Department of Neurology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea.
Department of Preventive Medicine, Ulsan University College of Medicine, Seoul, Republic of Korea; Department of Clinical Epidemiology and Biostatistics, Asan Medical Center, Seoul, Republic of Korea.
Mult Scler Relat Disord. 2020 Jul;42:102109. doi: 10.1016/j.msard.2020.102109. Epub 2020 Apr 28.
The efficacy of intravenous immunoglobulin (IVIG) in neuromyelitis optica spectrum disorders (NMOSD) has rarely been investigated, despite it being a conceivable therapeutic strategy based on its mode of action. We aimed to evaluate the efficacy of IVIG as an add-on therapy to azathioprine in the prevention of NMOSD relapse.
We retrospectively reviewed the medical records of NMO-IgG-positive NMOSD patients treated with IVIG infusions (0.4 g/kg/day) every one to three months as a part of combination therapy with azathioprine for more than six months. Treatment efficacy and safety were assessed based on the changes in the pre-IVIG and post-IVIG treatment annualized relapse rates (ARR), and on the proportion of relapse-free and progression-free patients and adverse events.
This study was performed on 20 patients (19 women; median age 52 years). After add-on therapy with IVIG, 19 patients (95%) showed a significant reduction in the median ARR from 1.1 [interquartile range, 0.6‒1.4] to 0.3 [interquartile range, 0‒0.6] (p < 0.001). Seven patients (35%) were relapse-free during 43.5 months (median) of treatment. The median Expanded Disability Status Scale (EDSS) score remained stable at 4.0. In addition, 80% of the patients showed no disability progression, and 25% of the patients experienced an improvement in EDSS. The median NMO-IgG titer decreased from 1:480 (n = 19) to 1:120 (n = 13) after treatment (p = 0.006) and was found to be negative in three patients. Six patients (30%) stopped IVIG due to relapses after 27.5 months (median; interquartile range, 15.3‒43.3) of IVIG initiation. There were no severe side effects that led to discontinuation of IVIG.
IVIG as add-on therapy may be associated with beneficial effects in preventing relapse and disability progression in NMO-IgG-positive NMOSD patients who have breakthrough disease activity despite immunosuppressive treatment with azathioprine. Further randomized controlled trials are necessary to validate the efficacy of IVIG in NMOSD.
尽管静脉注射免疫球蛋白 (IVIG) 的作用机制使其成为一种合理的治疗策略，但在视神经脊髓炎谱系疾病 (NMOSD) 中的疗效却鲜有研究。本研究旨在评估 IVIG 作为一种附加疗法，联合硫唑嘌呤用于预防 NMOSD 复发的疗效。
我们回顾性分析了接受 IVIG 输注（0.4g/kg/天）治疗的 NMO-IgG 阳性 NMOSD 患者的病历，这些患者在接受硫唑嘌呤治疗超过 6 个月后，将 IVIG 作为联合治疗的一部分，每 1-3 个月输注一次。治疗效果和安全性根据 IVIG 治疗前后的年化复发率（ARR）变化、无复发和无进展患者的比例以及不良反应来评估。
本研究共纳入 20 例患者（19 名女性；中位年龄 52 岁）。在添加 IVIG 治疗后，19 例患者（95%）ARR 中位数从 1.1[四分位距（IQR）：0.6-1.4]降至 0.3[IQR：0-0.6]（p<0.001）。7 例患者（35%）在治疗 43.5 个月（中位时间）期间无复发。扩展残疾状况量表（EDSS）评分中位数保持稳定在 4.0。此外，80%的患者无残疾进展，25%的患者 EDSS 评分有所改善。19 例患者的 NMOSD-IgG 滴度中位数从 1:480（n=19）降至 1:120（n=13）（p=0.006），3 例患者 NMOSD-IgG 转为阴性。6 例患者（30%）在开始 IVIG 治疗后 27.5 个月（中位数；IQR：15.3-43.3）因复发而停止 IVIG。无因严重不良反应而导致 IVIG 停药的情况。
对于在接受硫唑嘌呤免疫抑制治疗后仍有突破性疾病活动的 NMO-IgG 阳性 NMOSD 患者，IVIG 作为附加治疗可能与预防复发和残疾进展有关。有必要进行随机对照试验来验证 IVIG 在 NMOSD 中的疗效。
