Garibaldi Matteo, Pennisi Elena Maria, Bruttini Mirella, Bizzarri Veronica, Bucci Elisabetta, Morino Stefania, Talerico Caterina, Stoppacciaro Antonella, Renieri Alessandra, Antonini Giovanni
Unit of Neuromuscular Diseases, Department of Neurology Mental Health and Sensory Organs (NESMOS), Faculty of Medicine and Psychology, Sapienza University of Rome, Rome, Italy.
Department of Neurology, San Filippo Neri Hospital, Rome, Italy.
Neuromuscul Disord. 2015 Nov;25(11):869-72. doi: 10.1016/j.nmd.2015.08.011. Epub 2015 Sep 7.
A 69-year-old woman presented a dropped head, caused by severe neck extensor weakness that had started two years before. She had also developed a mild degree of dysphagia, rhinolalia, eyelid ptosis and proximal limb weakness during the last months. EMG revealed myopathic changes. Muscle MRI detected fatty infiltration in the posterior neck muscles and tongue. Muscle biopsy revealed fiber size variations, sporadic rimmed vacuoles, small scattered angulated fibers and a patchy myofibrillar network. Genetic analysis revealed homozygous (GCN)11 expansions in the PABPN1 gene that were consistent with recessive oculopharyngeal muscular dystrophy (OPMD). There are a few reports of the recessive form, which has a later disease onset with milder symptoms and higher clinical variability than the typical dominantly inherited form. This patient, who is the first Italian and the eighth worldwide reported case of recessive OPMD, is also the first case of OPMD with dropped-head syndrome, which thus expands the clinical phenotype of recessive OPMD.
一名69岁女性出现头部下垂,这是由两年前开始的严重颈部伸肌无力引起的。在过去几个月里,她还出现了轻度吞咽困难、鼻音、眼睑下垂和近端肢体无力。肌电图显示肌病性改变。肌肉磁共振成像检测到颈部后部肌肉和舌部有脂肪浸润。肌肉活检显示纤维大小不一、散在的边缘空泡、散在的小角形纤维和片状肌原纤维网络。基因分析显示聚腺苷酸结合蛋白核1基因(PABPN1)存在纯合子(GCN)11扩增,这与隐性眼咽型肌营养不良症(OPMD)相符。关于隐性形式的报道较少,与典型的显性遗传形式相比,其发病较晚,症状较轻,临床变异性更高。该患者是意大利首例、全球第八例报道的隐性OPMD病例,也是首例伴有头部下垂综合征的OPMD病例,从而扩展了隐性OPMD的临床表型。