者的经济负担数据以及生命质量。并通过在中国五个城市的专家咨询会(Delphi Panel)收集真实世界中采用目前临床上的常规治疗方案治疗的复发MCL患者年均的直接医疗成本、直接非医疗成本和间接成本数据并进行描述性统计分析。最后通过Microsoft Excel 2013构建年均经济负担模型对复发MCL患者的年均经济负担进行测算,并对关键参数进行单因素敏感性分析。结果 :对我国5个代表性城市的22家三甲医院31位血液科和肿瘤科(副主任以上)的临床专家进行调研显示,采用目前临床上的常规治疗方案治疗复发MCL患者的年均总成本为478,286元,其中年均直接医疗成本为390,052元,年均直接非医疗成本为22,045元,年均间接成本为66,189元。单因素敏感性分析显示,影响复发MCL患者的年均经济负担最大的两个因素是化疗相关的支持性用药花费和化疗相关的年均住院次数。结论 :套细胞淋巴瘤给社会及患者造成沉重的经济负担,政府和医疗机构应引起重视并采取合理的政策和措施降低套细胞淋巴瘤患者的经济负担,减轻化疗相关的花费极为重要。鼓励给药途径简单、疗效更好、安全性更高的创新靶向药物的开发和使用。
Objectives: Mantle cell leukemia is a rare, incurable, relapse disease, and life-threatening disease. Chemo-based therapy, although documented with a poor response, rapid progress, severe adverse events and only 1-2 years' median overall survival in relapse/refractory mantle cell leukemia(rrMCL) patients by multiple trials, were still the most common treatment in rrMCL patients in present clinical practice, due to the fact that lack of better treatment choices. This study aims to evaluate the economic burden(including direct and indirect cost) of chemobased therapy in rrMCL patients in China from societal perspective. Methods: A comprehensive literature review was performed to collect the published disease burden data on rrMCL patients. Questionnaires were designed to collect relevant clinical and costs information which not exist in published literature by 5 Delphi panels, conducted with hematologists and oncologists to document the treatment burden. MCL disease burden model is built via Microsoft Excel 2013 to assess the economic burden of MCL. One-way sensitive analysis was performed.Results: Total 31 hematologists and oncologists from Hangzhou, Wuhan, Xi'an, Guangzhou and Chengdu participated in Delphi panels. The average total annual cost per patients were 478,286 CNY, with the annual direct medical cost of 390,052 CNY, the direct non-medical cost was 22,045 CNY, and the annual indirect cost was 66,189 CNY. Sensitivity analysis shows that chemotherapy-related supportive drug cost and annual frequency of inpatient care are the factors which impact the result most.Conclusion: Chemo-based therapy, although clinically efficacious, imposing significant economic burden in China. This study highlights the critical need to introduce innovative therapies with better clinical efficacies, longer survival, reduced total cost and a simple admission for rrMCL patients in China.
Objectives: Mantle cell leukemia is a rare, incurable, relapse disease, and life-threatening disease. Chemo-based therapy, although documented with a poor response, rapid progress, severe adverse events and only 1-2 years' median overall survival in relapse/refractory mantle cell leukemia(rrMCL) patients by multiple trials, were still the most common treatment in rrMCL patients in present clinical practice, due to the fact that lack of better treatment choices. This study aims to evaluate the economic burden(including direct and indirect cost) of chemobased therapy in rrMCL patients in China from societal perspective. Methods: A comprehensive literature review was performed to collect the published disease burden data on rrMCL patients. Questionnaires were designed to collect relevant clinical and costs information which not exist in published literature by 5 Delphi panels, conducted with hematologists and oncologists to document the treatment burden. MCL disease burden model is built via Microsoft Excel 2013 to assess the economic burden of MCL. One-way sensitive analysis was performed.Results: Total 31 hematologists and oncologists from Hangzhou, Wuhan, Xi'an, Guangzhou and Chengdu participated in Delphi panels. The average total annual cost per patients were 478,286 CNY, with the annual direct medical cost of 390,052 CNY, the direct non-medical cost was 22,045 CNY, and the annual indirect cost was 66,189 CNY. Sensitivity analysis shows that chemotherapy-related supportive drug cost and annual frequency of inpatient care are the factors which impact the result most.Conclusion: Chemo-based therapy, although clinically efficacious, imposing significant economic burden in China. This study highlights the critical need to introduce innovative therapies with better clinical efficacies, longer survival, reduced total cost and a simple admission for rrMCL patients in China.
