ENOrphanet Journal of Rare Diseases | Efficacy of CRRT in Children with Organic Acidemia Complicated by Decompensated Acidosis
Date: April 05, 2025
Classification: Frontiers
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This study evaluates the application of Continuous Renal Replacement Therapy (CRRT) in children with organic acidemia complicated by decompensated acidosis. The results show that CRRT can significantly reduce levels of multiple metabolic toxins and organ injury markers, accelerate recovery, and provide a novel intervention strategy for clinical treatment.
Literature Overview
The article titled 'Efficacy and organ protective effects of continuous renal replacement therapy in children with organic acidemia complicated by decompensated acidosis: a retrospective study in PICU', published in the Orphanet Journal of Rare Diseases, reviews and summarizes the therapeutic effects of CRRT in children with organic acidemia (OA) complicated by decompensated acidosis. By comparing children treated with conventional therapy between 2013 and 2018 with those receiving CRRT combined therapy between 2019 and 2024, the study found that CRRT significantly reduced levels of lactate, ammonia, creatinine, and NSE, and shortened the duration of mechanical ventilation and PICU hospitalization. The article also notes that organ injury markers in the CRRT group did not increase significantly during treatment, while those in the conventional therapy group showed an upward trend after 24 hours. The results support the early application of CRRT during acute metabolic crises in OA to reduce further organ damage and improve survival rates.Background Knowledge
Organic acidemia (OA) is a group of inherited metabolic disorders caused by abnormalities in amino acid, fatty acid, or carbohydrate metabolism. The major types include methylmalonic acidemia (MMA), propionic acidemia (PA), and glutaric acidemia type I (GA-1). Due to specific enzyme deficiencies, organic acids and their metabolites accumulate in the body, leading to severe metabolic acidosis, multi-organ damage, and even death. Acute metabolic decompensation in OA is often triggered by infection, vomiting, surgery, or trauma, and early symptoms are typically non-specific, leading to delayed diagnosis. Current standard treatments include intravenous nutritional support, correction of acid-base imbalance, coenzyme supplementation, and protein restriction, but these measures have limited effectiveness in severe cases. Continuous Renal Replacement Therapy (CRRT) is an extracorporeal blood purification technique capable of continuously removing metabolic waste, regulating electrolytes, and maintaining acid-base balance, making it theoretically suitable for children with OA experiencing acute metabolic crises. While existing guidelines recommend CRRT for emergency management of urea cycle disorders with hyperammonemia, studies on its application in OA remain limited. This study retrospectively analyzes clinical data from children receiving CRRT or conventional therapy to explore its therapeutic value and organ-protective effects during acute OA crises, offering reference for future clinical strategies.
Research Methods and Experiment
The study conducted a retrospective analysis of 46 children with OA and decompensated metabolic acidosis admitted to the pediatric intensive care unit (PICU) at Xinhua Hospital, Shanghai Jiao Tong University, from 2013 to May 2024. The patients were divided into two groups: conventional therapy group (admitted between 2013 and 2018 without CRRT) and CRRT therapy group (admitted after May 2019 and initiated CRRT within 12 hours of admission). All patients were diagnosed with OA based on tandem mass spectrometry (MS/MS) and genetic testing. Metabolic decompensation was defined as arterial pH < 7.25 and base deficit < -3. Clinical data, including gender, age, GCS score, PRISM III score, blood gas, lactate, ammonia, NSE, troponin, myoglobin, liver and kidney function, were collected and analyzed at 24 hours, 48 hours, and 5 days post-treatment. CRRT parameters included the use of AN69 filter, blood flow rate of 5 ml/kg/min, dialysate flow rate of 20-30 ml/kg/h, and modified Ports solution as replacement fluid. Statistical analysis was performed using SPSS 26.0, with normally distributed data expressed as mean ± standard deviation, and non-normally distributed data expressed as median and interquartile range (IQR). Inter-group comparisons were conducted using t-tests or Mann-Whitney U tests.Key Findings and Insights
Significance and Future Directions
This study is the first systematic evaluation of CRRT efficacy and multi-organ protective effects in children with OA complicated by decompensated acidosis. The results indicate that CRRT can serve as an early intervention during acute metabolic decompensation in OA, significantly reducing toxin accumulation, organ injury markers, and ICU stay. Although mortality did not differ significantly between groups, the CRRT group had fewer deaths and no serious adverse events. This study provides a new clinical intervention model, especially in the context of increasing CRRT equipment availability, and may improve acute OA management. Future studies should include multi-center, prospective trials to further validate CRRT’s effectiveness and explore optimal treatment timing, dosage, and combination strategies.
Conclusion
This retrospective study evaluated the application of CRRT in children with organic acidemia (OA) complicated by decompensated acidosis. The results showed that CRRT significantly reduced levels of lactate, ammonia, creatinine, and NSE during early treatment stages. Compared to the conventional therapy group, the CRRT group had shorter mechanical ventilation and PICU hospitalization durations and no serious adverse reactions. These findings suggest that CRRT provides organ protection, especially in terms of neurological, cardiac, and renal function. Although mortality did not significantly decrease, fewer deaths were observed in the CRRT group, primarily due to progressive infections rather than persistent metabolic acidosis. The study also highlights that organ injury markers in the conventional therapy group increased after 24 hours, while no such trend was observed in the CRRT group, indicating its advantage in early intervention. Given the low incidence of OA and the single-center retrospective design, future multi-center prospective studies are necessary to validate the optimal treatment timing, dosage, and combination strategies of CRRT in OA. This study provides a new therapeutic perspective for acute OA management, especially in the context of widespread extracorporeal purification equipment, offering significant clinical reference value.
