Economic evaluation of different medicines for acute myeloid leukemia: a systematic review_Chinese Journal of Evidence-Based Medicine

Authors：

LI Rui ^1,2,3 , XIAO Feiyi ^1,4 , LIU Yuanyuan ⁵ , ZOU Xinru ⁶ , FU Jiani ^6,7 , YANG Kehu ^2,3 , ZHAO Kun ¹ ,  LI Xue ¹

1. China National Health Development Research Center, Beijing 100044, P. R. China;
2. Evidence Based Social Science Research Center, School of Public Health, Lanzhou University, Lanzhou 730000, P. R. China;
3. Evidence Based Medicine Center, School of Basic Medical Sciences, Lanzhou University, Lanzhou 730000, P. R. China;
4. School of Business Administration, Shenyang Pharmaceutical University, Shenyang 110016, P. R. China;
5. The First School of Clinical Medicine, Lanzhou University, Lanzhou 730000, P. R. China;
6. The Second School of Clinical Medicine, Lanzhou University, Lanzhou 730000, P. R. China;
7. First Affiliated Hospital of Xi’an Jiaotong University, Xi’an 710061, P. R. China;

Corresponding author：

LI Xue, Email: lx0204@126.com

Keywords：

Acute myeloid leukemia; Hypomethylating agents; Targeted medicine; Economic evaluation; Systematic review

DOI：

10.7507/1672-2531.202211023

Video：

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Objective To systematically review the health economic evaluation studies of medicines for the treatment of acute myeloid leukemia (AML). Methods The PubMed, EMbase, Cochrane Library, CBM, CNKI, and WanFang Data, as well as the CRD database specifically for health economics were electronically searched from inception to June 2022, and related journals in the field of health economics and the websites of HTA institutions in various countries were manually searched. The quality of the studies was assessed using the CHEERS checklist. The basic characteristics of health economics evaluation publications were summarized, the quality of model structures and methodologies was assessed and economic evaluation results were compared among different treatments. Results A total of 17 studies were included, and cost-effectiveness analyses were conducted from the perspectives of the health system, patients, the whole society, and medical insurance payers. The economic evaluation models were relatively unified, but there were differences in methods and results reporting, and the quality needed to be improved. The research objects were mainly the comparison of hypomethylating agents, targeted medicine and traditional chemotherapy regimens, as well as the comparison of different chemotherapy combinations and different drug dosages. Conclusion Real-world studies are mainly focused on traditional chemotherapy regimens, and model-based health economic evaluations, such as Markov models, are more frequently applied to newly developed targeted drugs and demethylation drugs. Among all treatments, the chemotherapy regimens including cytarabine, midostaurin, and decitabine are found to be more cost-effective.

Citation： LI Rui, XIAO Feiyi, LIU Yuanyuan, ZOU Xinru, FU Jiani, YANG Kehu, ZHAO Kun, LI Xue. Economic evaluation of different medicines for acute myeloid leukemia: a systematic review. Chinese Journal of Evidence-Based Medicine, 2023, 23(6): 638-646. doi: 10.7507/1672-2531.202211023 Copy

