隐藏的基线异质性可掩盖异种移植研究疗效：《科学报告》论文称基线感知分析检出被汇总分析漏掉的响应亚群
《科学报告》11月7日发表 Gül 与 Hekim 的论文《隐藏的基线异质性可掩盖随机化异种移植研究中的治疗效应》。论文指出，随机化与标准化分析流程是临床前肿瘤学的核心做法，但二者并不必然保证各组在基线时具有生物学可比性；作者使用一份在标准合同研究组织（CRO）条件下生成的真实世界异种移植疗效数据集进行检验。
发现方面，尽管按常规汇总指标看，各治疗组在研究开始时看似均衡，队列内部的基线肿瘤体积却存在潜在结构：把所有动物汇总到组水平的分析报告「无显著治疗效应」，而对肿瘤生长轨迹进行基线感知的评估后，一个响应者亚群的信号显现出来，此前正是被汇总平均掩盖了。作者进一步观察到，在常规临床前工作流程下，基线肿瘤体积异质性很常见，并同样能够改变对疗效的解读。
作者据此写道，基线肿瘤负荷是一个具有生物学后果的隐藏异质性来源；在不偏离标准实验实践的前提下，采用基线感知的分析策略，可以提高临床前疗效评估的敏感度与可解释性。该工作在马耳他癌症研究与创新中心基金会（CRIHM）XJENZA Malta 支持项目范围内完成，资助编号为 CRP-2024-11。论文为正式发表的同行评审论文，结论基于该真实世界数据集的再分析，不涉及新的动物实验结果。
（来源：《科学报告》）
Hidden Baseline Heterogeneity Can Mask Treatment Effects in Randomized Xenograft Studies: A Scientific Reports Paper Shows Baseline-Aware Analysis Revealing a Responder Subpopulation Pooled Analysis Missed
A paper published in Scientific Reports on November 7 by Gül and Hekim, titled "Hidden baseline heterogeneity can mask treatment effects in randomized xenograft studies," argues that randomization and standardized analytical workflows, while central to preclinical oncology, do not necessarily ensure biological comparability at baseline. The authors test this using a real-world xenograft efficacy dataset generated under standard contract research organization (CRO) conditions.
Although treatment groups appeared balanced at study entry by conventional summaries, baseline tumour volume exhibited latent structure within the cohort. Pooled group-level analyses reported no significant treatment effect, whereas baseline-aware evaluation of tumour growth trajectories revealed a responder subpopulation whose signal had been masked by aggregation. The authors also observed that baseline tumour volume heterogeneity is common under routine preclinical workflows and can similarly alter efficacy interpretation.
They conclude that baseline tumour burden is a biologically consequential source of hidden heterogeneity, and that baseline-aware analytical strategies can improve the sensitivity and interpretability of preclinical efficacy assessment without deviation from standard experimental practice. The work was conducted within the XJENZA Malta-supported program of the Foundation for Cancer Research and Innovation Hub Malta (CRIHM), under grant reference CRP-2024-11. The paper is formally published and peer-reviewed; its findings rest on re-analysis of the real-world dataset rather than new animal experiments.
(Source: Scientific)
来源：《科学报告》
发布：GSM·美国（发布链接）
图片来源：图：肿瘤组织显微图像（资料图）。CC BY-SA 3.0，经 Wikimedia Commons。 / Photo: Microscopic image of tumour tissue (file photo). CC BY-SA 3.0, via Wikimedia Commons.
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