精准医学背景下母方案设计临床应用的范围综述

doi:10.16460/j.issn1008-9969.2023.13.042

摘要/Abstract

摘要： 目的全面检索国内外母方案设计的相关研究,了解其临床应用所覆盖的研究领域,为国内护理人员开展母方案设计相关研究的实施和推广提供参考。方法采用Arksey和O'Malley提出的范围综述方法,系统检索中国知网、万方数据库、中国生物医学文献数据库、维普、PubMed、Cochrane Library、Embase、Web of Science,检索时限为从建库至2023年4月。根据纳入、排除标准筛选文献,2名研究者独立提取母方案设计核心要素及其应用领域相关资料,采用描述性分析及主题分析法对提取结果进行整理。结果共纳入104篇文献,研究领域涉及肿瘤、临床心理学、新型冠状病毒肺炎等的药物开发评估及个性化健康计划的制定。母方案在国内外应用存在着明显差异,主要体现在3个方面：（1）应用范围不同,国外主要用于评估新药、疗法或医学器材的有效性和安全性,而在我国广泛应用于中医药领域;（2）研究类型有别,国外主要应用多中心随机对照试验、队列研究,而我国此类型研究相对较少;（3）经验和技术支持方面,由于历史和文化背景等原因,国外研究者在母方案的设计和实施方面拥有更丰富的经验和技术支持。因此,在推广和应用母方案时,需充分考虑国内外的差异,并结合本土实际情况探索出适合自身条件下的研究模式和方法,以推进其更广泛和深入的应用。结论母方案的实施是临床研究模式的一次突破性创新,当前母方案设计的应用多集中在肿瘤学、新型冠状病毒肺炎领域,但伞式试验和篮式试验可用于健康领域,平台试验的范围可应用于临床心理学和数字心理健康干预,国内护理研究者应重视对母方案设计的应用,在其指导下开展相关研究,提升护士创新行为,积极寻找新方法和技术,循证优化试验设计,解决临床护理问题,快速适应精准医学的发展。

关键词: 母方案, 篮式试验, 伞式试验, 平台试验, 精准医学, 范围综述

Abstract: Objective To conduct a comprehensive search on studies regarding master protocols design at home and abroad, to understand the research fields in clinical application, and to provide reference for the implementation and promotion of the research about master protocol design by domestic nursing staff. Methods The scoping review proposed by Arksey and O'Malley was used, and we systematically searched literature in databases of CNKI, Wanfang Database, China Biomedical Literature Database, VIP Database, PubMed, Cochrane Library, Embase,and Web of science from the inception to April 2023. According to the inclusion and exclusion criteria, two researchers independently extracted the core elements of master protocol design and its application, and descriptive analysis and thematic analysis were used to sort out the data. Results A total of 104 pieces of literature were collected, covering oncology, psychology, traditional Chinese medicine, neurology, COVID-19 and drug development evaluation. There were significant differences in the application of master protocols between China and foreign countries, which can be mainly reflected in three aspects. 1) Different application scopes. In foreign countries master protocols were mainly used to evaluate the effectiveness and safety of new drugs, therapies or medical equipment, while in China it was widely applied to traditional Chinese medicine. 2) Different types of research. Multi-center randomized controlled trials and cohort studies were commonly used in foreign countries, while there was relatively less such research in China; 3) Differences in experience and technical support. Richer experience and technical support in the design and implementation of master protocols due to historical and cultural background were observed in foreign countries. Therefore, when promoting and applying master protocols, it was necessary to fully consider the differences between China and foreign countries, and explore suitable research models and methods that fit the local conditions, so as to promote the wider and deeper application. Conclusion The implementation of the master protocols is a breakthrough innovation of the clinical research. At present, the application of master protocols is mainly concentrated in the field of oncology and COVID-19, but the umbrella trial and basket trial can be used in the health field, and the platform trial can be applied to clinical psychology and digital mental health intervention. Domestic nursing researchers should pay attention to the application of master protocols and carry out researches under its guidance to optimize the trial design to adapt to the development of precision medicine.

Key words: master protocol, basket trial, umbrella trial, platform trial, precision medicine, scoping review

中图分类号:

R197.323.6

叶琴, 周立霞, 刘宁. 精准医学背景下母方案设计临床应用的范围综述[J]. 护理学报, 2023, 30(13): 42-46.

YE Qin, ZHOU Li-xia, LIU Ning. Clinical application of master protocol design in precision medicine: a scoping review[J]. Journal of Nursing, 2023, 30(13): 42-46.

