MRC Centre for Neuromuscular Diseases, UCL Institute of Neurology and National Hospital for Neurology and Neurosurgery, Queen Square, London, United Kingdom.
Clinical Genetics Department, St George's University Hospitals NHS Foundation Trust, London, United Kingdom.
Neuromuscul Disord. 2020 Jul;30(7):566-571. doi: 10.1016/j.nmd.2020.05.004. Epub 2020 May 21.
LPIN1 mutations are a known common cause of autosomal recessive, recurrent and life-threatening acute rhabdomyolysis of childhood-onset. The first episode of rhabdomyolysis usually happens in nearly all cases before the age of 5 and death is observed in 1/3 of patients. Here we present two cases of acute rhabdomyolysis with a milder phenotype caused by LPIN1 mutation presenting in adolescence (11 years old) and adulthood (40 years old) after Parvovirus infection and metabolic stress, respectively. In our opinion, the mutation types, epigenetic factors, the environment exposition to triggers or the existence of proteins with a similar structure of LPIN1, may have a role in modulating the onset of rhabdomyolysis. LPIN1 should be included on a panel of genes analysed in the investigation of adult individuals with rhabdomyolysis. Metabolic and viral stressors should be included in the list of possible rhabdomyolysis precipitant.
LPIN1 突变是一种已知的常染色体隐性遗传、复发性和危及生命的儿童期发作的横纹肌溶解症的常见原因。横纹肌溶解症的首次发作几乎所有病例均发生在 5 岁之前,1/3 的患者观察到死亡。在这里,我们介绍了两例由 LPIN1 突变引起的急性横纹肌溶解症病例,其表型较轻,分别在青少年(11 岁)和成年(40 岁)时期由细小病毒感染和代谢应激引起。我们认为,突变类型、表观遗传因素、暴露于触发因素的环境或具有与 LPIN1 相似结构的蛋白质的存在,可能在调节横纹肌溶解症的发作中起作用。LPIN1 应包含在横纹肌溶解症成年患者的基因分析面板中。代谢和病毒应激源应包含在可能的横纹肌溶解症诱发因素列表中。