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MYH2 相关肌病:慢性进行性眼外肌麻痹(CPEO)临床谱的扩展。

MYH2-related Myopathy: Expanding the Clinical Spectrum of Chronic Progressive External Ophthalmoplegia (CPEO).

机构信息

Department of Neurology, National Institute of Mental Health and Neuro Sciences (NIMHANS), Bengaluru, Karnataka, India.

Department of Medicine, Children's Hospitalof Eastern Ontario Research Institute, University of Ottawa and Division of Neurology, The Ottawa Hospital, Ottawa, Canada.

出版信息

J Neuromuscul Dis. 2023;10(4):727-730. doi: 10.3233/JND-230017.

Abstract

Chronic progressive external ophthalmoplegia (CPEO) is symptom complex with progressive ptosis and restricted ocular motility without diplopia. MYH2 myopathy is rare disorder presenting with CPEO and muscle weakness. We report two Indian patients of MYH2 myopathy with unique features. Patient-1 presented with early adult-onset esophageal reflux followed by, proximal lower limb weakness, proptosis, CPEO without ptosis. He had elevated creatine kinase along with characteristic muscle MRI findings of prominent semitendinosus and medial gastrocnemius involvement. Patient -2 presented with early adult onset CPEO without limb weakness. His creatine kinase was normal. Both the patients had novel MYH2 mutations: a homozygous 5'splice variation in intron 4 (c.348 + 2dup) in patient 1 and homozygous single base pair deletion in exon 32 (p. Ala1480ProfsTer11) in patient 2. Unique features noted include adult onset, isolated CPEO, proptosis, esophageal reflux disease and absence of skeletal abnormalities. MYH2 myopathy has to be considered in adult patients with CPEO.

摘要

慢性进行性眼外肌麻痹(CPEO)是一种以进行性上睑下垂和眼球运动受限为特征的综合征,不伴有复视。MYH2 肌病是一种罕见的疾病,以 CPEO 和肌无力为特征。我们报告了两名具有独特特征的印度 MYH2 肌病患者。患者 1 表现为早发性成年发病的食管反流,随后出现近端下肢无力、眼球突出、CPEO 但无上睑下垂。他的肌酸激酶升高,同时伴有特征性的肌肉 MRI 表现,即半腱肌和内侧腓肠肌明显受累。患者 2 表现为早发性成年发病的 CPEO,无肢体无力。他的肌酸激酶正常。两名患者均存在新的 MYH2 突变:患者 1 在内含子 4 中存在纯合 5'剪接变异(c.348+2dup),患者 2 存在外显子 32 中纯合单碱基缺失(p.Ala1480ProfsTer11)。注意到的独特特征包括成年发病、孤立性 CPEO、眼球突出、食管反流病和无骨骼异常。在 CPEO 的成年患者中应考虑 MYH2 肌病。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e256/10357141/516b15c205d0/jnd-10-jnd230017-g001.jpg

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