经典临床数据陷阱分析.pptVIP

经典临床数据陷阱分析 疑夕 内容提要 2013-06-03: SNTA的ganetespib肺癌II/III期试验结果 亚组分析数据一般不被认可 2014-03-10: LJPC的GCS-100慢性肾病II期试验结果 标准偏差过大导致均值失去意义 2014-10-06: ADHD的MDX成人ADHD III期试验结果 安慰剂组里的“刁民”不能随便剔除 2015-03-12: CUR的NSI-566渐冻症II期试验结果 一不小心就被绕进去的逻辑陷阱 2015-04-26: CLDN的MYDICAR心衰IIb期试验失败 治疗组患者比安慰剂组更健康造成有效假象 2013-06-03: SNTA公布ganetespib肺癌II/III期试验结果 Synta announces positive overall survival results from GALAXY-1 Phase 2b/3 trial of ganetespib in second-line non-small cell lung cancer. Median overall survival improved 32% in the all adenocarcinoma, intent-to-treat population. In the population selected for the ongoing GALAXY-2 Phase 3 trial, median overall survival improved 67%, Hazard Ratio = 0.61 (p=0.009). The magnitude and consistency of the activity reported today are very encouraging and bode well for the outcome of the GALAXY-2 Phase 3 study. Press Release中充斥着positive, encouraging, improve，事实果真如此吗？ 市场对ganetespib肺癌II/III期试验结果的反应 结果公布当日跌34%，随后半个月连续下跌。 Ganetespib肺癌II/III期试验真的成功了吗？ ITT组OS数据未达到统计学显著差异，亚组分析数据一般不被投资人认可。 2014-03-10: LJPC公布GCS-100慢性肾病II期试验结果 La Jolla Pharmaceutical Company reports positive, top-line results from Phase 2 clinical trial of GCS-100 in chronic kidney disease. The trial met its primary efficacy endpoint of a statistically significant improvement in kidney function. Key secondary endpoints were also met, and the effect on circulating galectin-3 levels was consistent with the effect on eGFR. Our experience with GCS-100 in this trial was very encouraging, and my patients tolerated the therapy well. Press Release中充斥着positive, encouraging，主要临床终点、次要临床终点都达到，事实果真如此吗？ 市场对GCS-100慢性肾病II期试验结果的反应 市场表现非常滑稽，数据公布当天涨65%，但是在随后的半个月内跌回原点。 GCS-100真的升高肾小球滤过率吗？ GFR, 肾小球滤过率, GFR下降是慢性肾病的标志，GCS-100慢性肾病II期临床以此为主要临床终点，安慰剂组、1.5 mg/m2组、30 mg/m2组分别-0.58、+1.26、+0.06，初看起来1.5 mg/m2组的GFR确实升高了，而且达到统计学显著差异。 GCS-100真的降低galectin-3水平吗？ galectin-3, GCS-100的靶点, 慢性肾病galectin-3水平升高，GCS-100慢性肾病II期临床以此为次要临床终点，安慰剂组、1.5 mg/m2组、30 mg/m2组分别+1.03、-0.88、+1.29，初看起来1.5 mg/m2组的galectin-3水平确实降低了。 低剂量组有效，高剂量组无效，事