利妥昔单抗治疗5例难治性视神经脊髓炎谱系疾病长期疗效观察

摘要

Objective To observe the long-term effect and safety in 5 refractory neuromyelitis optica spectrum disorder (NMOSD)patients treated with rituximab (RTX). Methods The clinical course and neurological function of 5 refractory NMOSD patients treated with RTX were retrospectively analyzed. These patients had no response to steroids or immunoglobulin in our department of neurology,Xuanwu Hospital. Annualized relapse rate (ARR)and the expanded disability status scale score (EDSS)were recorded and compared before and after treatment. And the efficacy and side effects of RTX were valued. Results Before RTX treatment,the median and interquartile range of EDSS and ARR were 8.5 (1.25 ) and 1 (2.12 ) times respectively. In the follow-up period (the medium time is 52 months),the median and interquartile range of EDSS and ARR were 2.5 (5.75 )and 0 (0.35 )times respectively,with significant reduction in ARR and improvements in EDSS. Mild side effect occurred in one case,no severe side effects occurred. Conclusions RTX can improve the clinical symptoms among refractory NMOSD patients in long-term observation,and could be an effective and safe therapy.%目的：观察利妥昔单抗治疗难治性视神经脊髓炎谱系疾病（neuromyelitis optica spectrum disorder， NMOSD）的长期临床疗效及安全性。方法回顾性分析作者医院神经内科应用利妥昔单抗治疗的5例难治性NMOSD 患者（经糖皮质激素或丙种球蛋白治疗无效）的用药经过和临床情况，比较治疗前后年复发率（ARR）和扩展残疾状态量表评分（EDSS）的差异，评价利妥昔单抗的疗效和副作用。结果应用利妥昔单抗治疗前，5例患者的 EDSS 和 ARR 中位数（四分位数）分别为8．5（1．25）分和1（2．12）次；经利妥昔单抗治疗后，随访期间（随访时间中位数为52个月）5例患者的 EDSS 和 ARR 中位数（四分位间距）分别为2．5（5．75）分和0（0．35）次，治疗前后患者的 EDSS 和 ARR 差异有统计学意义。治疗期间仅1例患者出现轻微不良反应，无严重不良反应发生。结论利妥昔单抗可改善难治性 NMOSD 患者的长期临床症状，可能是一种治疗难治性 NMOSD 的有效药物。