Long term use of Carglumic acid in patients with propionic and methylmalonic acidemia
DOI:
https://doi.org/10.54361/Keywords:
Carglumic acid, propionic acidemia, methylmalonic acidemiaAbstract
Objective: to evaluate the clinical long term use of carglumic acid in cases of propionic and methylmalonic acidemia Materials and Methods:13 patients diagnosed as PA, MMA at children hospital who are less than 2 years of age with no or minimal brain damage started on carglumic acid 50mg/kg/d divided 12h for at least 3 months were included in this study. Information’s were gathered from their medical files at Metabolic OPD during their regular visits and from medical files at paediatric/ ICU admissions if they encountered an acute crisis. Growth parameters, developmental assessment and frequency of admission due to crisis after treatment initiation were studied. In acute decomposition mean ammonia level, biochemical and haematological parameters and mean hospital stay were also observed. Results: 13 patients included in this study 8 propionic acidemia and 5 methylmallonic acidemia, started on Carglumic acid 50mg/kg/d for a period 3 to 9 months, 3 patients were admitted with metabolic decompensation, with average 2 days hospitalization. Conclusion: obvious decrease in no. Of hospitalizations due to metabolic decompensation as well as decrease in duration of hospital stay was noted in treated patients,Carglumic acid is well tolerated with no obvious side effects.
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