From the Department of Neurosciences (M. Puthenparampil, M.G., G.Z., A.M., P.G.), University of Padua; Multiple Sclerosis Centre (M. Puthenparampil, M.G., G.Z., P.P., F.R., P.G.), and Day Hospital and Centre for Advanced Neurological Therapies Unit, University Hospital of Padua; Department of Health Sciences (M. Ponzano, F.B.), Section of Biostatistics, University of Genova; Padua Neuroscience Centre (A.M.), University of Padua; Paediatric Neurology and Neurophysiology Unit (M.N., S.S.), Department of Women's and Children's Health, University Hospital of Padua; Neuroimmunology Group (M.N., S.S.), Paediatric Research Institute "Città della Speranza", Padua; and Neuroradiology Unit (A.D.P.), University Hospital of Padua, Italy.
Neurol Neuroimmunol Neuroinflamm. 2024 Sep;11(5):e200303. doi: 10.1212/NXI.0000000000200303. Epub 2024 Aug 14.
Although pediatric-onset multiple sclerosis (POMS) is characterized by a more rapid accumulation of CNS inflammation than adult-onset MS (AOMS), the therapeutic algorithms applied in POMS are usually based on AOMS therapeutic outcomes. To define a high-efficacy treatment (HET)-based strategy to treat POMS, we designed an observational retrospective study aimed at evaluating the efficacy and safety of natalizumab (NTZ) in naïve POMS and AOMS.
Starting from 160 patients, we applied a 2:1 (adult:pediatric) matching on propensity scores and obtained 32 patients with NTZ-treated POMS and 64 with AOMS, estimated from a multivariable logistic regression model. All patients were clinically and radiologically followed up every 6 months for a mean period of 46.0 ± 26.9 months.
Following re-baseline at month 6, no difference (log-rank test: = 0.924) in new and enlarging T2 white matter lesions, postcontrast T1 lesions, and relapse rate were observed between POMS and AOMS throughout the study. Progression independent of relapse activity (PIRA) was never observed in POMS, while 9 of 64 patients with AOMS (12.5%) had PIRA events during the follow-up (40.0 ± 25.9 months; log-rank value 0.0156). JCV seroconversion rate during NTZ infusion did not differ between POMS and AOMS (log-rank test = 0.3231). Finally, no serious adverse event was observed in both POMS and AOMS.
The favorable outcomes observed on clinical, especially in PIRA, and radiologic parameters strongly support the use of NTZ as a first-choice HET in POMS.
儿科发病多发性硬化症（POMS）的中枢神经系统炎症累积速度较成人发病多发性硬化症（AOMS）更快，然而 POMS 的治疗方案通常基于 AOMS 的治疗效果。为了确定一种针对 POMS 的高效治疗（HET）策略，我们设计了一项观察性回顾性研究，旨在评估那他珠单抗（NTZ）在初治 POMS 和 AOMS 中的疗效和安全性。
我们根据倾向评分进行了 2:1（成人：儿科）匹配，从 160 名患者中获得了 32 名接受 NTZ 治疗的 POMS 患者和 64 名 AOMS 患者，这是根据多变量逻辑回归模型估计的。所有患者均进行临床和影像学随访，平均随访时间为 46.0±26.9 个月。
在第 6 个月重新进行基线评估后，整个研究期间，POMS 和 AOMS 之间在新发病灶和扩大 T2 白质病变、钆增强 T1 病变和复发率方面均未观察到差异（对数秩检验： = 0.924）。在 POMS 中从未观察到与复发无关的进展（PIRA），而在 64 名 AOMS 患者中有 9 名（12.5%）在随访期间发生 PIRA 事件（40.0±25.9 个月；对数秩检验 值 0.0156）。在 NTZ 输注期间 JCV 血清转化率在 POMS 和 AOMS 之间没有差异（对数秩检验 = 0.3231）。最后，在 POMS 和 AOMS 中均未观察到严重不良事件。
在临床，尤其是在 PIRA 方面观察到的良好结果，以及影像学参数，强烈支持将 NTZ 作为 POMS 的首选 HET。
