Paediatric Neurology Unit, Children's Medicine Department, Children's Hospital, University Hospital of Nancy, France.
Sorbonne Université, CNRS, IRD, INRA, Institute of Ecology and Environmental Sciences, iEES Paris, UMR7618, France.
Eur J Paediatr Neurol. 2023 Jul;45:22-28. doi: 10.1016/j.ejpn.2023.05.001. Epub 2023 May 11.
To compare the efficacy and safety of newer and/or second-line disease-modifying treatments (DMTs) with interferon beta-1a.
This observational retrospective study included patients younger than 18 years old in the French KIDBIOSEP cohort who had a diagnosis of relapsing multiple sclerosis between 2008 and 2019 and received at least one DMT. Primary outcome was the annualized relapse rate (ARR). Secondary outcomes were the risk of new T2 or gadolinium-enhanced lesions on brain MRI.
Among 78 patients enrolled, 50 were exposed to interferon and 76 to newer DMTs. Mean ARR went from 1.65 during pre-treatment period to 0.45 with interferon (p < 0.001). Newer DMTs reduced ARR compared to interferon: fingolimod 0.27 (p = 0.013), teriflunomide 0.25 (p = 0.225), dimethyl-fumarate 0.14 (p = 0.045), natalizumab 0.03 (p = 0.007). Risk of new lesions on MRI was reduced with interferon compared to pre-treatment period; it decreased even more with newer DMTs for T2 lesions. Regarding risk of new gadolinium-enhanced lesions, the added value of new treatments compared to interferon was less obvious, except for natalizumab (p = 0.031).
In this real-world setting, newer DMTs showed better efficacy than interferon beta-1a on ARR and risk of new T2 lesions, with a good safety profile. Natalizumab tend to emerge as the most effective treatment.
比较新型和/或二线疾病修正治疗（DMT）与干扰素β-1a 的疗效和安全性。
本观察性回顾性研究纳入了法国 KIDBIOSEP 队列中年龄在 18 岁以下、2008 年至 2019 年期间被诊断为复发性多发性硬化症且至少接受过一种 DMT 的患者。主要结局是年化复发率（ARR）。次要结局是脑 MRI 上新 T2 或钆增强病变的风险。
在纳入的 78 例患者中，50 例接受了干扰素治疗，76 例接受了新型 DMT 治疗。ARR 从治疗前的 1.65 降至干扰素治疗后的 0.45（p<0.001）。与干扰素相比，新型 DMT 降低了 ARR：芬戈莫德 0.27（p=0.013），特立氟胺 0.25（p=0.225），富马酸二甲酯 0.14（p=0.045），那他珠单抗 0.03（p=0.007）。与治疗前相比，干扰素降低了 MRI 上新病灶的风险；新型 DMT 降低 T2 病变的风险甚至更大。关于新 gadolinium-enhanced 病变的风险，新型治疗与干扰素相比的附加价值并不明显，除了那他珠单抗（p=0.031）。
在这种真实环境下，新型 DMT 在 ARR 和新 T2 病变风险方面比干扰素β-1a 更有效，且安全性良好。那他珠单抗可能是最有效的治疗方法。
