PDF(Pubmed)
Abstract：
Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune disease of the central nervous system. Relapse and incomplete recovery from relapse are common in NMOSD. Most patients with NMOSD have IgG to aquaporin-4 (AQP4-IgG). New biological agents for AQP4-IgG-seropositive NMOSD, such as satralizumab, have become available for maintenance therapy. Satralizumab is an anti-interleukin-6 receptor monoclonal antibody. To date, few studies have evaluated satralizumab as an add-on treatment in pediatric NMOSD patients. Here, we report an 11-year-old girl with NMOSD who frequently relapsed under long-term treatment, including oral prednisone, rituximab, mycophenolate mofetil (MMF), and maintenance intravenous immunoglobulin treatment even with B-cell depletion. For the poor treatment response and to improve the efficacy of relapse prevention further, the patient received satralizumab treatment as an add-on therapy to MMF plus oral prednisone, with a dose of 120 mg administered subcutaneously at weeks 0, 2, and 4 and every 4 weeks after that. After initiating satralizumab, the patient remained relapse-free for 14 months at the last follow-up. Satralizumab might be effective and safe as an add-on treatment in refractory pediatric AQP4-IgG-seropositive NMOSD under B-cell depletion.
摘要：
视神经脊髓炎谱系障碍（NMOSD）是一种罕见的中枢神经系统自身免疫性疾病。复发和从复发中不完全恢复在NMOSD中是常见的。大多数患有NMOSD的患者具有IgG至水通道蛋白-4(AQP4-IgG)。AQP4-IgG血清阳性NMOSD的新生物制剂，比如satralizumab，已可用于维持治疗。Satralizumab是一种抗白细胞介素6受体单克隆抗体。迄今为止,很少有研究评估satralizumab作为儿科NMOSD患者的附加治疗.这里,我们报道了一名患有NMOSD的11岁女孩，在长期治疗下经常复发，包括口服泼尼松,利妥昔单抗,霉酚酸酯（MMF），和维持静脉注射免疫球蛋白治疗，即使B细胞耗尽。对于不良的治疗反应，进一步提高复发预防的疗效，患者接受了Satralizumab治疗，作为MMF加口服泼尼松的附加疗法，在第0、2和4周以及之后每4周皮下给予120mg剂量。在开始使用satralizumab后，在最后一次随访时,患者持续14个月无复发.Satralizumab作为B细胞耗竭下难治性儿科AQP4-IgG血清阳性NMOSD的附加治疗可能是有效和安全的。
