临床试验常用统计分析方法-单因素
合集下载
相关主题
- 1、下载文档前请自行甄别文档内容的完整性，平台不提供额外的编辑、内容补充、找答案等附加服务。
- 2、"仅部分预览"的文档,不可在线预览部分如存在完整性等问题,可反馈申请退款(可完整预览的文档不适用该条件!)。
- 3、如文档侵犯您的权益，请联系客服反馈,我们会尽快为您处理(人工客服工作时间：9:00-18:30)。
临床试验常用统计分析方法 ——单因素分析
问题的引入
临床试验 & 实效研究
临床试验
Clinical Trials
实效研究
Outcome Researches
Early Phase Clinical Trial Pivotal Trial/MRCT
Post Market and Surveillance Epidemiology and Health Survey
闭塞病变（%）
严重钙化（%）
1. 临床数据，归档于雅培血管部。 2. Zilver PTX 使用说明书。 3. LifeStent使用说明书。 4. S.M.A.R.T. Control使用说明书。 4. EverFlex 使用说明书。
264 7.8 0 34 25 45
ZILVER PTX RESILIENT STROLL DURABILITY II LEVANT II VIABAHN
Registry
All-cause mortality (%) 14.4 14.9 0.86 1.29*
Cardiac mortality (%) 7.5 6.0 0.73 1.41*
*
After adjustment for all differences between treatment groups.
试验设计与证据等级
Circulation. 2008;118:1675-1684
ORBITA研究的启示
Lancet 2018; 391: 31-40
Coronary Revascularization in Diabetic Patients: A Comparison of the Randomized and Observational Component of the
• More registry patients were white, more educated, more physically active and reported better quality of life.
Diabetic patients in BARI randomized to PTCA had significantly worse 5-year survival than patients assigned to CABG. This difference was not seen among BARI-eligible patients who opted to select their mode of revascularization.
Detre KM, et al. Circulation 1999; 99: 633-40
目标人群与研究人群 选择偏倚 vs. 外部真实性
不同研究12个月一期通畅率对比
86.3%1
83%2
81%3
80%4
77%5
Байду номын сангаас
73.5%6
50%8
SUPERB
患者基线
平均病变长度（cm） 支架断裂（%） 远段 SFA/PPA病变（%）
Get Approval Manuscripts/Proposals
临床试验基本概念 P值的含义
• 两组比较: 有效率 90% vs. 80% • 是否有显著差异?
有效率 (A vs B) 9 vs. 8
45 vs. 40 90 vs. 80
每组 N 10 50 100
p值 0.53 0.16 0.048
Off-Label Use and Outcome Track Late Treatment Outcome Drug-Drug & Drug-Device Interaction
Registry Clinical Outcome Health Economic Quality of Care Improvement Comparative Effectiveness
9. Viabahn 使用说明书。Dake, M., 全球视野：何时使用 SFA. LINC 2014中的药物洗脱支架
97 7.3 不适用 未知 21 不适用
Primary Endpoint Analysis of the RESOLUT ALL Comers Trial
(Cardiac death, target vessel MI, and clinically driven TLR) at 1 year
241
134
250
287
316
5.4
6.1
0.9
3.1
68
55
7.7
8.9
6.3
2
0.4
NA
20
73
34
279
17
24
48
21
37
257
19
43
10
5. Lutonix 使用说明书。 6. Laird 等人，用于治疗股浅动脉和近端腘动脉病变的镍钛合金支架
植入术和气囊血管成形术。
8. RESILIENT随机试验的12个月内结果。 循环心血管介入，2010 年6月，267-276
了解统计学指标背后的临床意义！
SIMPLICITY系列试验的启示
32mmHg *
HTN1: Hypertension. 2011;57:911-917; HTN2: Lancet 2010; 376: 1903–09 HTN3: NEJM published online March 29, 2014
5-Year Mortality Rates for Randomized and Registry Diabetic Patients
Randomized
PTCA CABG p Value RR
All-cause mortality (%) 34.5 19.4 0.0024 1.87
Cardiac mortality (%) 23.4 8.2 0.0002 3.10
Bypass Angioplasty Revascularization Investigation (BARI)
1,829 patients (19% with DM) with MVD were randomized to CABG or PTCA 2010 eligible patients (17% with DM) did not consent in RCT entered into registry
问题的引入
临床试验 & 实效研究
临床试验
Clinical Trials
实效研究
Outcome Researches
Early Phase Clinical Trial Pivotal Trial/MRCT
Post Market and Surveillance Epidemiology and Health Survey
闭塞病变（%）
严重钙化（%）
1. 临床数据，归档于雅培血管部。 2. Zilver PTX 使用说明书。 3. LifeStent使用说明书。 4. S.M.A.R.T. Control使用说明书。 4. EverFlex 使用说明书。
264 7.8 0 34 25 45
ZILVER PTX RESILIENT STROLL DURABILITY II LEVANT II VIABAHN
Registry
All-cause mortality (%) 14.4 14.9 0.86 1.29*
Cardiac mortality (%) 7.5 6.0 0.73 1.41*
*
After adjustment for all differences between treatment groups.
