Ghosh Dhrubajyoti, Pal Samhita, Lutz Michael, Luo Sheng
Department of Biostatistics and Bioinformatics, Duke University, Durham, NC, USA.
Department of Statistics, North Carolina State University, Raleigh, NC, USA.
J Alzheimers Dis. 2025 Aug 14:13872877251365621. doi: 10.1177/13872877251365621.
BackgroundPredicting the risk of clinical progression from cognitively normal (CN) status to mild cognitive impairment (MCI) or Alzheimer's disease (AD) is critical for early intervention in AD. Traditional survival models often fail to capture complex longitudinal biomarker patterns associated with disease progression.ObjectiveWe propose an ensemble survival analysis framework integrating multiple survival models to improve early prediction of clinical progression in initially cognitively normal individuals.MethodsWe analyzed longitudinal biomarker data from the Alzheimer's Disease Neuroimaging Initiative (ADNI) cohort, including 721 participants, limiting analysis to up to three visits (baseline, 6-month follow-up, 12-month follow-up). Of these, 142 (19.7%) experienced clinical progression to MCI or AD. Our approach combined penalized Cox regression (LASSO, Elastic Net) with advanced survival models (Random Survival Forest, DeepSurv, XGBoost). Model predictions were aggregated using ensemble averaging and Bayesian Model Averaging (BMA). Predictive performance was assessed using Harrell's concordance index (C-index) and time-dependent area under the curve (AUC).ResultsThe ensemble model achieved a peak C-index of 0.907 and an integrated time dependent AUC of 0.904, outperforming baseline-only models (C-index 0.608). One follow-up visit after baseline significantly improved prediction accuracy (48.1% C-index, 48.2% AUC gains), while adding a second follow-up provided only marginal gains (2.1% C-index, 2.7% AUC).ConclusionsOur ensemble survival framework effectively integrates diverse survival models and aggregation techniques to enhance early prediction of preclinical AD progression. These findings highlight the importance of leveraging longitudinal biomarker data, particularly one follow-up visit, for accurate risk stratification and personalized intervention strategies.
背景
预测从认知正常(CN)状态发展为轻度认知障碍(MCI)或阿尔茨海默病(AD)的临床进展风险对于AD的早期干预至关重要。传统的生存模型往往无法捕捉与疾病进展相关的复杂纵向生物标志物模式。
目的
我们提出了一个整合多个生存模型的集成生存分析框架,以改善对初始认知正常个体临床进展的早期预测。
方法
我们分析了来自阿尔茨海默病神经影像倡议(ADNI)队列的纵向生物标志物数据,包括721名参与者,分析限制在最多三次访视(基线、6个月随访、12个月随访)。其中,142人(19.7%)经历了向MCI或AD的临床进展。我们的方法将惩罚Cox回归(LASSO、弹性网络)与先进的生存模型(随机生存森林、DeepSurv、XGBoost)相结合。使用集成平均和贝叶斯模型平均(BMA)对模型预测进行汇总。使用Harrell一致性指数(C指数)和时间依赖性曲线下面积(AUC)评估预测性能。
结果
集成模型的峰值C指数为0.907,综合时间依赖性AUC为0.904,优于仅基于基线的模型(C指数0.608)。基线后一次随访显著提高了预测准确性(C指数提高48.1%,AUC提高48.2%),而增加第二次随访仅带来了微小的提高(C指数提高2.1%,AUC提高2.7%)。
结论
我们的集成生存框架有效地整合了多种生存模型和汇总技术,以增强临床前AD进展的早期预测。这些发现强调了利用纵向生物标志物数据,特别是一次随访,进行准确风险分层和个性化干预策略的重要性。