Wiethoff Sarah, Xiromerisiou Georgia, Bettencourt Conceição, Kioumi Anna, Tsiptsios Iakovos, Tychalas Athanasios, Evaggelia Markousi, George Kaltsounis, Makris Vasileios, Hardy John, Houlden Henry
Department of Molecular Neuroscience, UCL Institute of Neurology and National Hospital for Neurology and Neurosurgery, London, UK; MRC Centre for Neuromuscular Diseases, UCL Institute of Neurology and National Hospital for Neurology and Neurosurgery, London, UK.
Department of Neurology, Papageorgiou Hospital, Thessaloniki, Greece.
J Neurol Sci. 2014 Apr 15;339(1-2):220-2. doi: 10.1016/j.jns.2014.01.034. Epub 2014 Feb 1.
We present a 70-year-old male patient of Greek origin with choreatic movements of the tongue and face, lower limb muscle weakness, peripheral neuropathy, elevated creatinephosphokinase (CPK), acanthocytosis and haemolysis in the absence of Kell RBC antigens with an additional Factor IX-deficiency. Genetic testing for mutations in the three exons of the XK gene revealed a previously unreported hemizygous single base-pair frameshift deletion at exon 1 (c.229delC, p.Leu80fs). In conclusion, we hereby describe a rare phenotype of a patient with McLeod syndrome which was discovered coincidentally during routine blood group testing and consecutively genetically confirmed.
我们报告了一名70岁的希腊裔男性患者,其出现舌部和面部的舞蹈样动作、下肢肌肉无力、周围神经病变、肌酸磷酸激酶(CPK)升高、棘红细胞增多症以及在缺乏凯尔红细胞抗原的情况下发生溶血,此外还存在因子IX缺乏。对XK基因三个外显子的突变进行基因检测发现,外显子1存在一个先前未报道的半合子单碱基对移码缺失(c.229delC,p.Leu80fs)。总之,我们在此描述了一名麦克劳德综合征患者的罕见表型,该表型在常规血型检测中偶然发现,并随后通过基因检测得以证实。