Comparison of Plasmapheresis and Intravenous Immunoglobulin as Maintenance Therapies for Juvenile Myasthenia Gravis
Comparison of Plasmapheresis and Intravenous Immunoglobulin as Maintenance Therapies for Juvenile Myasthenia Gravis
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DOI：
10.1001/jamaneurol.2014.17
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发表时间：
2014-05-01
期刊：
影响因子：
29
通讯作者：
Kang, Peter B.
中科院分区：
文献类型：
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作者：
Liew, Wendy K. M.;Powell, Christine A.;Kang, Peter B.
青少年重症肌无力（MG）是一种相对罕见的自身免疫性疾病。血浆置换术（PLEX）与免疫球蛋白作为维持治疗的疗效比较尚不清楚，目的是确定PLEX或静脉注射免疫球蛋白（IVIG）治疗是否是更有效的维持治疗在这种疾病。这项回顾性分析超过33-一年的时间涉及54名儿童和青少年与青少年MG在一个专门的神经肌肉诊所和肌电图实验室在三级保健学术儿科医院。干预和IVIG。主要结果和测量对治疗的反应通过客观体格检查结果的改善和患者报告的症状和功能能力的改善来衡量。PLEX和IVIG的应答率都很高。在接受PLEX、IVIG或两种治疗的27例全身性青少年MG患者中，7例接受PLEX单独治疗的患者中有7例应答，10例接受IVIG单独治疗的患者中有5例应答，10例接受两种治疗的患者中有9例应答。接受PLEX与IVIG的患者之间的应答率存在显著差异（P= 0.04）。通过外周静脉通路开始PLEX的最小年龄为9岁，而接受IVIG的最小儿童为9个月大。胸腺切除术进行了17名儿童，其中11人经历了显着的术后improvementation.CONCLUSIONS和RELEVANCE本研究提供了III级证据，PLEX和IVIG都有很高的反应率作为维持治疗，是合理的治疗选择青少年MG。在这种情况下，血浆置换术的反应率可能比IVIG更一致。这些研究结果将提供一些指导的方法来治疗青少年MG，特别是因为结果有所不同，从那些研究重点是成人MG。
IMPORTANCE Juvenile myasthenia gravis (MG) is a relatively rare autoimmune disorder. The comparative efficacy of plasmapheresis (PLEX) vs immunoglobulin as maintenance therapy is unclear for this childhood disease.OBJECTIVE To determine whether PLEX or intravenous immunoglobulin (IVIG) therapy is more effective as maintenance therapy in this disease.DESIGN, SETTING, AND PARTICIPANTS This retrospective analysis over a 33-year period involved 54 children and adolescents with juvenile MG at a specialized neuromuscular clinic and electromyography laboratory at a tertiary care academic pediatric hospital.INTERVENTIONS Plasmapheresis and IVIG.MAIN OUTCOMES AND MEASURES Response to treatment was measured by both improvement in objective physical examination findings and the patients' reported improvement in symptoms and functional abilities.RESULTS Subjective and objective outcomes correlated well. Both PLEX and IVIG had high response rates. Of the 27 patients with generalized juvenile MG receiving PLEX, IVIG, or both treatments, 7 of 7 patients treated with PLEX alone responded, 5 of 10 patients treated with IVIG alone responded, and 9 of 10 patients who received both responded. There was a significant difference in response rates between patients who received PLEX vs IVIG (P=.04). The youngest age at which PLEX was initiated via peripheral venous access was 9 years, while the youngest child who received IVIG was 9 months old. Thymectomy was performed in 17 children, of whom 11 experienced significant postoperative improvement.CONCLUSIONS AND RELEVANCE This study provides class III evidence that PLEX and IVIG both have high response rates as maintenance therapies and are reasonable therapeutic options for juvenile MG. Plasmapheresis may have a more consistent response rate than IVIG in this setting. These findings will provide some guidance regarding the approach to therapy for juvenile MG, especially as the results differ somewhat from those of studies focusing on adult MG.