预测神经发育障碍青少年术后流涎严重程度：概念验证性多变量预后模型的开发

文献信息

中文摘要

引言： 预测流涎手术的结局有助于候选者筛选，并为患者和照护者塑造预期。本研究描述了一个预后预测模型的开发，用于预测神经发育障碍青少年术后32周时预期的流涎视觉模拟量表（VAS）评分。
方法： 利用荷兰一家三级医疗中心唾液控制诊所前瞻性收集的流涎患者匿名化数据库数据，开发了一个多变量线性模型。研究人群为2000年1月至2023年3月期间因流涎接受手术的非进展性神经发育障碍流涎患者。模型的结局指标为32周随访时的VAS。基线VAS被纳入模型，以考虑相对于基线的相对变化。采用自举法进行内部验证。
结果： 该模型基于227例患者（37.4%为女性，手术时中位年龄=14.0岁）开发。最终精简模型包含15个变量，表观合并R2=0.26，校正R2=0.21。内部验证得到的校准斜率为0.86，乐观校正后R2为0.16。若干参数对预测术后结局有显著贡献，包括年龄、基线VAS、手术类型、神经发育障碍类型、行走状态、泻药或促动力药使用、严重吞咽困难以及口腔吸吮和推进能力异常。
讨论： 本研究提供了概念验证，表明多变量线性预测模型可利用常规可获得的临床特征，估计接受手术的神经发育障碍青少年术后前部流涎的严重程度。该模型尚未经过外部验证，因此目前形式不适用于临床使用。相反，这些发现展示了在该人群中个体化结局预测的潜力，并支持开展多中心合作以进行外部验证和进一步改进。若得到验证，此类模型最终可帮助临床医生和照护者设定现实预期，并评估已选定手术的预期获益是否足以支持继续手术。

英文摘要

INTRODUCTION: Predicting outcomes of surgery for drooling could support candidate selection and shape expectations for patients and caregivers. This study describes the development of a prognostic prediction model for the expected drooling Visual Analog Scale (VAS) at 32-weeks post-surgery in youth with a neurodevelopmental disability.
METHODS: A multivariable linear model was developed with data from a prospectively gathered pseudonymized database of drooling patients from the Saliva Control Clinic of a Dutch tertiary care center. The population consisted of drooling patients with a non-progressive neurodevelopmental disability who underwent surgery for drooling between January 2000 and March 2023. The outcome measure for the model was the VAS at 32-week follow-up. The baseline VAS was incorporated in the model to take into account the relative change compared to baseline. Internal validation was performed using bootstrapping.
RESULTS: The model was developed based on 227 patients (37.4% female, median age at surgery = 14.0). The final parsimonious model included 15 variables, resulting in an apparent pooled R2 = 0.26 and an R2-adjusted= 0.21. Internal validation resulted in a calibration slope of 0.86 with an optimism-corrected R2 of 0.16. Several parameters contributed significantly to predicting the outcome after surgery. These included age, VAS at baseline, type of surgery, type of neurodevelopmental disability, ambulatory status, use of laxatives or prokinetic agents, severe dysphagia, and abnormal oral suction and propulsion capacity.
DISCUSSION: This study provides a proof of principle that a multivariable linear prediction model can estimate postoperative anterior drooling severity in youth with neurodevelopmental disabilities undergoing surgery, using routinely available clinical characteristics. The model has not undergone external validation and is therefore not intended for clinical use in its current form. Rather, these findings demonstrate the potential of individualized outcome prediction in this population and support multicenter collaboration for external validation and further refinement. If validated, such a model could eventually support clinicians and caregivers in setting realistic expectations and assessing whether the anticipated benefit of an already selected surgical procedure is sufficient to proceed with surgery.