Huppke Peter, Stark Wiebke, Zürcher Claudia, Huppke Brenda, Brück Wolfgang, Gärtner Jutta
Department of Pediatrics and Pediatric Neurology, Faculty of Medicine, Georg August University, Robert-Koch-Strasse 40, D-37075 Göttingen, Germany.
Arch Neurol. 2008 Dec;65(12):1655-8. doi: 10.1001/archneur.65.12.1655.
Natalizumab, a humanized monoclonal antibody raised against alpha4 integrins, is approved for treatment of active relapsing-remitting multiple sclerosis (RRMS) in adult patients.
To determine the safety, effectiveness, and tolerability of natalizumab use in pediatric patients with MS.
Case report.
Center for MS in childhood and adolescents, Göttingen, Germany.
Three pediatric patients with RRMS having a poor response to other immunomodulatory therapies or having intolerable adverse effects.
Natalizumab given every 4 weeks at a dosage of 3 to 5 mg/kg of body weight.
Cranial magnetic resonance (MR) imaging before treatment and every 6 months thereafter.
During 24, 16, and 15 months of treatment, no further relapses occurred in the 3 pediatric patients; all reported significant improvement in their quality of life. Follow-up MR imaging showed no new T2-weighted lesions or gadolinium-enhancing lesions. No adverse events were seen when dosage was adjusted to body weight.
Natalizumab treatment was effective and well tolerated in our pediatric patients with RRMS who did not respond to initial immunomodulatory treatments. Therefore, it is a promising second-line therapy for pediatric patients with RRMS.
那他珠单抗是一种针对α4整合素的人源化单克隆抗体，已被批准用于治疗成年患者的活动性复发缓解型多发性硬化症（RRMS）。
确定那他珠单抗在儿科MS患者中的安全性、有效性和耐受性。
病例报告。
德国哥廷根儿童和青少年多发性硬化症中心。
3例RRMS儿科患者，对其他免疫调节疗法反应不佳或有无法耐受的不良反应。
每4周给予那他珠单抗，剂量为3至5mg/kg体重。
治疗前及此后每6个月进行头颅磁共振（MR）成像检查。
在24个月、16个月和15个月的治疗期间，3例儿科患者均未再复发；所有患者均报告生活质量有显著改善。随访MR成像显示无新的T2加权病灶或钆增强病灶。根据体重调整剂量后未见不良事件。
那他珠单抗治疗对我们这些对初始免疫调节治疗无反应的儿科RRMS患者有效且耐受性良好。因此，它是儿科RRMS患者一种有前景的二线治疗方法。
