Seoyeong Pang, Yanyuan Du, Siyang Peng, Linghao Meng, Anni Xiong, Wenzeng Zhu
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引用次数: 0
摘要
背景:重症肌无力(MG)是一种神经肌肉交界处自身免疫性疾病,可导致骨骼肌无力。MG在病程中经常恶化,严重影响生活质量,增加肌无力危象的风险。现有的预测模型仍然很少。本研究建立了MG恶化的预测模型,以促进早期风险分层和个性化护理。研究设计与方法:回顾性分析2019年12月至2024年9月美国重症肌无力基金会(MGFA) I - III级重症肌无力患者437例。分析社会人口学、临床变量及病情恶化情况。通过单变量分析、最小绝对收缩和选择算子(LASSO)回归和多变量逻辑回归确定预测因子。采用受试者工作特征(ROC)分析、校准曲线和决策曲线分析评估模型的性能。结果:患者被随机分为训练组(n = 305)和验证组(n = 132)。恶化率具有可比性(26.52% vs. 31.15%, p = 0.331)。出现了六个预测因素:年龄、MGFA分类、胸腺切除术史、寒战、疲劳和情绪障碍(ED)。模态图具有较强的分辨性(训练AUC: 0.82,验证AUC: 0.83)和校正AUC (Hosmer-Lemeshow p < 0.05)。决策曲线分析在10-70%的概率阈值下证实了临床效用。结论:该nomogram综合了可获得的临床变量,可对MG恶化风险进行分层,从而实现早期干预。通过多中心前瞻性研究验证是必要的,以优化普遍性。
Predicting worsening risk in MGFA class I, II and III myasthenia gravis patients: development and validation of a predictive nomogram.
Background: Myasthenia gravis (MG), a neuromuscular junction autoimmune disorder, causes skeletal muscle weakness. MG worsening frequently occurs during the disease course, severely impairing quality of life and elevating myasthenic crisis risk. Existing predictive models remain scarce. This study developed a predictive model for MG worsening to facilitate early risk stratification and personalized care.
Research design & methods: Retrospective analysis included 437 the Myasthenia Gravis Foundation of America (MGFA) class I - III myasthenia gravis patients from December 2019 to September 2024. Sociodemographic, clinical variables and worsening status were analyzed. Predictors were identified via univariate analysis, the Least Absolute Shrinkage and Selection Operator (LASSO) regression, and multivariate logistic regression. Model performance was assessed using receiver operating characteristic (ROC) analysis, calibration curves, and decision curve analysis.
Results: Patients were randomized into training (n = 305) and validation (n = 132) cohorts. Worsening rates were comparable (26.52% vs. 31.15%, p = 0.331). Six predictors emerged: age, MGFA classification, thymectomy history, chills, fatigue, and emotional disturbances (ED). The nomogram demonstrated strong discrimination (AUC: 0.82 training, 0.83 validation) and calibration (Hosmer-Lemeshow p > 0.05). Decision curve analysis confirmed clinical utility at 10-70% probability thresholds.
Conclusion: This nomogram integrates accessible clinical variables to stratify MG worsening risk, enabling early intervention. Validation through multicenter prospective studies is warranted to optimize generalizability.
期刊介绍:
Expert Review of Clinical Immunology (ISSN 1744-666X) provides expert analysis and commentary regarding the performance of new therapeutic and diagnostic modalities in clinical immunology. Members of the International Editorial Advisory Panel of Expert Review of Clinical Immunology are the forefront of their area of expertise. This panel works with our dedicated editorial team to identify the most important and topical review themes and the corresponding expert(s) most appropriate to provide commentary and analysis. All articles are subject to rigorous peer-review, and the finished reviews provide an essential contribution to decision-making in clinical immunology.
Articles focus on the following key areas:
• Therapeutic overviews of specific immunologic disorders highlighting optimal therapy and prospects for new medicines
• Performance and benefits of newly approved therapeutic agents
• New diagnostic approaches
• Screening and patient stratification
• Pharmacoeconomic studies
• New therapeutic indications for existing therapies
• Adverse effects, occurrence and reduction
• Prospects for medicines in late-stage trials approaching regulatory approval
• Novel treatment strategies
• Epidemiological studies
• Commentary and comparison of treatment guidelines
Topics include infection and immunity, inflammation, host defense mechanisms, congenital and acquired immunodeficiencies, anaphylaxis and allergy, systemic immune diseases, organ-specific inflammatory diseases, transplantation immunology, endocrinology and diabetes, cancer immunology, neuroimmunology and hematological diseases.