引文
[1]谢彦,朱军.套细胞淋巴瘤诊断与治疗中国专家共识(2016版)解读[J].临床血液学杂志,2017(5):683-686.
[2]Herrmann A,Hoster E,Zwingers T,et al.Improvement of Overall Survival in Advanced Stage Mantle Cell Lymphoma[J].Journal of Clinical Oncology,2009,27(4):511-518.
[3]Yang QP,Zhang WY,Yu JB,Zhao S,Xu H,Wang WY,Bi CF,Zuo Z,Wang XQ,Huang J,Dai L.Subtype distribution of lymphomas in Southwest China:analysis of 6,382 cases using WHO classification in a single institution.Diagnostic pathology.2011 Dec;6(1):77.
[4]Huh J.Epidemiologic overview of malignant lymphoma[J].The Korean journal of hematology,2012,47(2):92-104.
[5]Cortelazzo,S.,Ponzoni,M.,et al.(2012).Crit Rev Oncol Hematol.82(1):78-101.
[6]McKay,P.,M.Leach,et al.Guidelines for the investigation and management of mantle cell lymphoma.Br J Haematol.2012,159(4):405-426.
[7]J,R.,et al.,Durable responses with the metronomic rituximab and thalidomide plus prednisone,etoposide,procarbazine,and cyclophosphamide regimen in elderly patients with recurrent mantle cell lymphoma.Cancer:AJournal of the American Cancer Society,2010(11):2655-2664.
[8]Hess,G.,et al.,Health-related quality of life data from a phase 3,international,randomized,open-label,multicenter study in patients with previously treated mantle cell lymphoma treated with ibrutinib versus temsirolimus.Leuk Lymphoma,2017.58(12):2824-2832.
[9]Shin,D.Y.,et al.,Results of a phase II study of vorinostat in combination with intravenous fludarabine,mitoxantrone,and dexamethasone in patients with relapsed or refractory mantle cell lymphoma:an interim analysis.Cancer Chemother Pharmacol,2016,77(4):865-73.
[10]Senbetta,M.,Robitaille,M.N.,McKenzie,R.S.,&Lefebvre,P.(2015).Cost of treatment failure in patients with mantle cell lymphoma:results of a large US observational study.Clinical Lymphoma,Myeloma and Leukemia,15,S218.
[11]Kozma,C.,Slaton,T.,McKenzie,R.S.,Schenkel,B.,&Ellis,L.(2015).Mantle Cell Lymphoma(MCL)Treatment Patterns,Healthcare Resource Use(HRU),and Costs in the United States.Clinical Lymphoma,Myeloma and Leukemia,15,S226.
[12]Chen Q,Jain N,Ayer T,et al.Economic Burden of Chronic Lymphocytic Leukemia in the Era of Oral Targeted Therapies in the United States[J].Journal of Clinical Oncology Official Journal of the American Society of Clinical Oncology,2017,35(2):166.
[13]陈冰等,中国皖南地区728例恶性淋巴瘤的构成分析.中国临床药理学与治疗学,2013(04):388-393.
[14]李小秋等,中国淋巴瘤亚型分布:国内多中心性病例10002例分析.诊断学理论与实践,2012(02):111-115.
[15]杨雷等,恶性淋巴瘤1126例临床特点分析.白血病·淋巴瘤,2010(5):290-292.
[16]余谦,2003~2010年广西医科大学第一附属医院1371例住院淋巴瘤患者流行病学分析,2011.
[17]刘志刚与徐才刚,淋巴瘤的发病现状与危险因素.西部医学,2013(07):961-963+967.
[18]2017年居民收入和消费支出情况.国家统计局:http://www.stats.gov.cn/tjsj/zxfb/201801/t20180118_1574931.html.
[19]中国工薪阶层信贷发展报告.西南财经大学中国家庭金融调查与研究中心联合中腾信:http://chfs.swufe.edu.cn/xiangqing.aspx?id=1620.
