Henry Katie, Nepal Desh, Valley Erin, Pedersen Connor, Duarte-García Alí, Putman Michael
Medical College of Wisconsin, Milwaukee.
Mayo Clinic, Rochester, Minnesota.
ACR Open Rheumatol. 2022 Oct;4(10):897-902. doi: 10.1002/acr2.11484. Epub 2022 Jul 26.
Comparative efficacy randomized controlled trials (RCTs) compare two active interventions in a head-to-head design. They are useful for informing clinical practice guidelines, but the degree to which such trials inform clinical practice guidelines in rheumatology is unknown.
The American College of Rheumatology (ACR) and European Alliance of Associations for Rheumatology (EULAR) websites were searched from January 1, 2017, to June 12, 2021, for clinical practice guidelines. RCTs referenced by each guideline were identified, and information regarding design and outcomes were extracted. Clinical practice recommendations from each guideline were also analyzed.
Fifteen ACR- and nine EULAR-endorsed guidelines were included, which cited 609 RCTs and provided 481 recommendations. Referenced RCTs enrolled an average of 418 patients (SD 985), most commonly evaluated biologic/targeted synthetic disease-modifying antirheumatic drugs (70.1%), and infrequently used a head-to-head design (28%). A minority of recommendations received a high level of evidence (LOE) by the Grades of Recommendation, Assessment, Development, and Evaluation (GRADE) methodology (2.9%) or an "A" grade by the Oxford Centre for Evidence based Medicine Standards (OCEBM) methodology (28.9%). LOE was higher for recommendations informed by RCTs (P < 0.001) or head-to-head RCTs (P = 0.008). Many recommendations received a strong recommendation despite low (8 [2.6%]) or very low (25 [8.3%]) LOE.
Less than one in six rheumatology guideline recommendations are informed by head-to-head RCTs. Recommendations that were informed by head-to-head RCTs were more likely to have a high LOE by both GRADE and OCEBM. Efforts to introduce more comparative efficacy RCTs should be undertaken.
比较疗效随机对照试验（RCT）采用直接比较的设计来对比两种活性干预措施。它们有助于为临床实践指南提供依据，但此类试验在多大程度上为风湿病学临床实践指南提供依据尚不清楚。
检索美国风湿病学会（ACR）和欧洲风湿病学协会联盟（EULAR）网站在2017年1月1日至2021年6月12日期间发布的临床实践指南。确定每个指南引用的RCT，并提取有关设计和结果的信息。还分析了每个指南的临床实践建议。
纳入了15项ACR认可和9项EULAR认可的指南，这些指南引用了609项RCT并提供了481条建议。所引用的RCT平均纳入418名患者（标准差985），最常评估的是生物制剂/靶向合成改善病情抗风湿药物（70.1%），很少采用直接比较设计（28%）。少数建议通过推荐分级、评估、制定和评价（GRADE）方法获得了高水平证据（LOE）（2.9%），或通过牛津循证医学中心标准（OCEBM）方法获得了“A”级（28.9%）。RCT提供依据的建议的LOE更高（P < 0.001），直接比较RCT提供依据的建议也是如此（P = 0.008）。尽管许多建议的LOE较低（8条[2.6%]）或非常低（25条[8.3%]），但仍获得了强烈推荐。
不到六分之一的风湿病学指南建议有直接比较RCT作为依据。有直接比较RCT作为依据的建议更有可能通过GRADE和OCEBM方法获得高LOE。应努力引入更多比较疗效RCT。
