Departments of General Pediatrics.
Pediatrics. 2014 Feb;133(2):e461-5. doi: 10.1542/peds.2012-1372. Epub 2014 Jan 27.
Mevalonate kinase deficiency (MKD) is a rare autosomal recessive disorder causing 1 of 2 phenotypes, hyperimmunoglobulin D syndrome and mevalonic aciduria, presenting with recurrent fever episodes, often starting in infancy, and sometimes evoked by stress or vaccinations. This autoinflammatory disease is caused by mutations encoding the mevalonate kinase (MVK) gene and is classified in the group of periodic fever syndromes. There is often a considerable delay in the diagnosis among pediatric patients with recurrent episodes of fever. We present a case of an 8-week-old girl with fever of unknown origin and a marked systemic inflammatory response. After excluding infections, a tentative diagnosis of incomplete Kawasaki syndrome was made, based on the finding of dilated coronary arteries on cardiac ultrasound and fever, and she was treated accordingly. However, the episodes of fever recurred, and alternative diagnoses were considered, which eventually led to the finding of increased excretion of mevalonic acid in urine. The diagnosis of MKD was confirmed by mutation analysis of the MVK gene. This case shows that the initial presentation of MKD can be indistinguishable from incomplete Kawasaki syndrome. When fever recurs in Kawasaki syndrome, other (auto-)inflammatory diseases must be ruled out to avoid inappropriate diagnostic procedures, ineffective interventions, and treatment delay.
标题:婴儿不明原因发热及冠状动脉扩张——Mevalonate 激酶缺陷症
摘要:Mevalonate 激酶缺陷症(MKD)是一种罕见的常染色体隐性遗传病,导致两种表型之一,即高免疫球蛋白 D 综合征和甲羟戊酸尿症,表现为反复发热发作,通常在婴儿期开始,有时由应激或疫苗接种引起。这种自身炎症性疾病是由编码甲羟戊酸激酶(MVK)基因的突变引起的,被归类为周期性发热综合征。儿科患者反复出现发热时,常常存在相当大的诊断延迟。我们报告了一例 8 周大的女孩,她出现不明原因发热和明显的全身炎症反应。在排除感染后,根据心脏超声发现扩张的冠状动脉和发热,我们做出了不完全川崎病的初步诊断,并进行了相应治疗。然而,发热再次发作,考虑了其他诊断,最终发现尿液中甲羟戊酸排泄增加。MVK 基因突变分析证实了 MKD 的诊断。该病例表明,MKD 的初始表现可能与不完全川崎病无法区分。当川崎病发热再次发作时,必须排除其他(自身)炎症性疾病,以避免不适当的诊断程序、无效的干预措施和治疗延误。