试验设计与证据等级
Circulation. 2008;118:1675-1684
ORBITA研究的启示
Lancet 2018; 391: 31-40
Coronary Revascularization in Diabetic Patients: A Comparison of the Randomized and Observational Component of the
• More registry patients were white, more educated, more physically active and reported better quality of life.
Diabetic patients in BARI randomized to PTCA had significantly worse 5-year survival than patients assigned to CABG. This difference was not seen among BARI-eligible patients who opted to select their mode of revascularization.
Detre KM, et al. Circulation 1999; 99: 633-40
目标人群与研究人群 选择偏倚 vs. 外部真实性
不同研究12个月一期通畅率对比
86.3%1
83%2
81%3
80%4
77%5
Байду номын сангаас
73.5%6
50%8
SUPERB
患者基线
平均病变长度（cm） 支架断裂（%） 远段 SFA/PPA病变（%）
Get Approval Manuscripts/Proposals
临床试验基本概念 P值的含义
• 两组比较: 有效率 90% vs. 80% • 是否有显著差异?
有效率 (A vs B) 9 vs. 8
45 vs. 40 90 vs. 80
每组 N 10 50 100
p值 0.53 0.16 0.048
Off-Label Use and Outcome Track Late Treatment Outcome Drug-Drug & Drug-Device Interaction
Registry Clinical Outcome Health Economic Quality of Care Improvement Comparative Effectiveness
9. Viabahn 使用说明书。Dake, M., 全球视野：何时使用 SFA. LINC 2014中的药物洗脱支架
97 7.3 不适用 未知 21 不适用
Primary Endpoint Analysis of the RESOLUT ALL Comers Trial
(Cardiac death, target vessel MI, and clinically driven TLR) at 1 year
241
134
250
287
316
5.4
6.1
0.9
3.1
68
55
7.7
8.9
6.3
2
0.4
NA
20
73
34
279
17
24
48
21
37
257
19
43
10
5. Lutonix 使用说明书。 6. Laird 等人，用于治疗股浅动脉和近端腘动脉病变的镍钛合金支架
植入术和气囊血管成形术。
8. RESILIENT随机试验的12个月内结果。 循环心血管介入，2010 年6月，267-276
了解统计学指标背后的临床意义！
SIMPLICITY系列试验的启示
32mmHg *
HTN1: Hypertension. 2011;57:911-917; HTN2: Lancet 2010; 376: 1903–09 HTN3: NEJM published online March 29, 2014
5-Year Mortality Rates for Randomized and Registry Diabetic Patients
Randomized
PTCA CABG p Value RR
All-cause mortality (%) 34.5 19.4 0.0024 1.87
Cardiac mortality (%) 23.4 8.2 0.0002 3.10
Bypass Angioplasty Revascularization Investigation (BARI)
1,829 patients (19% with DM) with MVD were randomized to CABG or PTCA 2010 eligible patients (17% with DM) did not consent in RCT entered into registry