After analyzing treatment patterns in chronic lymphocytic leukemia (CLL) (objective 1), we investigated the relative effectiveness of ibrutinib versus other commonly used treatments (objective 2) in patients with treatment-naïve and relapsed/refractory CLL, comparing patient-level data from two randomized registration trials with two real-world databases. Hazard ratios (HR) and 95% confidence intervals (CIs) were estimated using a multivariate Cox proportional hazards model, adjusted for differences in baseline characteristics. Rituximab-containing regimens were often prescribed in clinical practice. The most frequently prescribed regimens were fludarabine + cyclophosphamide + rituximab (FCR, 29.3%), bendamustine + rituximab (BR, 17.7%), and other rituximab-containing regimens (22.0%) in the treatment-naïve setting (n = 604), other non-FCR/BR rituximab-containing regimens (38.7%) and non-rituximab-containing regimens (28.5%) in the relapsed/refractory setting (n = 945). Adjusted HRs (95% CI) for progression-free survival (PFS) and overall survival (OS), respectively, with ibrutinib versus real-world regimens were 0.23 (0.14-0.37; p < 0.0001) and 0.40 (0.22-0.76; p = 0.0048) in the treatment-naïve setting, and 0.21 (0.16-0.27; p < 0.0001) and 0.29 (0.21-0.41; p < 0.0001) in the relapsed/refractory setting. When comparing real-world use of ibrutinib (n = 53) versus other real-world regimens in relapsed/refractory CLL (objective 3), adjusted HRs (95% CI) were 0.37 (0.22-0.63; p = 0.0003) for PFS and 0.53 (0.27-1.03; p < 0.0624) for OS. This adjusted analysis, based on nonrandomized patient data, suggests ibrutinib to be more effective than other commonly used regimens for CLL.

中文翻译：

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RESONATE-2™和RESONATE™中的单药依鲁替尼与现实世界中PHEDRA数据库中针对慢性淋巴细胞性白血病患者的治疗相比。

在分析了慢性淋巴细胞性白血病（CLL）的治疗模式（目标1）之后，我们比较了治疗未治疗和复发/难治性CLL患者中依鲁替尼与其他常用治疗方法（目标2）的相对有效性。两个具有两个真实世界数据库的随机注册试验。使用多元Cox比例风险模型估算了危险比（HR）和95％置信区间（CIs），并针对基线特征的差异进行了调整。临床实践中经常开出含有利妥昔单抗的治疗方案。在未经治疗的情况下（n = 604），最常用的方案是氟达拉滨+环磷酰胺+利妥昔单抗（FCR，29.3％），苯达莫司汀+利妥昔单抗（BR，17.7％）和其他含利妥昔单抗的方案（22.0％），在复发/难治性环境中，其他非FCR / BR利妥昔单抗治疗方案（38.7％）和非利妥昔单抗治疗方案（28.5％）（n = 945）。依鲁替尼相对于实际方案的无进展生存期（PFS）和总生存期（OS）的调整后HR（95％CI）分别为0.23（0.14-0.37; p <0.0001）和0.40（0.22-0.76; p初次治疗时为0.0048），复发/难治性时为0.21（0.16-0.27; p <0.0001）和0.29（0.21-0.41; p <0.0001）。当比较复发/难治性CLL的实际使用ibrutinib（n = 53）与其他实际治疗方案（目标3）时，PFS和HR调整后的HRs（95％CI）为0.37（0.22-0.63; p = 0.0003）。对于OS为0.53（0.27-1.03; p <0.0624）。根据非随机患者数据进行的这种调整后的分析，