Long-term efficacy, tolerability and retention rate of azathioprine in 103 aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder patients: a multicentre retrospective observational study from the UK
Long-term efficacy, tolerability and retention rate of azathioprine in 103 aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder patients: a multicentre retrospective observational study from the UK
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DOI：
10.1177/1352458514525870
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发表时间：
2014-10-01
影响因子：
5.8
通讯作者：
Jacob, Anu
中科院分区：
文献类型：
--
作者：
Elsone, Liene;Kitley, Joanna;Jacob, Anu
背景：硫唑嘌呤 (AZA) 是一种常见的免疫抑制药物，用于预防视神经脊髓炎 (NMO) 复发。目的：本文的目的是评估 AZA 在大型 NMO 队列中的疗效、耐受性和保留性。方法：我们对 103 例水通道蛋白 4 抗体阳性 NMO 和 NMO 的医疗记录进行了回顾性审查 接受 AZA 治疗的谱系障碍 (NMOSD) 患者。结果：这是报告的最大的接受 AZA 治疗的 AQP4-Ab 阳性患者队列。通过治疗，89% (n = 92) 的年化复发率中位数从 1.5 (IQR 0.6-4.0) 降低至 0 (IQR 0-0.27，p < 0.00005)。 61% (n = 63) 的中位随访时间为 18 个月，未复发。 78% 的神经功能改善或稳定。在最后一次随访时，46% 的患者停止治疗 (n = 47)。其中，62% (n = 29) 是因为副作用，19% (n = 9) 是因为死亡，15% (n = 7) 是因为持续的疾病活动，2% (n = I) 是因为怀孕。使用 Kaplan-Meyer 曲线，我们估计 73%、58%、47% 和 33% 的患者在开始治疗后将分别继续服用 AZA 超过 1 年、3 年、5 年和 10 年。结论：AZA 是 NMO 的一种适度有效的治疗方法。然而，许多患者随着时间的推移停用硫唑嘌呤，这似乎更多地反映了耐受性差而不是缺乏疗效。
Background: Azathioprine (AZA) is a common immunosuppressive drug used for relapse prevention in neuromyelitis optica (NMO).Objectives: The objective of this paper is to assess efficacy, tolerability and retention of AZA in a large NMO cohort.Methods: We conducted a retrospective review of medical records of 103 aquaporin-4 antibody-positive NMO and NMO spectrum disorder (NMOSD) patients treated with AZA.Results: This is the largest reported cohort of AQP4-Ab positive patients treated with AZA. Eighty-nine per cent (n = 92) had reduction in median annualised relapse rates from 1.5 (IQR 0.6-4.0) to 0 (IQR 0-0.27, p < 0.00005) with treatment. Sixty-one per cent (n = 63) remained relapse free at a median follow-up of 18 months. Neurological function improved or stabilised in 78%. At last follow-up, treatment was discontinued in 46% (n = 47). Of these, 62% (n = 29) were because of side effects, 19% (n = 9) because of death, 15% (n = 7) because of ongoing disease activity, and 2% (n = I) because of pregnancy. Using Kaplan-Meyer curves, we estimate that 73%, 58%, 47% and 33% of patients will remain on AZA for longer than one, three, five and 10 years, respectively, after initiation of treatment.Conclusions: AZA is a modestly effective treatment for NMO. However, many patients discontinue AZA over time and this seems to reflect poor tolerability more than lack of efficacy.