Liang Feng, Yin Zhaoxu, Li Yaqian, Li Guanxi, Ma Jing, Zhang Huiqiu, Xia Xiaoqian, Yao Make, Pang Xiaomin, Wang Juan, Chang Xueli, Guo Junhong, Zhang Wei
Department of Neurology, The First Hospital of Shanxi Medical University, No. 85, Jiefang South Street, Taiyuan, China.
First Clinical Medical College, Shanxi Medical University, Taiyuan, China.
Neurol Ther. 2024 Jun;13(3):551-562. doi: 10.1007/s40120-024-00590-0. Epub 2024 Mar 1.
This study aimed to establish and validate a nomogram prognostic model for predicting short-term efficacy of acetylcholine receptor antibody-positive (AChR-Ab+) generalized myasthenia gravis (GMG).
A retrospective observational study was conducted at the First Hospital of Shanxi Medical University, enrolling patients diagnosed with AChR-Ab+ GMG from May 2020 to September 2022. The primary outcome was the change in the Myasthenia Gravis Foundation of America (MGFA) post-intervention status after 6 months of standard treatment. Predictive factors were identified through univariate and multivariate logistic regression analyses, with significant factors incorporated into the nomogram. The bootstrap test was used for internal validation of the nomogram model. Model performance was assessed using calibration curves, receiver-operating characteristic curve analysis, and decision curve analysis (DCA).
A total of 90 patients were enrolled, of whom 30 achieved unchanged or worse status after 6 months of standard therapy. Univariate logistic regression analysis showed that quantitative myasthenia gravis score, gender, body mass index, course of disease, hemoglobin levels, and white blood cell counts were six potential predictors. These factors were used for multivariate logistic regression analysis, and a nomogram was constructed. The calibration curve showed that the predicted value was in good agreement with the actual value (p = 0.707), and the area under the curve value (0.792, 95% CI 0.686-0.899) indicated good discrimination ability. DCA suggests that this model has potential clinical application value.
The constructed nomogram, based on key patient indicators, shows promise as a clinically useful tool for predicting the short-term efficacy of treatment of AChR-Ab+ GMG. Validation in larger, multicenter cohorts is needed to further substantiate its applicability.
本研究旨在建立并验证一种列线图预后模型,用于预测乙酰胆碱受体抗体阳性(AChR-Ab+)的全身型重症肌无力(GMG)的短期疗效。
在山西医科大学第一医院进行了一项回顾性观察研究,纳入2020年5月至2022年9月期间诊断为AChR-Ab+ GMG的患者。主要结局是标准治疗6个月后美国重症肌无力基金会(MGFA)干预后状态的变化。通过单因素和多因素逻辑回归分析确定预测因素,并将显著因素纳入列线图。采用自抽样检验对列线图模型进行内部验证。使用校准曲线、受试者操作特征曲线分析和决策曲线分析(DCA)评估模型性能。
共纳入90例患者,其中30例在标准治疗6个月后病情无变化或恶化。单因素逻辑回归分析显示,重症肌无力定量评分、性别、体重指数、病程、血红蛋白水平和白细胞计数是六个潜在预测因素。将这些因素用于多因素逻辑回归分析,并构建了列线图。校准曲线显示预测值与实际值吻合良好(p = 0.707),曲线下面积值(0.792,95%CI 0.686-0.899)表明具有良好的区分能力。DCA表明该模型具有潜在的临床应用价值。
基于关键患者指标构建的列线图有望成为预测AChR-Ab+ GMG治疗短期疗效的临床有用工具。需要在更大规模的多中心队列中进行验证,以进一步证实其适用性。