托珠单抗治疗难治性视神经脊髓炎谱系疾病的疗效与安全性初步观察

发布时间：2018-10-29 11:38

【摘要】：目的观察托珠单抗治疗难治性视神经脊髓炎谱系疾病(neuromyelitis optica spectrum disorder,NMOSD)的疗效与安全性。方法纳入6例血清水通道蛋白抗体(anti-aquaporin-4 antibody,AQP4-Ig G)阳性且经糖皮质激素联合硫唑嘌呤治疗疗效不佳的NMOSD患者,予托珠单抗治疗(8 mg/kg,每个月1次静脉滴注,连续治疗1年以上),前瞻性评估患者的年复发率(annualized relapse rate,ARR)、扩展残疾状态量表评分(expanded disability status scale score,EDSS)及疼痛数字评价量表(numerical rating scales,NAS),评价托珠单抗的疗效及副作用。结果 5例女性和1例男性,年龄34~63岁,病程16~96个月,托珠单抗治疗6~16次,经治疗后平均ARR由1.28±0.58降为0(P0.05),平均EDSS评分由5.50±2.81降为3.83±3.48(P0.05),平均NAS评分由5.83±3.06降为2.5±2.07(P0.05),无严重不良反应发生。结论托珠单抗可改善难治性NMOSD患者的年复发率及神经病理性疼痛,可能是一种治疗难治性NMOSD的有效药物。
[Abstract]:Objective to observe the efficacy and safety of tozhu McAb in the treatment of refractory optic myelitis (neuromyelitis optica spectrum disorder,NMOSD). Methods six patients with NMOSD who were positive for anti-aquaporin-4 antibody,AQP4-Ig G and were treated with glucocorticoid combined with azathioprine were treated with Todzumab once a month (8 mg/kg, per month). The annual recurrence rate (annualized relapse rate,ARR), the extended Disability status scale (expanded disability status scale score,EDSS) and the Digital pain Evaluation scale (numerical rating scales,NAS) were evaluated prospectively. Objective: to evaluate the efficacy and side effects of stipulumab. Results five women and one male, aged 3463 years, had a course of 16 ~ 96 months. The mean ARR decreased from 1.28 卤0.58 to 0 (P0.05). The average EDSS score decreased from 5.50 卤2.81 to 3.83 卤3.48 (P0.05), and the average NAS score decreased from 5.83 卤3.06 to 2.5 卤2.07 (P0.05). Conclusion Torzumab can improve the annual recurrence rate and neuropathic pain in patients with refractory NMOSD. It may be an effective drug in the treatment of refractory NMOSD.
【作者单位】：中山大学附属第三医院;
【基金】：国家自然科学基金(编号:81471218) 广东省自然科学基金(编号:2014A030313014) 广东省科技计划项目(编号:2013B051000037)
【分类号】：R744.52

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