母方案设计用于冠心病中医药防治方案循证优化的思路和实施方法
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摘要
母方案(master protocol)的提出与实施是临床试验的一次重大变革,因其完善的试验网络、合理的试验设计和创新的统计分析方法,成为一种临床资源使用少和医学信息获得多的高效率临床试验新模式,其已在临床肿瘤研究中得到一定应用。本文结合辨证论治的特点,探讨将母方案试验设计应用于中医药临床研究,以"冠心病心绞痛-心肌梗死-心力衰竭"的临床试验设计为例,为同证候系疾病的中医临床研究的母方案设计提供新方法和新思路。
Master protocol is a great transformation of clinical trials with complete research network, reasonable design and innovative statistical analysis methods. It is a highly efficient new model of clinical trials which could obtain more medical information with less clinical resources. Clinical researches in the field of oncology using master protocol have already made delightful achievements. This paper introduces the design of clinical trials on angina pectoris of coronary heart disease, myocardial infarction and heart failure for instance and discusses the application of master protocol to clinical researches of Traditional Chinese Medicine combined with the differentiation of syndromes and treatments. We expect to provide new ideas and methods for the design of master protocol on diseases with similary syndrome pattern series of Traditional Chinese Medicine.
Master protocol is a great transformation of clinical trials with complete research network, reasonable design and innovative statistical analysis methods. It is a highly efficient new model of clinical trials which could obtain more medical information with less clinical resources. Clinical researches in the field of oncology using master protocol have already made delightful achievements. This paper introduces the design of clinical trials on angina pectoris of coronary heart disease, myocardial infarction and heart failure for instance and discusses the application of master protocol to clinical researches of Traditional Chinese Medicine combined with the differentiation of syndromes and treatments. We expect to provide new ideas and methods for the design of master protocol on diseases with similary syndrome pattern series of Traditional Chinese Medicine.
引文
1 Redman MW, Allegra CJ. The master protocol concept. Semin Oncol, 2015,42(5):724-730.
2 Woodcock J, LaVange LM. Master protocols to study multiple therapies, multiple diseases, or both. N Engl J Med, 2017, 377(1):62-70.
3 Herbst R, Rubin E, LaVange L, et al. Issue brief:conference on clinical cancer research. Available at:http://www.focr.org/sites/default/files/.CCCR12 Master Protocol.pdf.
4 Conley BA, Doroshow JH. Molecular analysis for therapy choice:NCI MATCH. Semin Oncol, 2014,41(3):297-299.
5 Kummar S, Williams PM, Lih CJ, et al. Application of molecular profiling in clinical trials for advanced metastatic cancers. J Natl Cancer Inst, 2015,107(4):djv003.
6 Barker AD, Sigman CC, Kelloff GJ, et al. I-SPY2:an adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy.Clin Pharm Ther, 2009, 86(1):97-100.
7 Kaplan R, Maughan T, Crook A, et al. Evaluating many treatments and biomarkers in oncology:a new design. J Clin Oncol, 2013,31(36):4562-4568.
8 Kim ES, Herbst RS, Wistuba II, et al. The BATTLE trial:personalizing therapy for lung cancer. Cancer Discov, 2011, 1(1):44-53.
9 Sleijfer S, Bogaerts J, Siu LL. Designing transformative clinical trials in the cancer genome era. J Clin Oncol, 2013, 31(15):1834-1841.
10 Herbst R, Rubin E, LaVange L, et al. Design of a disease-specific master protocol. Presented at the friends of cancer research/brookings conference on clinical cancer research,Washington, DC, November14, 2012.
11 Herbst RS, Gandara DR, Hirsch FR, et al. Lung master protocol(Lung-MAP)-a biomarker-driven protocol for accelerating development of therapies for squamous cell lung cancer:SWOG S1400. Clin Cancer Res, 2015, 21(7):1514-1524.
12 Steuer CE, Papadimitrakopoulou V, Herbst RS, et al. Innovative clinical trials:the LUNG-MAP study. Clin Pharmacol Ther, 2015,97(5):488-491.
13 Clinical trial designs for studying targeted therapies. Available at:https://am.asco.org/clinical-trial-designs-studying-targetedtherapies.
14 Berry DA. The brave new world of clinical cancer research:adaptive biomarker-driven trials integrating clinical practice with clinical research. Mol Oncol, 2015, 9(5):951-959.
15 Redig AJ, Janne PA. Basket trials and the evolution of clinical trial design in an era of genomic medicine. J Clin Oncol, 2015, 33(9):975-977.
16姜辉,吕肖芳,张丽,等.代谢组学在中医药现代化研究中的应用中华中医药杂志,2013,28(3):588-590.
19赵晨,张晓雨,胡嘉元,等.中医同证候系疾病临床研究母方案设计方法.中医杂志,2018, 59(2):111-115.
18中国心血管病报告编写组.《中国心血管病报告2016》概要.中国循环杂志,2017, 32(6):521-530.