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2.	应江伟, 杜灵彬, 郑艳泽, 等. 浙江省肿瘤登记地区2000~2009年白血病发病与死亡分析. 中国肿瘤, 2014, 23(7): 538-542.
3.	黄治虎, 陈宝安, 欧阳建, 等. 我国白血病流行病学调查的现状和对策. 临床血液学杂志, 2009, 22(2): 166-167.
4.	周建军, 陈睿, 王烨菁, 等. 上海市卢湾区居民2004-2011年白血病发病和死亡资料分析. 中华肿瘤防治杂志, 2017, 24(8): 507-511.
5.	斯垚翔, 钱虹, 刘玉琴, 等. 甘肃省2010年白血病发病和死亡分析. 中国肿瘤, 2015, 24(2): 81-88.
6.	董金凤. 2011-2012年福建省新发成人急性白血病的发病情况调查. 福建: 福建医科大学, 2014.
7.	刘玉琴, 赵凤菊, 陈万青, 等. 中国2009年白血病发病和死亡资料分析. 中国肿瘤, 2013, 22(7): 528-534.
8.	赵爽, 乔文亮, 邱志新, 等. 吉非替尼治疗晚期非小细胞肺癌的药物经济学系统评价. 华西医学, 2018, 33(1): 76-83.
9.	王莉, 胡善联, 孙鑫, 等. 卫生经济学分析的系统评价方法与挑战. 中国循证医学杂志, 2008, 8(11): 1007-1011.
10.	Husereau D, Drummond M, Petrou S, et al. Consolidated health economic evaluation reporting standards (CHEERS) statement. Value Health, 2013, 16(2): e1-e5.
11.	肖军, 孙谨芳, 王琦琦, 等. 卫生经济学评价报告指南及应用现状. 中华预防医学杂志, 2017, 51(3): 276-280.
12.	Huang BT, Wang Y, Du QF, et al. Analysis of efficacy and cost-effectiveness of high-dose arabinoside versus daunorubicin chemotherapy in older adult patients with acute myeloid leukemia by cytogenetic risk profile: retrospective review from China. Int J Hematol, 2011, 93(4): 474-481.
13.	Stein E, Xie J, Duchesneau E, et al. Cost effectiveness of midostaurin in the treatment of newly diagnosed FLT3-mutated acute myeloid Leukemia in the United States. Pharmacoeconomics, 2019, 37(2): 239-253.
14.	Tremblay G, Dolph M, Patel S, et al. Cost-effectiveness analysis for midostaurin versus standard of care in acute myeloid leukemia in the United Kingdom. Cost Eff Resour Alloc, 2018, 16: 33.
15.	Lambertenghi-Deliliers G, Oriani A, Pioltelli P, et al. Cost-effectiveness analysis of induction regimens containing anthracyclines in adult acute myelogenous leukemia. Leuk Lymphoma, 1991, 5(1): 33-41.
16.	Uyl-de Groot CA, Löwenberg B, Vellenga E, et al. Cost-effectiveness and quality-of-life assessment of GM-CSF as an adjunct to intensive remission induction chemotherapy in elderly patients with acute myeloid leukemia. Br J Haematol, 1998, 100(4): 629-636.
17.	Arenaza A, Diez R, Esteve J, et al. Cost-effectiveness of midostaurin in the treatment of acute myeloid leukemia with the FLT3 mutation in Spain. Clinicoecon Outcomes Res, 2019, 11: 683-694.
18.	Tremblay G, Cariou C, Recher C, et al. Cost-effectiveness of midostaurin in the treatment of newly diagnosed FLT3-mutated acute myeloid leukemia in France. Eur J Health Econ, 2020, 21(4): 543-555.
19.	Jacob LA, Aparna S, Lakshmaiah KC, et al. Decitabine compared with low-dose cytarabine for the treatment of older patients with newly diagnosed acute myeloid leukemia: a pilot study of safety, efficacy, and cost-effectiveness. Adv Hematol, 2015, 2015: 167029.
20.	Batty N, Yin Y, Wetzler M. Decitabine is more cost effective than cytarabine and daunorubicin in elderly acute myeloid leukemia patients. Journal of Cancer Research & Therapy, 2014, 2(4): 68-73.
21.	Coyle D, Villeneuve PJA. Economic evaluation of azacitidine in elderly patients with acute myeloid leukemia with high blast counts. Pharmacoecon Open, 2020, 4(2): 297-305.
22.	National Institute for Health and Care Excellence. TA399-Azacitidine for treating acute myeloid leukaemia with more than 30% bone marrow blasts. 2016.
23.	National Institute for Health and Care Excellence. TA545-Gemtuzumab ozogamicin for untreated acute myeloid leukaemia. 2018.
24.	National Institute for Health and Care Excellence. TA552-Liposomal cytarabine–daunorubicin for untreated acute myeloid leukaemia. 2018.
25.	National Institute for Health and Care Excellence. TA642-Gilteritinib for treating relapsed or refractory acute myeloid leukaemia. 2020.
26.	张伶俐, 颜建周, 袁力. 阿糖胞苷分别联合伊达比星、柔红霉素治疗急性非淋巴细胞白血病成本效果的比较分析. 药学与临床研究, 2016, 24(1): 66-68.
27.	张志萍, 朱秀美, 郭代红, 等. 急性非淋巴性白血病3种治疗方案的成本效果分析. 药物与临床, 2001, 16(2): 9-10.
28.	郭代红, 张志萍, 朱秀美, 等. 急性非淋巴性白血病7种化疗方案的成本-效果分析. 中国药业, 2003, 12(5): 51-52.

