Poothrikovil Rajesh P, Al Thihli Khalid, Al Futaisi Amna, Al Murshidi Fathiya
Department of Clinical PhysiologySultan Qaboos University Hospital , Muscat , Sultanate of Oman.
Department of Clinical GeneticsSultan Qaboos University Hospital , Muscat , Sultanate of Oman.
Neurodiagn J. 2019;59(3):142-151. doi: 10.1080/21646821.2019.1645549. Epub 2019 Aug 21.
Nonketotic hyperglycinemia (NKH) or glycine encephalopathy is an autosomal recessive disorder of glycine metabolism resulting in an excessive accumulation of glycine in all body tissues, including the central nervous system. It is caused by a biochemical defect in the glycine cleavage system and considered as a rare disorder with an estimated prevalence of 1:60,000. The neonatal form presents in the first few days of life with progressive encephalopathy, hypotonia, myoclonic jerks, hiccups, seizures, rapid progression to coma and often death due to central apnea. Surviving infants often have severe developmental delay and refractory seizures. Atypical forms of NKH present with heterogeneous and nonspecific disease course. Classical glycine encephalopathy usually carries a very poor prognosis. We describe two neonates who presented with neonatal encephalopathy, apnea, and progressive lethargy. Increased CSF glycine level along with an elevated CSF to plasma glycine ratio was suggestive of classic NKH. Burst suppression EEG and agenesis of the corpus callosum were supportive findings. Evolution of the EEG patterns and course of the disease are discussed in detail. Transient phases of clinical stabilization and normalized plasma biochemical results may not necessarily reflect the actual encephalopathic process. Serial EEGs are helpful to assess the efficacy of treatment and to modify the therapeutic approach.
非酮症高甘氨酸血症(NKH)或甘氨酸脑病是一种常染色体隐性遗传的甘氨酸代谢紊乱疾病,会导致包括中枢神经系统在内的所有身体组织中甘氨酸过度蓄积。它由甘氨酸裂解系统中的生化缺陷引起,被认为是一种罕见疾病,估计患病率为1:60,000。新生儿型在出生后的头几天出现进行性脑病、肌张力减退、肌阵挛性抽搐、打嗝、癫痫发作,迅速进展为昏迷,常因中枢性呼吸暂停而死亡。存活的婴儿通常有严重的发育迟缓及难治性癫痫。NKH的非典型形式表现为异质性和非特异性病程。典型的甘氨酸脑病通常预后很差。我们描述了两名表现为新生儿脑病、呼吸暂停和进行性嗜睡的新生儿。脑脊液甘氨酸水平升高以及脑脊液与血浆甘氨酸比值升高提示为典型NKH。爆发抑制脑电图和胼胝体发育不全是支持性发现。详细讨论了脑电图模式的演变和疾病进程。临床稳定的短暂阶段和血浆生化结果正常不一定反映实际的脑病过程。连续脑电图有助于评估治疗效果并调整治疗方法。