Menascu Shay, Fattal-Valevski Aviva, Vaknin-Dembinsky Adi, Milo Ron, Geva Keren, Magalashvili David, Dolev Mark, Flecther Shlomo, Kalron Alon, Miron Shmulik, Hoffmann Chen, Aloni Roy, Gurevich Michael, Achiron Anat
Multiple Sclerosis Center, Sheba Medical Center, Ramat-Gan, Israel; Sackler School of Medicine, Tel-Aviv University, Tel Aviv, Israel.
Sackler School of Medicine, Tel-Aviv University, Tel Aviv, Israel; Pediatric Neurology Unit, Tel-Aviv Sourasky Medical Center, Tel Aviv, Israel.
J Neurol Sci. 2022 Jan 15;432:120074. doi: 10.1016/j.jns.2021.120074. Epub 2021 Nov 27.
Approximately 40% of young-onset multiple sclerosis (MS) patients experience breakthrough disease, which carries a high risk for long-term disability, and requires using therapies beyond traditional first-line agents. Despite the increasing use of newer disease-modifying treatments (DMTs) in this population, data are not available to guide the need for escalating DMTs and there is a scarcity of data on the effects of natalizumab in children and young adults with active disease. We performed a retrospective analysis of the rate of No Evidence of Disease Activity (NEDA), tolerability, and safety of natalizumab in a multi-center cohort of 36 children and young adults with highly active MS. All patients had active disease and initiated treatment with natalizumab. The primary endpoint was the rate of achieving NEDA-3 status, within two years of natalizumab treatment. To examine a possible effect of age on the outcome of treatment, outcomes were also analyzed by pre-pubertal (n = 13 children aged 9-13 years) and pubertal subgroups (n = 23 young adolescents aged 14-20 years). The NEDA-3 status of the pre-pubertal group was 92% in the first and second year and in the pubertal group - 96% in the first year and 92% in the second year. Natalizumab reduced the number and volume of brain lesions in both pre-pubertal and pubertal groups. Treatment was well-tolerated, only 8 patients (22.2%) had adverse events during the 2-year study period. Our analysis shows that natalizumab is effective and well-tolerated in pre-pubertal and pubertal MS patients.
约40%的早发型多发性硬化症（MS）患者会出现疾病突破，这会带来长期残疾的高风险，且需要使用传统一线药物以外的疗法。尽管在这一人群中越来越多地使用新型疾病修正治疗（DMT），但尚无数据可指导升级DMT的必要性，且关于那他珠单抗对患有活动性疾病的儿童和青年的影响的数据也很匮乏。我们对36名患有高度活动性MS的儿童和青年的多中心队列中那他珠单抗的无疾病活动证据（NEDA）率、耐受性和安全性进行了回顾性分析。所有患者均患有活动性疾病并开始使用那他珠单抗治疗。主要终点是在那他珠单抗治疗的两年内达到NEDA-3状态的比率。为了研究年龄对治疗结果的可能影响，还按青春期前（n = 13名9至13岁儿童）和青春期亚组（n = 23名14至20岁青少年）分析了结果。青春期前组在第一年和第二年的NEDA-3状态为92%，青春期组在第一年为96%，第二年为92%。那他珠单抗减少了青春期前和青春期组的脑损伤数量和体积。治疗耐受性良好，在为期2年的研究期间只有8名患者（22.2%）出现不良事件。我们的分析表明，那他珠单抗在青春期前和青春期的MS患者中有效且耐受性良好。
