(342) Epidemiology and Outcomes of Renal Replacement Therapy in Children With Inborn Errors of Metabolism

Introduction: Children with inborn errors of metabolism (IEMs) experience life-threatening metabolic crises requiring renal replacement therapy (RRT) in a pediatric intensive care unit (PICU) to clear toxic metabolites, minimize neurologic morbidity, and improve survival. Limited knowledge of these children’s epidemiology and outcomes come from small case series, so we aimed to provide novel multicenter data from 2019 to 2023 using a contemporary, quality-controlled database.

Methods: With local IRB and Virtual Pediatrics Systems (VPS, LLC; Los Angeles, CA) approval, the VPS “Virtual PICU” database was queried for children with an IEM (per the VPS “Star Code” diagnoses) < 18 years of age who received RRT in contributing PICUs from January 1, 2019, to December 31, 2023. Demographics, diagnoses, procedures, illness severity scores, and clinical outcomes including length of stay and mortality were extracted. Descriptive data reported as n (%) or median (IQR).

Results: Among the 1037 children with IEMs from 72 PICUs who underwent RRT, 57% were male, 44% white, and the age distribution was 11% aged 0-29d, 17% 29d-2y, 12% 2-6y, 20% 6-12y, and 40% 12-18y. The most common IEM Star Codes names were urea cycle disorders (30%), metabolism disorders (23%), and bilirubin excretion disorders (14%). The median PIM 3 score was -3.28 (-4.88 - -2.7). The median PRISM 3 score was 10 (5-15). The most common RRT modality was continuous renal replacement therapy (52%), followed by hemodialysis (42%), peritoneal dialysis (5%) and sustained low-efficiency dialysis (1%). The median time to RRT from hospital admission was 8.8 (1.4-33.8) days. The median RRT duration was 1.46 (0.15-5.56) days. The median hospital length of stay was 25.0 (9.3-55.9) days. Over one-quarter (28%) of the patients died.

Conclusions: We report the first contemporary multicenter description of RRT in children with IEM, describing care and outcomes of more than 1000 children. Further investigation is warranted to optimize RRT usage in children with IEM diagnoses.