摘要
在能证明药物疗效从而获得上市批准的关键性临床研究中,选择的主要终点应能够准确客观地反映新药为患者带来的临床获益。目前血液肿瘤的关键性临床研究中常用的疗效终点包括总生存期(OS)、无进展生存期(PFS)等,还有特异性疗效终点如输血不依赖(TI)、完全细胞学反应(CCyR)和主要分子学反应(MMR)等。血液肿瘤是一类异质性较大的恶性疾病,应基于不同血液肿瘤的病程特点和治疗目标,选择恰当的主要终点。
In the pivotal clinical trials that can prove the efficacy of the drug to obtain marketing approval, the primary endpoint should be able to accurately and objectively reflect the clinical benefits from the study drug for patients. At present, the commonly used efficacy endpoints in pivotal trials of hematological malignancies include overall survival(OS), progression-free survival(PFS), etc., as well as specific efficacy endpoints such as transfusion independent(TI) and complete cytogenetic response(CCyR) and major molecular response(MMR), etc. Hematological malignancies are a group illness with heterogeneity, whilst the deep understanding of illness per se is important for the primary endpoint selection in pivotal trials.
作者
徐应永
XU Ying-yong(Zai Lab(Shanghai)Co.,Ltd,SHANGHAI 201210,China)
出处
《中国新药与临床杂志》
CAS
CSCD
北大核心
2021年第9期614-618,共5页
Chinese Journal of New Drugs and Clinical Remedies
关键词
血液肿瘤
临床试验
药物批准
总生存期
无进展生存期
hematologic neoplasms
clinical trial
drug approval
overall survival
progression-free survival
