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Κυριακή 29 Μαΐου 2022

Carglumic acid in methylmalonic acidemia: Use of breast milk as an alternative vehicle to water

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Carglumic acid in methylmalonic acidemia: Use of breast milk as an alternative vehicle to water

We aim to report the use of breast milk (BM) as an alternative vehicle in a neonate with methylmalonic acidemia rejecting carglumic acid (NCG) diluted in water. The patient presented symptomatic acidemia and hyperammonemia and after refusal of oral NCG administration, the clinician consulted the Pharmacy Department for advice. Data sheet of NCG does not recommend administration in other vehicle than water. Consequently, a dissolution test was conducted in BM showing correct dissolution. The solution was well tolerated, and plasma ammonium concentrations remained within range in subsequent analytical controls.


Abstract

What Is Known and Objective

Carbaglu® or N-carbamylglutamate (NCG) is not recommended for administration in a vehicle other than water. We aim to report the use of breast milk (BM) as an alternative vehicle in a neonate rejecting NCG diluted in water.

Case Summary

A neonate diagnosed with methylmalonic acidemia presented symptomatology of acidemia and hyperammonemia. After the patient refused oral NCG administration, a dissolution test was conducted in BM showing correct dissolution. The NCG-BM solution was tolerated and plasma ammonium concentrations remained within range in subsequent analytical controls.

What Is New and Conclusion

BM as a vehicle for NCG is a safe and effective option for patients who refuse suspension in water and could lead to better treatment compliance in paediatric patients.

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