From the Department of Neurology, Yale School of Medicine, New Haven, CT (A.C.L., K.N.S., G.J.F.).
Department of Biostatistical Sciences, Wake Forest University School of Medicine, Winston-Salem, NC (M.C., C.L.).
Stroke. 2019 Aug;50(8):2044-2049. doi: 10.1161/STROKEAHA.118.023851. Epub 2019 Jun 26.
Background and Purpose- Clinical trials in spontaneous intracerebral hemorrhage (ICH) have used volume cutoffs as inclusion criteria to select populations in which the effects of interventions are likely to be the greatest. However, optimal volume cutoffs for predicting poor outcome in deep locations (thalamus versus basal ganglia) are unknown. Methods- We conducted a 2-phase study to determine ICH volume cutoffs for poor outcome (modified Rankin Scale score of 4-6) in the thalamus and basal ganglia. Cutoffs with optimal sensitivity and specificity for poor outcome were identified in the ERICH ([Ethnic/Racial Variations of ICH] study; derivation cohort) using receiver operating characteristic curves. The cutoffs were then validated in the ATACH-2 trial (Antihypertensive Treatment of Acute Cerebral Hemorrhage-2) by comparing the c-statistic of regression models for outcome (including dichotomized volume) in the validation cohort. Results- Of the 3000 patients enrolled in ERICH, 1564 (52%) had deep ICH, of whom 1305 (84%) had complete neuroimaging and outcome data (660 thalamic and 645 basal ganglia hemorrhages). Receiver operating characteristic curve analysis identified 8 mL in thalamic (area under the curve, 0.79; sensitivity, 73%; specificity, 78%) and 18 mL in basal ganglia ICH (area under the curve, 0.79; sensitivity, 70%; specificity, 83%) as optimal cutoffs for predicting poor outcome. The validation cohort included 834 (84%) patients with deep ICH and complete neuroimaging data enrolled in ATACH-2 (353 thalamic and 431 basal ganglia hemorrhages). In thalamic ICH, the c-statistic of the multivariable outcome model including dichotomized ICH volume was 0.80 (95% CI, 0.75-0.85) in the validation cohort. For basal ganglia ICH, the c-statistic was 0.81 (95% CI, 0.76-0.85) in the validation cohort. Conclusions- Optimal hematoma volume cutoffs for predicting poor outcome in deep ICH vary by the specific deep brain nucleus involved. Utilization of location-specific volume cutoffs may improve clinical trial design by targeting deep ICH patients that will obtain maximal benefit from candidate therapies.
背景与目的-自发性脑出血（ICH）的临床试验曾使用容积截断值作为纳入标准，以选择干预效果可能最大的人群。然而，预测深部位置（丘脑与基底节）不良预后的最佳容积截断值尚不清楚。方法-我们进行了一项两阶段研究，旨在确定丘脑和基底节ICH 预后不良（改良Rankin 量表评分 4-6）的容积截断值。通过接收者操作特征曲线，在 ERICH（[脑出血的种族/民族差异]研究；推导队列）中确定了预测不良预后的最佳敏感性和特异性的截断值。然后，通过比较验证队列中结局（包括二分容积）回归模型的 C 统计量，在 ATACH-2 试验（急性脑出血降压治疗-2）中验证了这些截断值。结果-在 ERICH 纳入的 3000 例患者中，1564 例（52%）患有深部 ICH，其中 1305 例（84%）有完整的神经影像学和结局数据（660 例丘脑出血和 645 例基底节出血）。接收者操作特征曲线分析确定了 8 mL 为丘脑（曲线下面积，0.79；敏感性，73%；特异性，78%）和 18 mL 为基底节 ICH（曲线下面积，0.79；敏感性，70%；特异性，83%）为预测不良预后的最佳截断值。验证队列包括 ATACH-2 纳入的 834 例（84%）深部 ICH 患者和完整的神经影像学数据（353 例丘脑出血和 431 例基底节出血）。在丘脑 ICH 中，包括二分 ICH 容积的多变量结局模型的 C 统计量在验证队列中为 0.80（95%CI，0.75-0.85）。对于基底节 ICH，在验证队列中，C 统计量为 0.81（95%CI，0.76-0.85）。结论-预测深部 ICH 不良预后的最佳血肿容积截断值因涉及的特定深部脑核而异。利用特定部位的容积截断值可以通过针对将从候选治疗中获得最大获益的深部 ICH 患者来改善临床试验设计。
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