Progressive multiple sclerosis poses a considerable challenge in the evaluation of disease progression and treatment response owing to its multifaceted pathophysiology. Traditional clinical measures such as the Expanded Disability Status Scale are limited in capturing the full scope of disease and treatment effects. Advanced imaging techniques, including MRI and PET scans, have emerged as valuable tools for the assessment of neurodegenerative processes, including the respective role of adaptive and innate immunity, detailed insights into brain and spinal cord atrophy, lesion dynamics and grey matter damage. The potential of cerebrospinal fluid and blood biomarkers is increasingly recognized, with neurofilament light chain levels being a notable indicator of neuro-axonal damage. Moreover, patient-reported outcomes are crucial for reflecting the subjective experience of disease progression and treatment efficacy, covering aspects such as fatigue, cognitive function and overall quality of life. The future incorporation of digital technologies and wearable devices in research and clinical practice promises to enhance our understanding of functional impairments and disease progression. This Review offers a comprehensive examination of these diverse evaluation tools, highlighting their combined use in accurately assessing disease progression and treatment efficacy in progressive multiple sclerosis, thereby guiding more effective therapeutic strategies.

由于进行性多发性硬化症的病理生理学具有多方面，因此在评估疾病进展和治疗反应方面构成了相当大的挑战。传统的临床测量方法（如扩展残疾状况量表）在捕捉疾病和治疗效果的全部范围方面受到限制。先进的成像技术，包括 MRI 和 PET 扫描，已成为评估神经退行性过程的宝贵工具，包括适应性免疫和先天免疫的各自作用、对大脑和脊髓萎缩、病变动力学和灰质损伤的详细见解。脑脊液和血液生物标志物的潜力越来越受到认可，神经丝轻链水平是神经轴突损伤的显着指标。此外，患者报告的结果对于反映疾病进展和治疗效果的主观体验至关重要，涵盖疲劳、认知功能和整体生活质量等方面。未来将数字技术和可穿戴设备纳入研究和临床实践，有望增强我们对功能障碍和疾病进展的理解。本综述对这些不同的评估工具进行了全面检查，强调了它们在准确评估进行性多发性硬化症的疾病进展和治疗效果方面的联合使用，从而指导更有效的治疗策略。