Zia Mohammad I, Siu Lillian L, Pond Greg R, Chen Eric X
Department of Medical Oncology and Hematology, Princess Margaret Hospital and University of Toronto, Toronto, Ontario, Canada.
J Clin Oncol. 2005 Oct 1;23(28):6982-91. doi: 10.1200/JCO.2005.06.679.
To determine whether promising results from phase II studies could be reproduced in phase III studies, and to examine which characteristics of phase II studies might be of predictive value for subsequent phase III studies.
We searched for all phase III studies of chemotherapy in advanced solid malignancies, published in the English language literature from July 1998 to June 2003. Each phase III study was reviewed to identify preceding phase II studies. Phase II and phase III studies included in this analysis must have used identical regimens. Data were extracted from both phase II and phase III studies.
Of 181 phase III studies identified, 43 used therapeutic regimens identical to those in 49 preceding phase II studies. Twelve phase III studies (28%) were "positive." The vast majority (81%) of phase III studies have lower response rates than preceding phase II studies, with a mean difference of 12.9% among all studies analyzed. None of the phase II study characteristics evaluated significantly predicted for "positive" phase III studies, but the sample size of phase II studies demonstrated a trend toward being predictive (P = .083).
Promising results from phase II studies frequently do not translate into "positive" phase III studies. Response rates in most phase III studies are lower than those in preceding phase II studies.
确定II期研究的阳性结果能否在III期研究中重现，并探究II期研究的哪些特征可能对后续III期研究具有预测价值。
我们检索了1998年7月至2003年6月发表在英文文献中的所有晚期实体恶性肿瘤化疗的III期研究。对每项III期研究进行回顾，以确定之前的II期研究。本分析纳入的II期和III期研究必须使用相同的治疗方案。从II期和III期研究中提取数据。
在确定的181项III期研究中，43项使用的治疗方案与之前49项II期研究相同。12项III期研究（28%）为“阳性”。绝大多数（81%）III期研究的缓解率低于之前的II期研究，在所有分析的研究中平均差异为12.9%。所评估的II期研究特征均未显著预测“阳性”III期研究，但II期研究的样本量显示出一定的预测趋势（P = 0.083）。
II期研究的阳性结果通常无法转化为“阳性”III期研究。大多数III期研究的缓解率低于之前的II期研究。
