Bose Smriti, Thompson Joseph P, Sadalage Girija, Karim Abid, Jacob Saiju
Department of Neurology University Hospitals Birmingham Birmingham United Kingdom.
Clinical Immunology Service University of Birmingham Birmingham United Kingdom.
Mov Disord Clin Pract. 2021 Jun 18;8(6):868-874. doi: 10.1002/mdc3.13261. eCollection 2021 Aug.
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, < 0.0001; pre-QoL vs. post-QoL, = 0.0003) and sustained after 5 years of treatment (pre-mRS vs. present mRS, = 0.0003; pre-QoL vs. present QoL, = 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.
僵人综合征（SPS）是一种自身免疫性疾病，涉及针对脊髓抑制性突触多种成分的抗体，其中谷氨酸脱羧酶抗体是主要的免疫标志物。SPS每年每百万人口中约影响1例患者。静脉注射免疫球蛋白（IVIG）的疗效已得到证实，但关于定期使用IVIG的长期疗效研究有限。
使用患者报告问卷回顾SPS及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学变化（达拉卡斯标准），从三级神经免疫学诊所确定患者。治疗后对他们进行随访，以评估对IVIG的反应。
共有23例患者符合入选标准。患者的人口统计学特征和临床表现与文献报道相似。IVIG治疗4至10周内，功能能力（通过改良Rankin量表[mRS]评估）和生活质量（QoL）有显著改善（治疗前mRS与治疗后mRS，<0.0001；治疗前QoL与治疗后QoL，=0.0003），且在治疗5年后仍持续改善（治疗前mRS与当前mRS，=0.0003；治疗前QoL与当前QoL，=0.0002）。
本文描述了23例SPS及相关综合征患者最大的单中心经验之一，并且是首个使用患者报告评分系统（伯明翰免疫调节治疗反应[BRIT]）确立定期使用IVIG长期疗效的研究。在定期使用IVIG近5年的时间里，生活质量和功能评分持续改善。建议使用BRIT评分评估初始反应以及监测SPS免疫调节的持续改善情况。
