SPORADIC LATE-ONSET NEMALINE MYOPATHY AS A RARE CAUSE OF SLOWLY PROGRESSIVE MUSCLE WEAKNESS WITH YOUNG ADULT ONSET
SPORADIC LATE-ONSET NEMALINE MYOPATHY AS A RARE CAUSE OF SLOWLY PROGRESSIVE MUSCLE WEAKNESS WITH YOUNG ADULT ONSET
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DOI：
10.1002/mus.24509
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发表时间：
2015-05-01
期刊：
影响因子：
3.4
通讯作者：
Uesaka, Yoshikazu
中科院分区：
文献类型：
--
作者：
Maeda, Meiko Hashimoto;Ohta, Hikari;Uesaka, Yoshikazu
简介：散发性迟发性线状体肌病（SLONM）是一种罕见的难治性获得性肌病，以进行性肌无力和萎缩为特征，通常在中晚期成人发病。自体外周血造血干细胞移植（auto-PBSCT）是治疗SLONM的有效方法。研究方法：在这项研究中，我们进行了临床特征，肌肉组织病理学分析，并在自体外周血干细胞移植后的肌肉力量监测在一个27岁的艾滋病毒阴性的男子单克隆丙种球蛋白病。结果：他在接受大剂量美法仑和自体外周血干细胞移植治疗后显示出肌肉力量的改善。结论：考虑到最近成功治疗SLONM的报道，早期和正确诊断这种与单克隆丙种球蛋白病相关的疾病是很重要的。SLONM应被添加到疾病列表中，以考虑在鉴别诊断进行性肌无力与年轻成人发病。肌肉神经51：772-774，2015
Introduction: Sporadic late-onset nemaline myopathy (SLONM) is a rare intractable acquired myopathy characterized by progressive muscle weakness and atrophy, usually with middle to late adult onset. Autologous peripheral blood stem cell transplantation (auto-PBSCT) has been reported to be a promising treatment for SLONM. Methods: In this study we performed clinical characterization, muscle histopathological analysis, and muscle power monitoring after auto-PBSCT in a 27-year-old HIV-negative man with monoclonal gammopathy. Results: He showed improved muscle strength after treatment with high-dose melphalan and auto-PBSCT. Conclusions: Considering the recent reports of successful treatment of SLONM, early and correct diagnosis of this condition in association with monoclonal gammopathy is important. SLONM should be added to the list of diseases to consider in the differential diagnosis of progressive muscle weakness with young adult onset. Muscle Nerve51:772-774, 2015