Duration of extracorporeal therapy in acute maple syrup urine disease: a kinetic model
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Maple syrup urine disease (MSUD, MIM 248600) can be complicated by metabolic crises necessitating extracorporeal removal therapy (ECRT). Since leucine levels are usually not immediately available during therapy, an accurate kinetic model of leucine plasma levels during removal would be useful to establish the duration of ECRT. Such a kinetic model is available for neonates undergoing continuous ECRT (CECRT) with a leucine clearance ≥35 ml min−1 1.73 m−2. The current study tests the validity of this model in older children. Plasma leucine levels were obtained from eleven ECRT sessions [seven CECRT and four intermittent hemodialysis (HDi) sessions] in seven children aged 1–14 years. No hemodynamic instability or neurological complications were observed during treatment. HDi provided a higher leucine clearance and required shorter sessions than CECRT (5.4±0.6 vs. 17.1±6.0 h). All patients regained precrisis neurological status except for one patient who had severe neurological damage (severe cerebral edema) at the time of dialysis and subsequently died despite efficient leucine removal. A leucine clearance ≥50 ml min−1 1.73 m−2 is required to obtain a kinetic model similar to that reported in neonates, both with CECRT and HDi. This model should be helpful in predicting the duration of therapy needed to attain desired leucine levels.
KeywordsMaple syrup urine disease Hemofiltration Hemodialysis Kinetic modelling Leucine clearance Amino acid metabolism Inborn error of metabolism