Department of Rheumatology and Internal Medicine, Medical University of Bialystok, Bialystok, Poland
J Rheumatol. 2010 Jan;37(1):105-15. doi: 10.3899/jrheum.090661. Epub 2009 Dec 1.
To assess the validation status of echocardiography with continuous Doppler (echo-Doppler) as an outcome measure in pulmonary arterial hypertension associated with systemic sclerosis (PAH-SSc).
Structured literature review on full-text English articles was performed using the PubMed and Cochrane databases. Assessment of validation of echo-Doppler was based on the OMERACT filter criteria with the domains truth (face, content, construct, and criterion validity), discrimination, and feasibility.
Out of 35 studies eligible for analysis, only 5 included well defined PAH-SSc subgroups (World Health Organization criteria). Echo was considered as having face validity based on expert opinion and high number of studies using echo for evaluation of patients with SSc. Echo was considered partially validated with respect to criterion validity based on significant correlations between echo measures and right-heart catheterization in patients with SSc at risk of PAH/PH. However, echo was found to lack specificity (lack of content validity), since measurements of echo pulmonary pressure may be influenced by left-heart disease and interstitial lung disease. Data from general populations of patients with scleroderma indicate that evaluation of pulmonary artery pressure by echo might not be available in all PAH-SSc patients because of technical factors. No studies enabling evaluation of the discriminant capacity over time and treatment of echo in PAH-SSc could be identified.
Further studies are needed to fully validate echo-Doppler as an outcome measure in PAH-SSc. These studies would include cross-sectional analysis of baseline measures and longitudinal data of placebo and verum groups in randomized controlled trials of patients with PAH-SSc.
评估连续多普勒超声心动图（echo-Doppler）作为系统性硬皮病相关肺动脉高压（PAH-SSc）结局测量的验证状态。
使用 PubMed 和 Cochrane 数据库对全文为英文的文献进行结构化文献回顾。根据 OMERACT 筛选标准，通过真实性（表面、内容、构建和标准有效性）、判别力和可行性来评估 echo-Doppler 的验证情况。
在符合分析条件的 35 项研究中，只有 5 项研究纳入了明确界定的 PAH-SSc 亚组（世界卫生组织标准）。根据专家意见和大量使用 echo 评估 SSc 患者的研究，echo 被认为具有表面有效性。基于 echo 测量值与 SSc 患者发生肺动脉高压/PH 风险时右心导管检查之间的显著相关性，echo 在一定程度上被认为具有标准有效性。然而，echo 被认为缺乏特异性（内容有效性缺失），因为 echo 肺动脉压的测量可能受到左心疾病和间质性肺疾病的影响。来自硬皮病患者一般人群的数据表明，由于技术因素，并非所有 PAH-SSc 患者的 echo 都可用于评估肺动脉压。没有研究能够评估 PAH-SSc 中 echo 的判别能力和治疗效果。
需要进一步研究来充分验证 echo-Doppler 作为 PAH-SSc 的结局测量。这些研究将包括在 PAH-SSc 患者的随机对照试验中，对基线测量值进行横断面分析以及安慰剂和真实治疗组的纵向数据。
