Flavier S, Rolland M O, Eude M, Fédou C, Brun J F, Maire I, Mercier J, Raynaud E
Service central de physiologie clinique, Unité d'exploration métabolique (CERAMM), Hôpital Lapeyronie, CHU de Montpellier et Inserm, équipe ERI 25 / EA 4202 Muscle et pathologies, Montpellier.
Ann Biol Clin (Paris). 2007 Sep-Oct;65(5):550-4.
McArdle's disease is a metabolic myopathy characterized by a myophosphorylase deficiency resulting in an inability to degrade glycogen stores. We report the case of a 48 years old patient who complained since adolescence of rest and exercise myalgias and presented a chronic increased plasma creatine kinase activity. First, a maximal exercise test was performed. This test demonstrated a quasi lack of rise of respiratory exchange ratio and of blood lactate, possibly due to a glycogenolytic/glycolytic pathway deficiency. Second, a biopsy of vastus lateralis muscle was performed using Bergström needle. As expected, the analysis of mitochondrial function was normal. The in vitro screening test of the glycogenolysis/glycolysis pathway showed a lack of lactate production in presence of glycogen substrate. The study of muscular metabolism of glycogen revealed a glycogen accumulation and a decrease of active and total phosphorylase activities. These data allowed us to diagnose a type V glycogenosis, or McArdle's disease. The patient appeared heterozygous for the most frequent mutation (p.R50X).
麦克尔迪氏病是一种代谢性肌病,其特征是肌磷酸化酶缺乏,导致无法降解糖原储备。我们报告了一例48岁患者,该患者自青少年期起就抱怨休息和运动时出现肌痛,且血浆肌酸激酶活性长期升高。首先,进行了最大运动试验。该试验显示呼吸交换率和血乳酸几乎没有升高,这可能是由于糖原分解/糖酵解途径缺陷所致。其次,使用贝格斯特龙针进行了股外侧肌活检。正如预期的那样,线粒体功能分析正常。糖原分解/糖酵解途径的体外筛选试验显示,在存在糖原底物的情况下缺乏乳酸生成。对肌肉糖原代谢的研究显示糖原积累以及活性和总磷酸化酶活性降低。这些数据使我们能够诊断出V型糖原贮积病,即麦克尔迪氏病。该患者在最常见的突变(p.R50X)方面表现为杂合子。