Mandelli Veronica, Severino Ines, Eyler Lisa, Pierce Karen, Courchesne Eric, Lombardo Michael V
medRxiv. 2024 May 8:2024.05.08.24307039. doi: 10.1101/2024.05.08.24307039.
Phenotypic heterogeneity in early language, intellectual, motor, and adaptive functioning (LIMA) features are amongst the most striking features that distinguish different types of autistic individuals. Yet the current diagnostic criteria uses a single label of autism and implicitly emphasizes what individuals have in common as core social-communicative and restricted repetitive behavior difficulties. Subtype labels based on the non-core LIMA features may help to more meaningfully distinguish types of autisms with differing developmental paths and differential underlying biology. Using relatively large (n=615) publicly available data from early developing (24-68 months) standardized clinical tests tapping LIMA features, we show that stability-based relative cluster validation analysis can identify two robust and replicable clusters in the autism population with high levels of generalization accuracy (98%). These clusters can be described as Type I versus Type II autisms differentiated by relatively high versus low scores on LIMA features. These two types of autisms are also distinguished by different developmental trajectories over the first decade of life. Finally, these two types of autisms reveal striking differences in functional and structural neuroimaging phenotypes and their relationships with gene expression. This work emphasizes the potential importance of stratifying autism by a Type I versus Type II distinction focused on LIMA features and which may be of high prognostic and biological significance.
早期语言、智力、运动及适应性功能(LIMA)特征方面的表型异质性是区分不同类型自闭症个体的最显著特征之一。然而,当前的诊断标准使用单一的自闭症标签,并隐含地强调个体在核心社交沟通和受限重复行为困难方面的共同之处。基于非核心LIMA特征的亚型标签可能有助于更有意义地区分具有不同发育路径和不同潜在生物学特征的自闭症类型。利用来自早期发育阶段(24 - 68个月)涉及LIMA特征的标准化临床测试的相对较大规模(n = 615)的公开可用数据,我们表明基于稳定性的相对聚类验证分析能够在自闭症群体中识别出两个稳健且可重复的聚类,其泛化准确率较高(98%)。这些聚类可描述为I型与II型自闭症,它们在LIMA特征上的得分相对较高或较低。这两种类型的自闭症在生命的第一个十年中也具有不同的发育轨迹。最后,这两种类型的自闭症在功能和结构神经影像学表型及其与基因表达的关系方面表现出显著差异。这项工作强调了以I型与II型区分来对自闭症进行分层的潜在重要性,这种区分聚焦于LIMA特征,可能具有很高的预后和生物学意义。