参考文献

[1] Subbiah V.The next generation of evidence-based medicine[J]. Nat Med, 2023,29(1):49-58. DOI:10.1038/s41591-022-02160-z.
[2] Redman MW, Allegra CJ.The master protocol concept[J]. Semin Oncol,2015,42(5):724-730.DOI:10.1053/j.seminoncol.2015.07.009.
[3] Di Liello R, Piccirillo MC, Arenare L, et al.Master protocols for precision medicine in oncology: overcoming methodology of randomized clinical trials[J]. Life (Basel), 2021,11(11):1253. DOI: 10.3390/life11111253.
[4] Woodcock J, LaVange LM.Master protocols to study multiple therapies, multiple diseases,or both[J]. N Engl J Med, 2017,377(1):62-70. DOI:10.1056/NEJMra1510062.
[5] 于亚南,杜培艳,刘骏,等. 精准医学创新性临床试验设计“主方案”研究的概念、设计与案例[J]. 中国新药杂志,2020, 29(23):2712-2717.DOI:10.3969/j.issn.1003-3734.2020.23.014.
[6] Arksey H, O'Malley L. Scoping studies: towards a methodological framework[J]. Int J Soc Res Method, 2005, 8(1): 19-32. DOI:10.1080/1364557032000119616.
[7] Aromataris E, Riitano D.Constructing a search strategy and searching for evidence. A guide to the literature search for a systematic review[J]. Am J Nurs, 2014,114(5):49-56. DOI: 10.1097/01.NAJ.0000446779.99522.f6.
[8] Liu W, Zhou L, Feng L, et al.BuqiTongluo granule for ischemic stroke, stable angina pectoris, diabetic peripheral neuropathy with Qi deficiency and blood stasis syndrome: rationale and novel basket design[J]. Front Pharmacol, 2021(12):764669. DOI: 10.3389/fphar.2021.764669.
[9] Lamb JJ, Stone M, D'Adamo CR, et al. Personalized lifestyle intervention and functional evaluation health outcomes survey: presentation of the LIFEHOUSE study using N-of-one tent-umbrella-bucket design[J]. J Pers Med, 2022,12(1):115. DOI: 10.3390/jpm12010115.
[10] Millen GC, Yap C.Adaptive trial designs: what are multiarm, multistage trials?[J]. Arch Dis Child Educ Pract Ed, 2020,105(6):376-378.DOI:10.1136/archdischild-2019-317826.
[11] 关之玥,刘岩,张晶晶,等. 母方案平台试验设计方法及其在相关领域中的应用[J]. 中国循证医学杂志,2021,21(8):986-992. DOI:10.7507/1672-2531.202104005.
[12] Gold, S, Bofill Roig, M, Miranda, J, et al. Platform trials and the future of evaluating therapeutic behavioural interventions[J]. Nat Rev Psychol, 2022,1(1):7-8. DOI:10.1038/s44159-021-00012-0.
[13] Werner-Seidler A, Hitchcock C, Bevan A, et al.A cluster randomized controlled platform trial comparing group memory specificity training (MEST) to group psychoeducation and supportive counselling (PSC) in the treatment of recurrent depression[J]. Behav Res Ther, 2018(105):1-9. DOI: 10.1016/j.brat.2018.03.004.
[14] Wells A, Fisher P, Myers S, et al.Metacognitive therapy in treatment-resistant depression: a platform trial[J]. Behav Res Ther,2012,50(6):367-373. DOI:10.1016/j.brat.2012.02.004.
[15] Dickson D, Johnson J, Bergan R, et al.The master observational trial: a new class of master protocol to advance precision medicine[J]. Cell, 2020,180(1):9-14. DOI:10.1016/j.cell.2019.12.009.
[16] Renfro LA, Sargent DJ.Statistical controversies in clinical research: basket trials, umbrella trials, and other master protocols: a review and examples[J]. Ann Oncol, 2017,28(1):34-43. DOI:10.1093/annonc/mdw413.
[17] Hyman DM, Solit DB, Arcila ME, et al.Precision medicine at Memorial Sloan Kettering Cancer Center: clinical next-generation sequencing enabling next-generation targeted therapy trials[J]. Drug Discov Today, 2015,20(12):1422-18. DOI:10.1016/j.drudis.2015.08.005.
[18] 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[J]. 中医杂志, 2018,59(2):111-115. DOI:10.13288/j.11-2166/r.2018.02.006.
[19] 陈莹, 常静玲, 代恒恒, 等. 精准医学模式下的母方案篮式试验设计方法及其在中医药领域中的应用[J]. 中国循证医学杂志, 2022, 22(4):483-489.
[20] 高珺,苏丽文,袁鹰,等. 抗肿瘤药物主方案试验设计进展及统计考量[J].中国卫生统计, 2021,38(6):950-955. DOI:10.3969/j.issn.1002-3674.2021.06.035.
[21] Angus DC, Berry S, Lewis RJ, et al.The REMAP-CAP (randomized embedded multifactorial adaptive platform for community-acquired pneumonia) study. rationale and design[J]. Ann Am Thorac Soc, 2020,17(7):879-891. DOI: 10.1513/AnnalsATS.202003-192SD.

Metrics

Viewed

Full text

Abstract

Cited

Shared