Suppr超能文献

慢性移植物抗宿主病治疗反应的评估:美国国立卫生研究院慢性移植物抗宿主病临床试验标准共识发展项目:IV. 反应标准工作组报告

Measuring therapeutic response in chronic graft-versus-host disease: National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: IV. Response Criteria Working Group report.

作者信息

Pavletic Steven Z, Martin Paul, Lee Stephanie J, Mitchell Sandra, Jacobsohn David, Cowen Edward W, Turner Maria L, Akpek Gorgun, Gilman Andrew, McDonald George, Schubert Mark, Berger Ann, Bross Peter, Chien Jason W, Couriel Daniel, Dunn J P, Fall-Dickson Jane, Farrell Ann, Flowers Mary E D, Greinix Hildegard, Hirschfeld Steven, Gerber Lynn, Kim Stella, Knobler Robert, Lachenbruch Peter A, Miller Frederick W, Mittleman Barbara, Papadopoulos Esperanza, Parsons Susan K, Przepiorka Donna, Robinson Michael, Ward Michael, Reeve Bryce, Rider Lisa G, Shulman Howard, Schultz Kirk R, Weisdorf Daniel, Vogelsang Georgia B

机构信息

National Cancer Institute, National Institutes of Health, Bethesda, Maryland 20892-1203, USA.

出版信息

Biol Blood Marrow Transplant. 2006 Mar;12(3):252-66. doi: 10.1016/j.bbmt.2006.01.008.

Abstract

The lack of standardized criteria for quantitative measurement of therapeutic response in clinical trials poses a major obstacle for the development of new agents in chronic graft-versus-host disease (GVHD). This consensus document was developed to address several objectives for response criteria to be used in chronic GVHD-related clinical trials. The proposed measures should be practical for use both by transplantation and nontransplantation medical providers, adaptable for use in adults and in children, and focused on the most important chronic GVHD manifestations. The measures should also give preference to quantitative, rather than semiquantitative, measures; capture information regarding signs, symptoms, and function separately from each other; and use validated scales whenever possible to demonstrate improved patient outcomes and meet requirements for regulatory approval of novel agents. Based on these criteria, we propose a set of measures to be considered for use in clinical trials, and forms for data collection are provided (). Measures should be made at 3-month intervals and whenever major changes are made in treatment. Provisional definitions of complete response, partial response, and progression are proposed for each organ and for overall outcomes. The proposed response criteria are based on current expert consensus opinion and are intended to improve consistency in the conduct and reporting of chronic GVHD trials, but their use remains to be demonstrated in practice.

摘要

在临床试验中,缺乏用于定量测量治疗反应的标准化标准是慢性移植物抗宿主病(GVHD)新型药物研发的主要障碍。本共识文件旨在确定用于慢性GVHD相关临床试验的反应标准的几个目标。拟议的测量方法应便于移植和非移植医疗人员使用,适用于成人和儿童,并关注慢性GVHD的最重要表现。这些测量方法还应优先采用定量而非半定量的方法;分别获取有关体征、症状和功能的信息;并尽可能使用经过验证的量表来证明患者预后得到改善,并满足新型药物监管批准的要求。基于这些标准,我们提出了一套拟议用于临床试验的测量方法,并提供了数据收集表格()。测量应每3个月进行一次,以及在治疗发生重大变化时进行。针对每个器官和总体结果,提出了完全缓解、部分缓解和病情进展的临时定义。拟议的反应标准基于当前专家共识意见,旨在提高慢性GVHD试验实施和报告的一致性,但其实际应用仍有待验证。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验