19 Thygesen K, Alpert JS, White HD. Universal definition of myocardial infarction. Eur Heart J, 2007, 28(20):2525-2538.
20邓伏雪,王小娟,胡云凤,等.急性心肌梗死并发首次心力衰竭患者在院死亡率及临床预后分析.西安交通大学学报(医学版),2015,36(1):135-140.
21杨利.邓铁涛教授“冠心三论”湖南中医药导报,2004, 10(6):8-10.
22王东海,董波.冠心病心绞痛证型分布的回顾性分析.世界中西医结合杂志,2015, 10(3):387-389.
23张敏州,王磊.邓铁涛教授论治冠心病介入术后病证的学术思想探析.中医药管理杂志,2006,14(1):32-33.
24霍根红,李娟.中医药防治PCI术后支架内血栓研究述评.中医学报,2016, 31(3):426-429.
25 Fox JL. Master protocol for squamous cell lung cancer readies for launch. J Nat Biotechnol, 2014, 32(2):116-118.
2 Woodcock J, LaVange LM. Master protocols to study multiple therapies, multiple diseases, or both. N Engl J Med, 2017, 377(1):62-70.
3 Herbst R, Rubin E, LaVange L, et al. Issue brief:conference on clinical cancer research. Available at:http://www.focr.org/sites/default/files/.CCCR12 Master Protocol.pdf.
4 Conley BA, Doroshow JH. Molecular analysis for therapy choice:NCI MATCH. Semin Oncol, 2014,41(3):297-299.
5 Kummar S, Williams PM, Lih CJ, et al. Application of molecular profiling in clinical trials for advanced metastatic cancers. J Natl Cancer Inst, 2015,107(4):djv003.
6 Barker AD, Sigman CC, Kelloff GJ, et al. I-SPY2:an adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy.Clin Pharm Ther, 2009, 86(1):97-100.
7 Kaplan R, Maughan T, Crook A, et al. Evaluating many treatments and biomarkers in oncology:a new design. J Clin Oncol, 2013,31(36):4562-4568.
8 Kim ES, Herbst RS, Wistuba II, et al. The BATTLE trial:personalizing therapy for lung cancer. Cancer Discov, 2011, 1(1):44-53.
9 Sleijfer S, Bogaerts J, Siu LL. Designing transformative clinical trials in the cancer genome era. J Clin Oncol, 2013, 31(15):1834-1841.
10 Herbst R, Rubin E, LaVange L, et al. Design of a disease-specific master protocol. Presented at the friends of cancer research/brookings conference on clinical cancer research,Washington, DC, November14, 2012.
11 Herbst RS, Gandara DR, Hirsch FR, et al. Lung master protocol(Lung-MAP)-a biomarker-driven protocol for accelerating development of therapies for squamous cell lung cancer:SWOG S1400. Clin Cancer Res, 2015, 21(7):1514-1524.
12 Steuer CE, Papadimitrakopoulou V, Herbst RS, et al. Innovative clinical trials:the LUNG-MAP study. Clin Pharmacol Ther, 2015,97(5):488-491.
13 Clinical trial designs for studying targeted therapies. Available at:https://am.asco.org/clinical-trial-designs-studying-targetedtherapies.
14 Berry DA. The brave new world of clinical cancer research:adaptive biomarker-driven trials integrating clinical practice with clinical research. Mol Oncol, 2015, 9(5):951-959.
15 Redig AJ, Janne PA. Basket trials and the evolution of clinical trial design in an era of genomic medicine. J Clin Oncol, 2015, 33(9):975-977.
16姜辉,吕肖芳,张丽,等.代谢组学在中医药现代化研究中的应用中华中医药杂志,2013,28(3):588-590.
19赵晨,张晓雨,胡嘉元,等.中医同证候系疾病临床研究母方案设计方法.中医杂志,2018, 59(2):111-115.
18中国心血管病报告编写组.《中国心血管病报告2016》概要.中国循环杂志,2017, 32(6):521-530.
19 Thygesen K, Alpert JS, White HD. Universal definition of myocardial infarction. Eur Heart J, 2007, 28(20):2525-2538.
20邓伏雪,王小娟,胡云凤,等.急性心肌梗死并发首次心力衰竭患者在院死亡率及临床预后分析.西安交通大学学报(医学版),2015,36(1):135-140.
21杨利.邓铁涛教授“冠心三论”湖南中医药导报,2004, 10(6):8-10.
22王东海,董波.冠心病心绞痛证型分布的回顾性分析.世界中西医结合杂志,2015, 10(3):387-389.
23张敏州,王磊.邓铁涛教授论治冠心病介入术后病证的学术思想探析.中医药管理杂志,2006,14(1):32-33.
24霍根红,李娟.中医药防治PCI术后支架内血栓研究述评.中医学报,2016, 31(3):426-429.
25 Fox JL. Master protocol for squamous cell lung cancer readies for launch. J Nat Biotechnol, 2014, 32(2):116-118.