Martin Paul J, Weisdorf Daniel, Przepiorka Donna, Hirschfeld Steven, Farrell Ann, Rizzo J Douglas, Foley Ronan, Socie Gerard, Carter Shelly, Couriel Daniel, Schultz Kirk R, Flowers Mary E D, Filipovich Alexandra H, Saliba Rima, Vogelsang Georgia B, Pavletic Steven Z, Lee Stephanie J
Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Washington 98109-1024, USA, and Hopital St. Louis, Paris, France.
Biol Blood Marrow Transplant. 2006 May;12(5):491-505. doi: 10.1016/j.bbmt.2006.03.004.
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.
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、开展和分析涉及该疾病患者的临床试验变得困难，即便有前景良好的治疗方案可供选择。本共识文件旨在提供一种克服这些障碍的方法。慢性GVHD的临床试验应遵循良好的试验设计和实践原则。纳入和排除标准应在不影响结果解读的前提下，允许尽可能多的受试者参与。慢性GVHD特征的预入组评估应标准化。方案应就研究药物及其他干预措施的给药提供明确指导。评估反应的方法应提前定义并验证。应选择能反映临床获益的疗效终点。需要专业的生物统计学支持以确保试验结果的有效性和可靠性。在临床试验设计中采用一致的标准来评估在致病途径中具有活性的药物，可能会推动慢性GVHD治疗取得进展。
