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Subcutaneous Immunoglobulin for maintenance therapy in stiff-person syndrome: one-year follow-up in two patients
Neuromuscular Disorders ( IF 2.8 ) Pub Date : 2020-11-01 , DOI: 10.1016/j.nmd.2020.09.024
E Fileccia 1 , R Rinaldi 2 , G M Minicuci 3 , R D'Angelo 3 , L Bartolomei 4 , R Liguori 5 , V Donadio 1
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Stiff person syndrome is a rare condition characterised by prolonged stiffness with superimposed muscle spasms. Immunotherapy relies mainly on intravenous immunoglobulin, steroids and plasma exchange. Azathioprine or rituximab are other possible options. We describe two patients who showed a good clinical response with intravenous immunoglobulin and persistence of the clinical improvement after shifting to equivalent dosage of subcutaneous immunoglobulin. Both patients received a diagnosis of stiff person syndrome based on their clinical symptoms (episodes of stiffness and spasms) and presence of antiglutamic acid decarboxylase. Treatment with intravenous immunoglobulin was started with improvement of symptoms as reported by patients and confirmed also by the spasm frequency scale and modified Ashworth scale. After clinical stabilisation in order to avoid the hospitalisation required for intravenous immunoglobulin treatment a switch to subcutaneous immunoglobulins was made. After one year of follow-up from the switch, the patients show clinical stability. Their scores on the modified Ashworth scale, spasm frequency scale and on the 10 Meter Walking Test were also stable. Subcutaneous formulation of immunoglobulin could be as effective as intravenous immunoglobulin in the maintenance treatment of Stiff person syndrome, although studies involving a larger cohort of patients are needed in order to confirm our anecdotal experience.

中文翻译:

用于僵人综合征维持治疗的皮下免疫球蛋白:两名患者的一年随访

僵硬人综合征是一种罕见的疾病,其特征是长期僵硬并伴有肌肉痉挛。免疫治疗主要依靠静脉注射免疫球蛋白、类固醇和血浆置换。硫唑嘌呤或利妥昔单抗是其他可能的选择。我们描述了两名患者,他们在静脉注射免疫球蛋白后表现出良好的临床反应,并且在换用等效剂量的皮下免疫球蛋白后临床改善持续存在。根据他们的临床症状(僵硬和痉挛发作)和抗谷氨酸脱羧酶的存在,两名患者都被诊断为僵硬的人综合征。静脉注射免疫球蛋白治疗开始于患者报告的症状改善,并通过痉挛频率量表和改良的 Ashworth 量表证实。在临床稳定后,为了避免静脉注射免疫球蛋白治疗所需的住院治疗,转为皮下注射免疫球蛋白。经过一年的转换随访,患者表现出临床稳定性。他们在改良的 Ashworth 量表、痉挛频率量表和 10 米步行测试中的分数也很稳定。在僵人综合征的维持治疗中,皮下注射免疫球蛋白可能与静脉注射免疫球蛋白一样有效,尽管需要更多患者队列的研究来证实我们的轶事经验。他们在改良的 Ashworth 量表、痉挛频率量表和 10 米步行测试中的分数也很稳定。在僵人综合征的维持治疗中,皮下注射免疫球蛋白可能与静脉注射免疫球蛋白一样有效,尽管需要更多患者队列的研究来证实我们的轶事经验。他们在改良的 Ashworth 量表、痉挛频率量表和 10 米步行测试中的分数也很稳定。在僵人综合征的维持治疗中,皮下注射免疫球蛋白可能与静脉注射免疫球蛋白一样有效,尽管需要更多患者队列的研究来证实我们的轶事经验。
更新日期:2020-11-01
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