Division of Neurology, St Michael's Hospital, University of Toronto, Toronto, Ontario, Canada.
Centre d'Esclerosi Múltiple de Catalunya (Cemcat), Hospital Universitari Vall d'Hebron, Barcelona, Spain.
Nat Rev Neurol. 2019 May;15(5):287-300. doi: 10.1038/s41582-019-0170-8.
Personalized treatment is ideal for multiple sclerosis (MS) owing to the heterogeneity of clinical features, but current knowledge gaps, including validation of biomarkers and treatment algorithms, limit practical implementation. The contemporary approach to personalized MS therapy depends on evidence-based prognostication, an initial treatment choice and evaluation of early treatment responses to identify the need to switch therapy. Prognostication is directed by baseline clinical, environmental and demographic factors, MRI measures and biomarkers that correlate with long-term disability measures. The initial treatment choice should be a shared decision between the patient and physician. In addition to prognosis, this choice must account for patient-related factors, including comorbidities, pregnancy planning, preferences of the patients and their comfort with risk, and drug-related factors, including safety, cost and implications for treatment sequencing. Treatment response has traditionally been assessed on the basis of relapse rate, MRI lesions and disability progression. Larger longitudinal data sets have enabled development of composite outcome measures and more stringent standards for disease control. Biomarkers, including neurofilament light chain, have potential as early surrogate markers of prognosis and treatment response but require further validation. Overall, attainment of personalized treatment for MS is complex but will be refined as new data become available.
个性化治疗是多发性硬化症 (MS) 的理想选择,因为其临床特征具有异质性,但目前的知识空白,包括生物标志物和治疗算法的验证,限制了其实际应用。当代个性化 MS 治疗方法取决于基于证据的预后评估、初始治疗选择和早期治疗反应评估,以确定是否需要转换治疗方案。预后由基线临床、环境和人口统计学因素、MRI 测量值和与长期残疾测量值相关的生物标志物决定。初始治疗选择应该是患者和医生之间的共同决策。除了预后,这个选择还必须考虑患者相关因素,包括合并症、妊娠计划、患者的偏好及其对风险的舒适度,以及药物相关因素,包括安全性、成本和治疗顺序的影响。治疗反应传统上是根据复发率、MRI 病变和残疾进展来评估的。更大的纵向数据集使复合结果测量和更严格的疾病控制标准得以发展。生物标志物,包括神经丝轻链,具有作为预后和治疗反应的早期替代标志物的潜力,但需要进一步验证。总的来说,实现 MS 的个性化治疗很复杂,但随着新数据的出现,它将得到进一步完善。