■硬人综合征谱系障碍(SPSD)是一组罕见的致残神经免疫障碍。SPSD通常需要免疫疗法,特别是在对对症治疗反应不足的情况下。治疗性血浆置换(TPE)在SPSD中的安全性和有效性仍不确定。
■为了描述安全性,耐受性,和TPE对SPSD患者的疗效。
■一项回顾性观察性研究。
■对1997年至2021年在约翰霍普金斯医院(JHH)看到的SPSD患者进行了回顾性审查。患者人口统计学/病史,检查/诊断结果,治疗反应,记录TPE相关并发症。评估临床特征之间的任何关联,包括年龄,性别,临床表型,和免疫疗法的时间,治疗后3个月对TPE反应。在JHH接受TPE治疗的18例患者和在外部机构接受TPE治疗的6例患者的亚组在TPE治疗后3个月评估对症药物使用的任何变化。还对SPSD和TPE进行了文献综述。
■39例SPSD患者接受了TPE治疗(JHH21例,外部机构18例);中位年龄48岁,77%为女性,中位数改良Rankin量表3;平均初始抗GAD65抗体滴度为23,508U/mL。24例患者(62%)有经典SPS,10人(26%)有SPS+,2例(5%)出现进行性脑脊髓炎伴强直和肌阵挛性,和3(8%)有纯小脑共济失调。所有患者均接受对症治疗,30(77%)以前收到IVIg,和3(8%)以前接受利妥昔单抗。4名患者(10%)出现TPE相关不良事件。一个人出现了无症状的低血压,另一个患有线血栓和感染,2例发生了不危及生命的出血事件.23例(59%)患者报告TPE后症状改善。在24名患者的亚组中,TPE治疗后3个月评估了对症药物使用的任何变化,14(58%)需要更少的GABA能对症药物。文献综述确定了57例SPSD患者;43例(75%)报告TPE后暂时改善。
■大多数接受TPE治疗的患者有改善。此外,大多数患者在TPE治疗后评估对症药物使用的任何变化,在TPE治疗后数月不再需要同样多的对症药物治疗.TPE在SPSD中表现出安全和良好的耐受性。需要进一步的研究来评估TPE在SPSD中的长期疗效,并确定哪些患者可能从TPE中受益最大。
UNASSIGNED: Stiff person syndrome spectrum disorders (SPSD) are a rare group of disabling neuroimmunological disorders. SPSD often requires immune therapies, especially in the setting of inadequate response to symptomatic treatments. The safety and efficacy of therapeutic plasma exchange (TPE) in SPSD remains uncertain.
UNASSIGNED: To describe the safety, tolerability, and efficacy of TPE in patients with SPSD.
UNASSIGNED: A retrospective observational study.
UNASSIGNED: A retrospective review of SPSD patients seen at Johns Hopkins Hospital (JHH) from 1997 to 2021 was performed. Patient demographics/history, examination/diagnostic findings, treatment response, and TPE-related complications were recorded. Assessment for any associations between clinical characteristics, including age, sex, clinical phenotype, and time on immunotherapy, and response to TPE 3 months after treatment was performed. A subgroup of 18 patients treated with TPE at JHH and 6 patients treated with TPE at outside institutions were evaluated for any change in usage of symptomatic medications 3 months after the TPE treatment. Literature review of SPSD and TPE was also conducted.
UNASSIGNED: Thirty-nine SPSD patients were treated with TPE (21 at JHH and 18 at outside institutions); median age 48 years, 77% female, median modified Rankin Scale 3; mean initial anti-
GAD65 antibody titer was 23,508 U/mL. Twenty-four patients (62%) had classic SPS, 10 (26%) had SPS-plus, 2 (5%) had progressive encephalomyelitis with rigidity and myoclonus, and 3 (8%) had pure cerebellar ataxia. All patients were on symptomatic treatments, 30 (77%) previously received IVIg, and 3 (8%) previously received rituximab. Four patients (10%) had a TPE-related adverse event. One developed asymptomatic hypotension, another had both line thrombosis and infection, and two had non-life-threatening bleeding events. Twenty-three (59%) patients reported improvement in symptoms after TPE. Of the subgroup of 24 patients evaluated for any change in usage of symptomatic medications 3 months after the TPE treatment, 14 (58%) required fewer GABAergic symptomatic medications. Literature review identified 57 additional patients with SPSD; 43 (75%) reported temporary improvement after TPE.
UNASSIGNED: The majority of patients treated with TPE had improvement. Moreover, most patients evaluated for any change in usage of symptomatic medications after the TPE treatment no longer required as much symptomatic medications months after TPE. TPE appears safe and well-tolerated in SPSD. Further studies are needed to assess the long-term efficacy of TPE in SPSD and identify which patients may benefit the most from TPE.