1. National Cancer Institute. SEER stat fact sheets: chronic myeloid leukemia. 2020.
2. 应江伟, 杜灵彬, 郑艳泽, 等. 浙江省肿瘤登记地区2000~2009年白血病发病与死亡分析. 中国肿瘤, 2014, 23(7): 538-542.
3. 黄治虎, 陈宝安, 欧阳建, 等. 我国白血病流行病学调查的现状和对策. 临床血液学杂志, 2009, 22(2): 166-167.
4. 周建军, 陈睿, 王烨菁, 等. 上海市卢湾区居民2004-2011年白血病发病和死亡资料分析. 中华肿瘤防治杂志, 2017, 24(8): 507-511.
5. 斯垚翔, 钱虹, 刘玉琴, 等. 甘肃省2010年白血病发病和死亡分析. 中国肿瘤, 2015, 24(2): 81-88.
6. 董金凤. 2011-2012年福建省新发成人急性白血病的发病情况调查. 福建: 福建医科大学, 2014.
7. 刘玉琴, 赵凤菊, 陈万青, 等. 中国2009年白血病发病和死亡资料分析. 中国肿瘤, 2013, 22(7): 528-534.
8. 赵爽, 乔文亮, 邱志新, 等. 吉非替尼治疗晚期非小细胞肺癌的药物经济学系统评价. 华西医学, 2018, 33(1): 76-83.
9. 王莉, 胡善联, 孙鑫, 等. 卫生经济学分析的系统评价方法与挑战. 中国循证医学杂志, 2008, 8(11): 1007-1011.
10. Husereau D, Drummond M, Petrou S, et al. Consolidated health economic evaluation reporting standards (CHEERS) statement. Value Health, 2013, 16(2): e1-e5.
11. 肖军, 孙谨芳, 王琦琦, 等. 卫生经济学评价报告指南及应用现状. 中华预防医学杂志, 2017, 51(3): 276-280.
12. Huang BT, Wang Y, Du QF, et al. Analysis of efficacy and cost-effectiveness of high-dose arabinoside versus daunorubicin chemotherapy in older adult patients with acute myeloid leukemia by cytogenetic risk profile: retrospective review from China. Int J Hematol, 2011, 93(4): 474-481.
13. Stein E, Xie J, Duchesneau E, et al. Cost effectiveness of midostaurin in the treatment of newly diagnosed FLT3-mutated acute myeloid Leukemia in the United States. Pharmacoeconomics, 2019, 37(2): 239-253.
14. Tremblay G, Dolph M, Patel S, et al. Cost-effectiveness analysis for midostaurin versus standard of care in acute myeloid leukemia in the United Kingdom. Cost Eff Resour Alloc, 2018, 16: 33.
15. Lambertenghi-Deliliers G, Oriani A, Pioltelli P, et al. Cost-effectiveness analysis of induction regimens containing anthracyclines in adult acute myelogenous leukemia. Leuk Lymphoma, 1991, 5(1): 33-41.
16. Uyl-de Groot CA, Löwenberg B, Vellenga E, et al. Cost-effectiveness and quality-of-life assessment of GM-CSF as an adjunct to intensive remission induction chemotherapy in elderly patients with acute myeloid leukemia. Br J Haematol, 1998, 100(4): 629-636.
17. Arenaza A, Diez R, Esteve J, et al. Cost-effectiveness of midostaurin in the treatment of acute myeloid leukemia with the FLT3 mutation in Spain. Clinicoecon Outcomes Res, 2019, 11: 683-694.
18. Tremblay G, Cariou C, Recher C, et al. Cost-effectiveness of midostaurin in the treatment of newly diagnosed FLT3-mutated acute myeloid leukemia in France. Eur J Health Econ, 2020, 21(4): 543-555.
19. Jacob LA, Aparna S, Lakshmaiah KC, et al. Decitabine compared with low-dose cytarabine for the treatment of older patients with newly diagnosed acute myeloid leukemia: a pilot study of safety, efficacy, and cost-effectiveness. Adv Hematol, 2015, 2015: 167029.
20. Batty N, Yin Y, Wetzler M. Decitabine is more cost effective than cytarabine and daunorubicin in elderly acute myeloid leukemia patients. Journal of Cancer Research & Therapy, 2014, 2(4): 68-73.
21. Coyle D, Villeneuve PJA. Economic evaluation of azacitidine in elderly patients with acute myeloid leukemia with high blast counts. Pharmacoecon Open, 2020, 4(2): 297-305.
22. National Institute for Health and Care Excellence. TA399-Azacitidine for treating acute myeloid leukaemia with more than 30% bone marrow blasts. 2016.
23. National Institute for Health and Care Excellence. TA545-Gemtuzumab ozogamicin for untreated acute myeloid leukaemia. 2018.
24. National Institute for Health and Care Excellence. TA552-Liposomal cytarabine–daunorubicin for untreated acute myeloid leukaemia. 2018.
25. National Institute for Health and Care Excellence. TA642-Gilteritinib for treating relapsed or refractory acute myeloid leukaemia. 2020.
26. 张伶俐, 颜建周, 袁力. 阿糖胞苷分别联合伊达比星、柔红霉素治疗急性非淋巴细胞白血病成本效果的比较分析. 药学与临床研究, 2016, 24(1): 66-68.
27. 张志萍, 朱秀美, 郭代红, 等. 急性非淋巴性白血病3种治疗方案的成本效果分析. 药物与临床, 2001, 16(2): 9-10.
28. 郭代红, 张志萍, 朱秀美, 等. 急性非淋巴性白血病7种化疗方案的成本-效果分析. 中国药业, 2003, 12(5): 51-52.

Chinese Journal of Evidence-Based Medicine

Economic evaluation of different medicines for acute myeloid leukemia: a systematic review

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