随机对照试验与真实世界队列中周围动脉疾病队列的患者代表性：CLEVER试验与PORTRAIT注册研究
Patient representativeness of a peripheral artery disease cohort in a randomized control trial versus a real-world cohort: The CLEVER trial versus the PORTRAIT registry.
作者信息
Scierka Lindsey E, Jelani Qurat-Ul-Ain, Smolderen Kim G, Gosch Kensey, Spertus John A, Mena-Hurtado Carlos, Jones Philip, Dreyer Rachel P
机构信息
Vascular Medicine Outcomes Program, Section of Cardiovascular Medicine, Department of Internal Medicine, Yale University, New Haven, CT, USA.
Saint Luke's Mid America Heart Institute, Kansas City, MO, USA.
出版信息
Contemp Clin Trials. 2022 Jan;112:106624. doi: 10.1016/j.cct.2021.106624. Epub 2021 Nov 16.
INTRODUCTION
Peripheral artery disease (PAD) is a burdensome disease. It is unclear whether PAD cohorts enrolled in contemporary randomized control trials (RCT) are representative of the real-world PAD populations in terms of their patient characteristics.
METHODS
We compared baseline patient characteristics and health status (as measured by the Peripheral Artery Questionnaire [PAQ]) between the randomized CLEVER study, and the real-world PORTRAIT registry. CLEVER was an RCT of PAD patients enrolled from 29 centers across the US and Canada comparing revascularization with stenting versus optimal medical therapy (OMT) and supervised exercise therapy (SET) plus OMT. PORTRAIT was a multicenter, prospective study of patients with new or worsening PAD symptoms across ten sites in the US.
RESULTS
The final cohort consisted of 879 patients (n = 119 from CLEVER, n = 760 from PORTRAIT (24.5% black, 41.4% women). While CLEVER enrolled patients with aortoiliac disease, only 16.0% of the PORTRAIT cohort had isolated aortoiliac disease. Compared with CLEVER, patients from PORTRAIT were older (64.0 ± 9.5 versus 68.9 ± 9.5 yrs., p ≤0.001), had more severe disease as measured by the ankle brachial index (0.7 ± 0.2 versus 0.9 ± 0.2, p ≤0.001) and a higher prevalence of cardiovascular risk factors including hyperlipidemia and diabetes (all p values<0.05). Both cohorts had similar disease-specific health status as measured by the PAQ summary score (47.4 ± 21.9 versus 43.6 ± 18.4, p = 0.07).
CONCLUSION
In this comparative study, real-world patients with PAD were older and sicker when compared to an RCT with similar indications. This gap may be bridged by improving enrollment of underrepresented high-risk patients in PAD trials testing strategies for PAD symptom relief.
CLINICAL TRIAL REGISTRATION
https://clinicaltrials.gov/ct2/show/NCT01419080?term=portrait&rank=1NCT01419080.
引言
外周动脉疾病（PAD）是一种负担沉重的疾病。目前尚不清楚参与当代随机对照试验（RCT）的PAD队列在患者特征方面是否代表了真实世界中的PAD人群。
方法
我们比较了随机对照的CLEVER研究与真实世界的PORTRAIT注册研究中患者的基线特征和健康状况（通过外周动脉问卷[PAQ]测量）。CLEVER是一项针对从美国和加拿大29个中心招募的PAD患者的RCT，比较了血管重建术与支架置入术、最佳药物治疗（OMT）以及监督运动疗法（SET）加OMT的效果。PORTRAIT是一项在美国十个地点对出现新的或恶化的PAD症状的患者进行的多中心前瞻性研究。
结果
最终队列由879名患者组成（n = 119名来自CLEVER，n = 760名来自PORTRAIT（24.5%为黑人，41.4%为女性）。虽然CLEVER纳入了患有主髂动脉疾病的患者，但PORTRAIT队列中只有16.0%患有孤立性主髂动脉疾病。与CLEVER相比，PORTRAIT的患者年龄更大（64.0 ± 9.5岁对68.9 ± 9.5岁，p≤0.001），通过踝臂指数测量的疾病更严重（0.7 ± 0.2对0.9 ± 0.2，p≤0.001），并且包括高脂血症和糖尿病在内的心血管危险因素患病率更高（所有p值<0.05）。通过PAQ总结评分测量，两个队列的疾病特异性健康状况相似（47.4 ± 21.9对43.6 ± 18.4，p = 0.07）。
结论
在这项比较研究中，与具有相似适应症的RCT相比，真实世界中的PAD患者年龄更大且病情更严重。通过在测试PAD症状缓解策略的PAD试验中增加代表性不足的高危患者的入组，可能会弥合这一差距。
临床试验注册
https://clinicaltrials.gov/ct2/show/NCT01419080?term=portrait&rank=1NCT01419080 。
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