Quantitative Assessment of Response to Long-Term Treatment with Intravenous Immunoglobulin in Patients with Stiff Person Syndrome.
Quantitative Assessment of Response to Long-Term Treatment with Intravenous Immunoglobulin in Patients with Stiff Person Syndrome.
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DOI：
10.1002/mdc3.13261
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发表时间：
2021-08
影响因子：
4
通讯作者：
Jacob S
中科院分区：
文献类型：
--
作者：
Bose S;Thompson JP;Sadalage G;Karim A;Jacob S
僵硬人综合征（SPS）是一种自身免疫性疾病，涉及针对脊髓抑制突触的几种成分的抗体，其中谷氨酸脱羧酶抗体是主要的免疫标记物。每年每百万人中约有1名SPS患者。静脉注射免疫球蛋白（IVIG）的作用已经确立，但关于常规免疫球蛋白长期疗效的研究有限。使用患者报告的调查问卷，回顾SPS和相关综合征的临床细节和长期治疗反应。根据经典临床症状、自身免疫特征和神经生理变化（达拉卡标准）从三级神经免疫学诊所确定患者。他们在治疗后进行随访，以评估IVIG的反应。共有23例患者符合选择标准。患者的人口统计资料和临床表现与文献报道相似。IVIG治疗后4 - 10周内患者的功能能力（用改良Rankin量表[mRS]评估）和生活质量（QoL）均有显著改善（mRS前vs mRS后，P < 0.0001； QoL前vs后，P = 0.0003），并持续治疗5年（mRS前vs现mRS， P = 0.0003； QoL前vs现QoL， P = 0.0002）。本文描述了23例SPS及相关综合征患者的最大单中心经验之一，并首次使用患者报告的评分系统（伯明翰免疫调节治疗反应[BRIT]）建立了常规IVIG的长期疗效。在常规使用IVIG后的近5年内，生活质量和功能评分持续改善。建议使用BRIT评分来评估初始反应以及监测SPS免疫调节的持续改善。
Stiff person syndrome (SPS) is an autoimmune condition involving antibodies against several components of the inhibitory synapse in the spinal cord, with glutamic acid decarboxylase antibodies being the predominant immune marker. SPS affects approximately 1 patient per million population per year. The effect of intravenous immunoglobulin (IVIG) has been established, but studies on the long‐term efficacy of regular IVIG are limited. To review clinical details and long‐term treatment response using a patient‐reported questionnaire in SPS and related syndromes. Patients were identified from a tertiary neuroimmunology clinic based on classical clinical symptoms, autoimmune profiles, and neurophysiological changes (Dalakas criteria). They were followed up after treatment to assess the response to IVIG. A total of 23 patients fulfilled the selection criteria. Patients' demographic profiles and clinical presentations were akin to that reported in literature. There was significant improvement in the functional ability (assessed by the modified Rankin scale [mRS]) and quality of life (QoL) following treatment with IVIG within 4 to 10 weeks (pre‐mRS vs. post‐mRS, P < 0.0001; pre‐QoL vs. post‐QoL, P = 0.0003) and sustained after 5 years of treatment (pre‐mRS vs. present mRS, P = 0.0003; pre‐QoL vs. present QoL, P = 0.0002). This article describes one of the largest single‐center experiences of 23 patients with SPS and related syndromes and is the first to establish the long‐term efficacy of regular IVIG using a patient‐reported scoring system (Birmingham Response to Immunomodulatory Therapy [BRIT]). Consistent improvement in QoL and functional scores were seen over nearly 5 years after regular use of IVIG. It is recommended to use BRIT scores to assess the initial response as well as to monitor continued improvement to immunomodulation in SPS.
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DOI：
10.1212/nxi.0000000000000696
发表时间：
2020-05-01
影响因子：
8.8
作者：
Munoz-Lopetegi, Amaia;de Bruijn, Marienke A. A. M.;Titulaer, Maarten J.
通讯作者：
Titulaer, Maarten J.
影响因子：
--
作者：
McKeon, Andrew;Robinson, Maisha T.;Pittock, Sean J.
通讯作者：
Pittock, Sean J.
影响因子：
8.6
作者：
Gerschlager, W;Brown, P
通讯作者：
Brown, P
影响因子：
16.4
作者：
FLANAGAN, JC
通讯作者：
FLANAGAN, JC
影响因子：
6
作者：
Dalakas, MC
通讯作者：
Dalakas, MC