[20]Trends in catastrophic health expenditure in India:1993 to 2014.Available from:http://www.who.int/bulletin/volumes/96/1/17-191759-ab/en/.
[2]Herrmann A,Hoster E,Zwingers T,et al.Improvement of Overall Survival in Advanced Stage Mantle Cell Lymphoma[J].Journal of Clinical Oncology,2009,27(4):511-518.
[3]Yang QP,Zhang WY,Yu JB,Zhao S,Xu H,Wang WY,Bi CF,Zuo Z,Wang XQ,Huang J,Dai L.Subtype distribution of lymphomas in Southwest China:analysis of 6,382 cases using WHO classification in a single institution.Diagnostic pathology.2011 Dec;6(1):77.
[4]Huh J.Epidemiologic overview of malignant lymphoma[J].The Korean journal of hematology,2012,47(2):92-104.
[5]Cortelazzo,S.,Ponzoni,M.,et al.(2012).Crit Rev Oncol Hematol.82(1):78-101.
[6]McKay,P.,M.Leach,et al.Guidelines for the investigation and management of mantle cell lymphoma.Br J Haematol.2012,159(4):405-426.
[7]J,R.,et al.,Durable responses with the metronomic rituximab and thalidomide plus prednisone,etoposide,procarbazine,and cyclophosphamide regimen in elderly patients with recurrent mantle cell lymphoma.Cancer:AJournal of the American Cancer Society,2010(11):2655-2664.
[8]Hess,G.,et al.,Health-related quality of life data from a phase 3,international,randomized,open-label,multicenter study in patients with previously treated mantle cell lymphoma treated with ibrutinib versus temsirolimus.Leuk Lymphoma,2017.58(12):2824-2832.
[9]Shin,D.Y.,et al.,Results of a phase II study of vorinostat in combination with intravenous fludarabine,mitoxantrone,and dexamethasone in patients with relapsed or refractory mantle cell lymphoma:an interim analysis.Cancer Chemother Pharmacol,2016,77(4):865-73.
[10]Senbetta,M.,Robitaille,M.N.,McKenzie,R.S.,&Lefebvre,P.(2015).Cost of treatment failure in patients with mantle cell lymphoma:results of a large US observational study.Clinical Lymphoma,Myeloma and Leukemia,15,S218.
[11]Kozma,C.,Slaton,T.,McKenzie,R.S.,Schenkel,B.,&Ellis,L.(2015).Mantle Cell Lymphoma(MCL)Treatment Patterns,Healthcare Resource Use(HRU),and Costs in the United States.Clinical Lymphoma,Myeloma and Leukemia,15,S226.
[12]Chen Q,Jain N,Ayer T,et al.Economic Burden of Chronic Lymphocytic Leukemia in the Era of Oral Targeted Therapies in the United States[J].Journal of Clinical Oncology Official Journal of the American Society of Clinical Oncology,2017,35(2):166.
[13]陈冰等,中国皖南地区728例恶性淋巴瘤的构成分析.中国临床药理学与治疗学,2013(04):388-393.
[14]李小秋等,中国淋巴瘤亚型分布:国内多中心性病例10002例分析.诊断学理论与实践,2012(02):111-115.
[15]杨雷等,恶性淋巴瘤1126例临床特点分析.白血病·淋巴瘤,2010(5):290-292.
[16]余谦,2003~2010年广西医科大学第一附属医院1371例住院淋巴瘤患者流行病学分析,2011.
[17]刘志刚与徐才刚,淋巴瘤的发病现状与危险因素.西部医学,2013(07):961-963+967.
[18]2017年居民收入和消费支出情况.国家统计局:http://www.stats.gov.cn/tjsj/zxfb/201801/t20180118_1574931.html.
[19]中国工薪阶层信贷发展报告.西南财经大学中国家庭金融调查与研究中心联合中腾信:http://chfs.swufe.edu.cn/xiangqing.aspx?id=1620.
[20]Trends in catastrophic health expenditure in India:1993 to 2014.Available from:http://www.who.int/bulletin/volumes/96/1/17-191759-ab/en/.