Division of Pediatric Genetics, Metabolism, and Genomic Medicine, Department of Pediatrics, University of Michigan Health System, Ann Arbor, MI.
Consulting for Statistics, Computing and Analytics Research, University of Michigan, Ann Arbor, MI.
J Pediatr. 2022 Jul;246:116-122.e1. doi: 10.1016/j.jpeds.2022.03.043. Epub 2022 Mar 28.
To assess the outcomes of neonates in a contemporary multi-institutional cohort who receive renal replacement therapy (RRT) for hyperammonemia.
We performed a retrospective analysis of 51 neonatal patients with confirmed inborn errors of metabolism that were treated at 9 different children's hospitals in the US between 2000 and 2015.
Twenty-nine patients received hemodialysis (57%), 21 patients received continuous renal replacement therapy (41%), and 1 patient received peritoneal dialysis (2%). The median age at admission of both survivors (n = 33 [65%]) and nonsurvivors (n = 18) was 3 days. Peak ammonia and ammonia at admission were not significantly different between survivors and nonsurvivors. Hemodialysis, having more than 1 indication for RRT in addition to hyperammonemia, and complications during RRT were all risk factors for mortality. After accounting for multiple patient factors by multivariable analyses, hemodialysis was associated with a higher risk of death compared with continuous renal replacement therapy. When clinical factors including evidence of renal dysfunction, number of complications, concurrent extracorporeal membrane oxygenation, vasopressor requirement, and degree of hyperammonemia were held constant in a single Cox regression model, the hazard ratio for death with hemodialysis was 4.07 (95% CI 0.908-18.2, P value = .067). To help providers caring for neonates with hyperammonemia understand their patient's likelihood of survival, we created a predictive model with input variables known at the start of RRT.
Our large, multicenter retrospective review supports the use of continuous renal replacement therapy for neonatal hyperammonemia.
评估在接受肾脏替代疗法（RRT）治疗高氨血症的当代多机构队列中的新生儿的结局。
我们对 2000 年至 2015 年间在美国 9 家不同儿童医院接受治疗的 51 例确诊先天性代谢错误的新生儿患者进行了回顾性分析。
29 例患者接受血液透析（57%），21 例患者接受连续肾脏替代治疗（41%），1 例患者接受腹膜透析（2%）。幸存者（n=33[65%]）和非幸存者（n=18）的中位入院年龄均为 3 天。幸存者和非幸存者的峰值氨和入院时氨无显著差异。血液透析、除高氨血症外还有超过 1 个 RRT 适应证以及 RRT 期间的并发症均为死亡的危险因素。通过多变量分析考虑多个患者因素后，与连续肾脏替代治疗相比，血液透析与更高的死亡风险相关。当在单 Cox 回归模型中固定包括肾功能障碍证据、并发症数量、同时进行体外膜氧合、血管加压药需求和高氨血症程度等临床因素时，血液透析死亡的风险比为 4.07（95%CI 0.908-18.2，P 值=.067）。为了帮助治疗高氨血症的新生儿的提供者了解其患者的生存率，我们创建了一个预测模型，输入的变量是在开始 RRT 时已知的。
我们的大型多中心回顾性研究支持使用连续肾脏替代疗法治疗新生儿高氨血症。
