回归断点设计与随机对照试验估计：在真菌性溃疡治疗试验中的应用
Regression Discontinuity and Randomized Controlled Trial Estimates: An Application to The Mycotic Ulcer Treatment Trials.
作者信息
Oldenburg Catherine E, Venkatesh Prajna N, Krishnan Tiruvengada, Rajaraman Revathi, Srinivasan Muthiah, Ray Kathryn J, O'Brien Kieran S, Glymour M Maria, Porco Travis C, Acharya Nisha R, Rose-Nussbaumer Jennifer, Lietman Thomas M
机构信息
a Francis I. Proctor Foundation, University of California , San Francisco , USA.
b Aravind Eye Care System , Madurai , India.
出版信息
Ophthalmic Epidemiol. 2018 Aug;25(4):315-322. doi: 10.1080/09286586.2018.1469156. Epub 2018 May 2.
PURPOSE
We compare results from regression discontinuity (RD) analysis to primary results of a randomized controlled trial (RCT) utilizing data from two contemporaneous RCTs for treatment of fungal corneal ulcers.
METHODS
Patients were enrolled in the Mycotic Ulcer Treatment Trials I and II (MUTT I & MUTT II) based on baseline visual acuity: patients with acuity ≤ 20/400 (logMAR 1.3) enrolled in MUTT I, and >20/400 in MUTT II. MUTT I investigated the effect of topical natamycin versus voriconazole on best spectacle-corrected visual acuity. MUTT II investigated the effect of topical voriconazole plus placebo versus topical voriconazole plus oral voriconazole. We compared the RD estimate (natamycin arm of MUTT I [N = 162] versus placebo arm of MUTT II [N = 54]) to the RCT estimate from MUTT I (topical natamycin [N = 162] versus topical voriconazole [N = 161]).
RESULTS
In the RD, patients receiving natamycin had mean improvement of 4-lines of visual acuity at 3 months (logMAR -0.39, 95% CI: -0.61, -0.17) compared to topical voriconazole plus placebo, and 2-lines in the RCT (logMAR -0.18, 95% CI: -0.30, -0.05) compared to topical voriconazole.
CONCLUSIONS
The RD and RCT estimates were similar, although the RD design overestimated effects compared to the RCT.
目的
我们利用两项同期进行的治疗真菌性角膜溃疡的随机对照试验（RCT）数据，将回归断点（RD）分析结果与随机对照试验的主要结果进行比较。
方法
根据基线视力将患者纳入真菌性溃疡治疗试验I和II（MUTT I和MUTT II）：视力≤20/400（logMAR 1.3）的患者纳入MUTT I，视力>20/400的患者纳入MUTT II。MUTT I研究了局部应用那他霉素与伏立康唑对最佳矫正视力的影响。MUTT II研究了局部应用伏立康唑加安慰剂与局部应用伏立康唑加口服伏立康唑的效果。我们将RD估计值（MUTT I的那他霉素组[N = 162]与MUTT II的安慰剂组[N = 54]）与MUTT I的RCT估计值（局部应用那他霉素[N = 162]与局部应用伏立康唑[N = 161]）进行比较。
结果
在RD分析中，与局部应用伏立康唑加安慰剂相比，接受那他霉素治疗的患者在3个月时平均视力提高4行（logMAR -0.39，95%CI：-0.61，-0.17）；与局部应用伏立康唑相比，在RCT中平均视力提高2行（logMAR -0.18，95%CI：-0.30，-0.05）。
结论
RD和RCT的估计值相似，尽管与RCT相比，RD设计高估了疗效。
Zotero 插件