摘要目的:比较卡谷氨酸（N-carbamylglutamate，NCG）与连续性肾脏替代疗法（continuous renal replacement therapy，CRRT）在有机酸血症（organic acidemia，OA）失代偿期高氨血症危象中的降氨效果，探讨NCG在OA失代偿期的疗效。方法:采用多中心回顾性研究，选择2024年1月至2025年2月广州医科大学附属妇女儿童医疗中心、泉州市妇幼保健院及南方医科大学珠江医院收治的OA合并高氨血症危象患儿为研究对象，根据治疗方式分为NCG组与CRRT组，比较两组患儿的基线特征、血氨下降速度及幅度，并分析NCG的剂量-效应关系。结果:共纳入10例患儿（丙酸血症3例，甲基丙二酸血症7例），NCG组、CRRT组各5例。CRRT组基线血氨水平高于NCG组［1 250.0（765.0，1 502.0）μmol/L比515.0（394.3，537.1）μmol/L］，但差异无统计学意义（ P=0.056）。治疗12 h后，CRRT组与NCG组血氨下降百分比差异无统计学意义（66.1%比55.2%， P=0.548）。两组在3 h［107.33 μmol/（L·h）比38.67 μmol/（L·h）］和6 h［57.17 μmol/（L·h）比37.92 μmol/（L·h）］内的降氨速度比较，差异亦无统计学意义（ P>0.05）。剂量-效应分析显示，NCG剂量与降氨速度呈中等正相关（ r=0.7），线性回归模型提示剂量可解释65%的降氨速度变异（ R2=0.65）。 结论:NCG是OA失代偿期的一种耐受性良好且有效的治疗方法，可能减少或避免CRRT治疗，未来需更大样本研究确定其有效药物剂量。

abstractsObjective:To compare the ammonia reduction efficacy of N-carbamylglutamate (NCG) and continuous renal replacement therapy (CRRT) during hyperammonemia crisis in decompensated organic academia (OA) and to study the therapeutic effects of NCG in this condition.Methods:From January 2024 to February 2025, patients with OA and hyperammonemia crisis admitted to three hospitals were retrospectively studied. The patients were assigned into NCG group and CRRT group according to treatment methods. Their baseline profiles, the speed and magnitude of ammonia reduction and dose-effect relationships of NCG were analyzed.Results:A total of 10 patients were enrolled [3 cases of propionic acidemia (PA) and 7 cases of methylmalonic acidemia (MMA)], including 5 cases in NCG group and 5 in CRRT group. The median baseline blood ammonia level was higher in CRRT group than NCG group [1 250.0 (765.0, 1 502.0) μmol/L vs. 515.0 (394.3, 537.1) μmol/L] but not statistically significant ( P=0.056). After 12 h of treatment, no significant differences of ammonia reduction existed between the two groups (66.1% vs. 55.2%, P=0.548). Meanwhile, at 3 h[107.33 μmol/(L·h) vs. 38.67 μmol/(L·h)] and 6 h [57.17 μmol/(L·h) vs. 37.92 μmol/(L·h)], the speeds of ammonia reduction showed no significant differences between the two groups ( P>0.05). Dose-effect analysis showed a moderate positive correlation between NCG dosage and the speed of ammonia reduction ( r=0.7) and linear regression indicated that dosage explained 65% of the variability in ammonia reduction speed ( R2=0.65). Conclusions:NCG is a well-tolerated and effective treatment for acute decompensated OA and may reduce or even avoid CRRT treatment. Further studies with larger sample sizes are needed to determine the effective range of NCG dosage.