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...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。展开更多
The rapid advances in the understanding of oncogenic process and the maturation of affordable precision diagnostic tools have enabled the development of targeted therapeutic agents,such as those targeting BCR-ABL,epit...The rapid advances in the understanding of oncogenic process and the maturation of affordable precision diagnostic tools have enabled the development of targeted therapeutic agents,such as those targeting BCR-ABL,epithelial growth factor receptor L858R,EML4-anaplastic lymphoma kinase,and BRAF V600E,to treat cancers that harbor specific molecular alterations.Traditionally,each targeted drug has been developed for a particular tumor type where such alteration is most frequently found.Recently,the widespread adoption of next generation sequencing has led to an increase in the identification of rare and ultra-rare alterations,and,in some cases,the same rare alterations are found across multiple tumor types.The rarity of these alterations makes clinical trials traditionally designed for specific tumor types infeasible.As a result,tissue-agnostic trials have been developed to study the efficacy of these treatments and increase patient access.This review summarizes current successful cases of tissue-agnostic development,such as drugs targeting tropomyosin receptor kinase fusions,and proposes the next wave of potential tissue-agnostic targets,including fusions of ROS1,anaplastic lymphoma kinase,fibroblast growth factor receptor,and rearranged during transfection.In addition,the advantages and the challenges of such approach are discussed in the context of clinical development and approval.展开更多
文摘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。
基金Li IW and Krishnamurthy N were supported by the Emperor Science Awards,an initiative of Stand Up To Cancer(SU2C),made possible by support from Genentech,Bristol-Myers Squibb Company,and Novartis.
文摘The rapid advances in the understanding of oncogenic process and the maturation of affordable precision diagnostic tools have enabled the development of targeted therapeutic agents,such as those targeting BCR-ABL,epithelial growth factor receptor L858R,EML4-anaplastic lymphoma kinase,and BRAF V600E,to treat cancers that harbor specific molecular alterations.Traditionally,each targeted drug has been developed for a particular tumor type where such alteration is most frequently found.Recently,the widespread adoption of next generation sequencing has led to an increase in the identification of rare and ultra-rare alterations,and,in some cases,the same rare alterations are found across multiple tumor types.The rarity of these alterations makes clinical trials traditionally designed for specific tumor types infeasible.As a result,tissue-agnostic trials have been developed to study the efficacy of these treatments and increase patient access.This review summarizes current successful cases of tissue-agnostic development,such as drugs targeting tropomyosin receptor kinase fusions,and proposes the next wave of potential tissue-agnostic targets,including fusions of ROS1,anaplastic lymphoma kinase,fibroblast growth factor receptor,and rearranged during transfection.In addition,the advantages and the challenges of such approach are discussed in the context of clinical development and approval.
