National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: VI. Design of clinical trials working group report
National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: VI. Design of clinical trials working group report
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DOI：
10.1016/j.bbmt.2006.03.004
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发表时间：
2006-05-01
影响因子：
4.3
通讯作者：
Lee, SJ
中科院分区：
文献类型：
--
作者：
Martin, PJ;Weisdorf, D;Lee, SJ
慢性移植物抗宿主病(GVHD)的复杂性以及缺乏成熟的研究方法，使得设计、进行和分析涉及该疾病受试者的临床试验变得困难，即使有希望的治疗方案也是如此。制定这份协商一致文件是为了提供一种克服这些障碍的方法。慢性移植物抗宿主病的临床试验应遵循良好的试验设计和实践原则。纳入和排除标准应允许尽可能多的受试者参与，而不影响对结果的解释。应规范招生前慢性GVHD特征评估。该方案应就研究、用药和其他干预措施的管理提供明确的指导。评估反应的方法应事先确定和验证。疗效终点的选择应反映临床益处。需要专家生物统计学支持，以确保试验结果的有效性和可靠性。在临床试验设计中使用一致的标准来评估在致病途径中具有活性的药物可以促进慢性移植物抗宿主病治疗的进展。(C)2006年美国血液和骨髓移植学会。
The complexity of chronic graft-versus-host disease (GVHD) and the lack of established research methods have made it difficult to design, conduct, and analyze clinical trials involving subjects with this disease, even when promising treatment options are available. This consensus document was developed to offer an approach for overcoming these obstacles. Clinical trials in chronic GVHD should adhere to principles of good trial design and practice. Inclusion and exclusion criteria should allow as many subjects to participate as possible without compromising the interpretation of results. Pre-enrollment assessment of chronic GVHD characteristics should be standardized. The protocol should provide clear guidance about administration of study medication and other interventions. Methods of assessing response should be defined and validated in advance. Efficacy endpoints should be selected to reflect clinical benefit. Expert biostatistical support is needed to ensure the validity and reliability of trial results. The use of consistent standards in clinical trial designs to evaluate agents that have activity in pathogenic pathways could facilitate advances in the treatment of chronic GVHD. (C) 2006 American Society for Blood and Marrow Transplantation.