Baheer Abdulwahhab Saja, Ahmed Amna, Ben Omran Tawfeg
Department of Genetics, Sidra Medicine, Doha, QAT.
College of Health Sciences, Qatar University, Doha, QAT.
Cureus. 2025 Aug 23;17(8):e90845. doi: 10.7759/cureus.90845. eCollection 2025 Aug.
Carbonic anhydrase VA (CA-VA) deficiency is a rare mitochondrial disorder affecting bicarbonate-dependent metabolic pathways. It commonly presents in neonates with hyperammonemia, lactic acidosis, and metabolic crisis, often mimicking more prevalent urea cycle and organic acid disorders. This rare metabolic disorder has been reported across different populations, with cases documented worldwide. We report the case of a three-day-old full-term Indian female neonate presenting with lethargy and poor feeding. Initial labs revealed hyperammonemia, severe lactic acidosis, hypoglycemia, and ketosis. Despite ammonia scavenger therapy, hyperammonemia persisted, necessitating continuous renal replacement therapy. Metabolic workup suggested mitochondrial dysfunction. Genetic testing identified a homozygous exon 6 deletion in the CA5A gene, confirming CA-VA deficiency. She was discharged on carglumic acid and supplements, with clinical and biochemical improvement. CA-VA deficiency should be included in the differential diagnosis of neonatal metabolic decompensation, particularly in South Asian infants. Early intervention can lead to favorable outcomes, underscoring the importance of prompt metabolic and genetic evaluation.
碳酸酐酶VA(CA-VA)缺乏症是一种罕见的线粒体疾病,会影响依赖碳酸氢盐的代谢途径。它通常在新生儿中表现为高氨血症、乳酸酸中毒和代谢危机,常常类似于更常见的尿素循环和有机酸紊乱。这种罕见的代谢紊乱在不同人群中均有报道,全球范围内都有病例记录。我们报告了一例三天大的足月印度女新生儿,表现为嗜睡和喂养困难。初始实验室检查显示高氨血症、严重乳酸酸中毒、低血糖和酮症。尽管进行了氨清除剂治疗,但高氨血症仍持续存在,需要持续进行肾脏替代治疗。代谢检查提示线粒体功能障碍。基因检测在CA5A基因中发现了纯合外显子6缺失,确诊为CA-VA缺乏症。她出院时服用了卡谷氨酸和补充剂,临床和生化指标均有改善。CA-VA缺乏症应纳入新生儿代谢失代偿的鉴别诊断,特别是在南亚婴儿中。早期干预可带来良好的结果,凸显了及时进行代谢和基因评估的重要性。