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AQP4 抗体阳性 NMOSD 发作时的血浆吸附疗法的疗效和安全性:一项倾向评分匹配队列研究。

Efficacy and safety of apheresis therapy in AQP4 antibody-positive NMOSD attack: A propensity score-matched cohort study.

机构信息

Center for Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.

China National Clinical Research Center for Neurological Diseases, Beijing, China.

出版信息

CNS Neurosci Ther. 2024 May;30(5):e14780. doi: 10.1111/cns.14780.

Abstract

OBJECTIVE

Plasma exchange (PE) and immunoadsorption (IA) are recognized as effective ways to treat attacks in AQP4 antibody-positive NMOSD, but high-quality evidence was lacking. To evaluate the efficacy and safety of PE/IA plus intravenous methylprednisolone (IVMP) in NMOSD attacks using propensity scores to match IVMP as control.

METHODS

Patients were from a prospective observational cohort study. Stratification and interval propensity score matching (PSM) were used to reduce selection bias by matching baseline characteristics (gender, age, time to IVMP, EDSS at attack) between PE/IA + IVMP and IVMP group (in a ratio of 1:2). The primary endpoint of efficacy was EDSS change at 6 months. Adverse events and changes in laboratory tests were recorded.

RESULTS

Four hundred and eleven attacks of 336 patients were screened for PSM, and 90 attacks (30 PE/IA + IVMP and 60 IVMP) were included in the analysis. There were significant differences in EDSS [6.25 vs. 6.75; IQR (4.50-8.38 vs. 5.00-8.00), p = 0.671] and visual acuity [median logMAR = 0.35 vs. 1.00; IQR (0.30-0.84 vs. 0.95-1.96), p = 0.002] change between two groups at 6 months. PE/IA + IVMP treatment demonstrated predictive capacity for good recovery as indicated by an area under the curve (AUC) of 0.726. Fibrinogen reduction was found during PE/IA + IVMP treatment [n = 15 (50.00%)], but no severe adverse events led to apheresis treatment discontinuation.

DISCUSSION

After PSM analysis, IVMP+PE/IA in acute attack of NMOSD achieved better and continuous improvement in neurological function within 6 months compared with IVMP alone. PE/IA treatment showed a good safety profile.

摘要

目的

血浆置换(PE)和免疫吸附(IA)已被认为是治疗 AQP4 抗体阳性 NMOSD 发作的有效方法,但缺乏高质量的证据。本研究采用倾向性评分匹配 IVMP 作为对照,评估 PE/IA+静脉注射甲基强的松龙(IVMP)治疗 NMOSD 发作的疗效和安全性。

方法

患者来自前瞻性观察队列研究。采用分层和区间倾向性评分匹配(PSM),通过匹配 PE/IA+IVMP 组和 IVMP 组之间的基线特征(性别、年龄、IVMP 时间、发作时 EDSS),以减少选择偏倚(比例为 1:2)。疗效的主要终点是 6 个月时 EDSS 的变化。记录不良事件和实验室检查的变化。

结果

对 336 例患者的 411 次发作进行 PSM 筛选,纳入 90 次发作(30 次 PE/IA+IVMP 和 60 次 IVMP)进行分析。两组在 EDSS [6.25 比 6.75;IQR(4.50-8.38 比 5.00-8.00),p=0.671]和视力[中位数 logMAR=0.35 比 1.00;IQR(0.30-0.84 比 0.95-1.96),p=0.002]的变化方面存在显著差异。PE/IA+IVMP 治疗在 6 个月时具有良好恢复的预测能力,曲线下面积(AUC)为 0.726。在 PE/IA+IVMP 治疗期间发现纤维蛋白原降低[n=15(50.00%)],但没有严重不良事件导致终止血浆置换治疗。

讨论

经过 PSM 分析,NMOSD 急性发作时,IVMP+PE/IA 与单独 IVMP 相比,在 6 个月内神经功能的改善更好且持续。PE/IA 治疗具有良好的安全性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f198/11126786/9c99df262d81/CNS-30-e14780-g004.jpg

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