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青少年特发性脊柱侧凸曲线进展风险的预测模型建立:127 例患者的前瞻性队列研究。

Prognostic model development for risk of curve progression in adolescent idiopathic scoliosis: a prospective cohort study of 127 patients.

机构信息

Department of Health, Medicine and Caring Sciences, Unit of Physiotherapy, Linköping University, Linköping, Sweden.

Department of Clinical Science, Intervention and Technology (CLINTEC), Division of Orthopaedics and Biotechnology, Karolinska Institutet, Stockholm; Department of Reconstructive Orthopaedics, Karolinska University Hospital Huddinge, Stockholm, Sweden.

出版信息

Acta Orthop. 2024 Sep 13;95:536-544. doi: 10.2340/17453674.2024.41911.

Abstract

BACKGROUND AND PURPOSE

The study's purpose was to develop and internally validate a prognostic survival model exploring baseline variables for adolescent idiopathic scoliosis curve progression.

METHODS

A longitudinal prognostic cohort analysis was performed on trial data (n = 135) including girls and boys, Cobb angle 25-40°, aged 9-17 years, remaining growth > 1 year, and previously untreated. Prognostic outcome was defined as curve progression of Cobb angle of > 6° prior to skeletal maturity. 34 candidate prognostic variables were tested. Time-to-event was measured with 6-month intervals. Cox proportional hazards regression survival model (CoxPH) was used for model development and validation in comparison with machine learning models (66.6/33.3 train/test data set). The models were adjusted for treatment exposure.

RESULTS

The final primary prognostic model included 127 patients, predicting progress with acceptable discriminative ability (concordance = 0.79, 95% confidence interval [CI] 0.72-0.86). Significant prognostic risk factors were Risser stage of 0 (HR 4.6, CI 2.1-10.1, P < 0.001), larger major curve Cobb angle (HRstandardized 1.5, CI 1.1-2.0, P = 0.005), and higher score on patient-reported pictorial Spinal Appearance Questionnaire (pSAQ) (HRstandardized 1.4, CI 1.0-1.9, P = 0.04). Treatment exposure, entered as a covariate adjustment, contributed significantly to the final model (HR 3.1, CI 1.5-6.0, P = 0.001). Sensitivity analysis displayed that CoxPH maintained acceptable discriminative ability (AUC 0.79, CI 0.65-0.93) in comparison with machine learning algorithms.

CONCLUSION

The prognostic model (Risser stage, Cobb angle, pSAQ, and menarche) predicted curve progression of > 6° Cobb angle with acceptable discriminative ability. Adding patient report of the pSAQ may be of clinical importance for the prognosis of curve progression.

摘要

背景与目的

本研究旨在开发并内部验证一个预测性生存模型,用于探索青少年特发性脊柱侧凸曲线进展的基线变量。

方法

对试验数据(n=135)进行了纵向预后队列分析,纳入了女孩和男孩,Cobb 角 25-40°,年龄 9-17 岁,剩余生长>1 年,且未经治疗。预后结果定义为在骨骼成熟前 Cobb 角进展>6°。测试了 34 个候选预后变量。使用 6 个月的时间间隔测量时间事件。使用 Cox 比例风险回归生存模型(CoxPH)与机器学习模型(66.6/33.3 训练/测试数据集)进行模型开发和验证。该模型针对治疗暴露进行了调整。

结果

最终的主要预后模型纳入了 127 名患者,具有可接受的判别能力(一致性=0.79,95%置信区间[CI]0.72-0.86)。显著的预后危险因素包括 Risser 分期 0 期(HR4.6,CI2.1-10.1,P<0.001)、较大的主弯 Cobb 角(HR标准化1.5,CI1.1-2.0,P=0.005)和较高的患者报告的脊柱外观问卷(pSAQ)评分(HR标准化 1.4,CI1.0-1.9,P=0.04)。作为协变量调整纳入的治疗暴露对最终模型有显著贡献(HR3.1,CI1.5-6.0,P=0.001)。敏感性分析显示,CoxPH 与机器学习算法相比,保持了可接受的判别能力(AUC0.79,CI0.65-0.93)。

结论

该预后模型(Risser 分期、Cobb 角、pSAQ 和初潮)预测 Cobb 角>6°的曲线进展,具有可接受的判别能力。增加患者对 pSAQ 的报告可能对曲线进展的预后具有重要的临床意义。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6453/11395820/e3e2cd9fd53f/ActaO-95-41911-g001.jpg

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