A Novel Method to Combine Assessment of Benefit and Harm: Outcome Measures in Rheumatology 3x3 Methodology Applied to Two Active Comparator Trials
A Novel Method to Combine Assessment of Benefit and Harm: Outcome Measures in Rheumatology 3x3 Methodology Applied to Two Active Comparator Trials
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DOI：
10.1002/acr.23590
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发表时间：
2019-02-01
影响因子：
4.7
通讯作者：
Curtis, Jeffrey R.
中科院分区：
文献类型：
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作者：
Boers, Maarten;Singh, Jasvinder A.;Curtis, Jeffrey R.
目的 风湿病结果测量 (OMERACT) 3x3 方法在个体患者层面同时分析获益和危害的发生。我们将此方法应用于最近的 2 个类风湿性关节炎 (RA) 试验数据集。方法 早期侵袭性类风湿关节炎治疗 (TEAR) 和类风湿关节炎积极治疗比较 (RACAT) 随机试验结果的安全性根据 OMERACT 定义为无不良事件 (AE)、非严重 AE 和严重 AE。治疗效果定义为良好、中等或无反应。没有任何 AE 的良好治疗反应被标记为绝对成功，没有治疗反应但至少有 1 个 AE 被认为是完全失败。根据需要，通过卡方检验或精确检验来评估益处和危害之间的关联。结果 在 TEAR 中，755 名患者中的 612 名患者在 48 周时获得了缓解数据：14% 的患者获得了不合格的成功，9% 的患者获得了完全的失败，治疗组之间没有差异。治疗反应和 AE 发生率不相关。在 RACAT 中，353 名患者中有 309 名在 48 周时获得了缓解数据：6% 的患者获得了完全成功，11% 的患者获得了完全失败，治疗组之间没有差异。有效率和 AE 率呈负相关。 AE 和严重 AE 的频率随着响应的降低而增加 (P = 0.008)。结论 我们发现一些证据表明 AE 的同时出现可能会降低临床反应。
Objective The Outcome Measures in Rheumatology (OMERACT) 3x3 method analyzes the occurrence of benefit and harm simultaneously at the individual patient level. We applied this method to 2 recent rheumatoid arthritis (RA) trial data sets. Methods The Treatment of Early Aggressive Rheumatoid Arthritis (TEAR) and the Rheumatoid Arthritis Comparison of Active Therapies (RACAT) randomized trial outcomes for safety were defined according to OMERACT as having no adverse events (AEs), non-serious AEs, and serious AEs. Treatment efficacy was defined as good, moderate, or no response. A good treatment response without any AEs was labeled an unqualified success, and no treatment response but at least 1 AE was considered an unmitigated failure. The association between benefit and harm was assessed by chi-square or exact tests, as appropriate. Results In TEAR, 612 of 755 patients had response data at 48 weeks: 14% of patients experienced unqualified success and 9% had unmitigated failure, with no difference between the treatment arms. Treatment response and AE rates were not correlated. In RACAT, 309 of 353 patients had response data at 48 weeks: 6% of patients experienced unqualified success and 11% had unmitigated failure, with no differences between the treatment arms. Response and AE rates were negatively correlated. The frequency of AEs and serious AEs increased as response decreased (P = 0.008). Conclusion We found some evidence that clinical response may be reduced by the co-occurrence of AEs.