Rationale for adequate enzyme replacement therapy for children with cystic fibrosis: a cross-sectional study

Background. The issues of choosing the most effective method for calculating pancreatin (lipase units per body weight and/or fat content of meals) and optimal doses of pancreatic enzymes in cystic fibrosis (CF) continue to be widely discussed among specialists. Objective. Estimation of the dose of pancreatin for children with CF using two calculation methods in order to develop an individual correction algorithm for enzyme replacement therapy. Methods. 140 children (80 boys, 60 girls) aged 1 to 18 years (mean age 6.4±5.2 years) with a confirmed diagnosis of CF and pancreatic deficiency (fecal elastase-1 < 200 pg/g) were examined. Children were divided into age groups: group 1 consisted of children aged 1 to 2 years (n=40); group 2 - 3 to 5 years (n=41); group 3 - 6 to 9 years (n=43,); group 4 - 10 through 18 years (n=26). Doses of pancreatin were evaluated by a questionnaire method using diet diaries, which included data on food consumption and the dose of pancreatin for 3 days. When comparing the actual dose of pancreatin with the recommended dose for CF, the recommendations of the ESPEN-ESPGHAN-ECFS expert group (2016) were considered to be reference. Results. The analysis showed that the daily amount of fat in meals statistically significantly increases from 64.1 to 104.0 g at the age of 1 to 18 years (p1-4<,0.001). At the same time, the dose of pancreatin (lipase units per day) in this group is statistically significantly reduced -from 10,620.0 to 7,000.0 U/kg (p1-4<,0.001). A similar trend was observed in the doses of pancreatin per the main and additional meals. The dose of pancreatin per main meals progressively decreased with age and was at the lower level of recommended values in children of the 4th (10-18 years) age group. Doses of pancreatin for additional meals (lunch, afternoon snack, meal at night) were particularly inadequate and did not exceed the lower level of recommended values in all age groups. Calculation of pancreatic enzymes by the second method using an estimate of the fat content in meal showed that in the general group of children, the dose of pancreatin was 1,500 U/g of fat in meal per day, which is lower than the recommended range of 2,000-4,000 U/g. Conclusion. The results obtained indicate the need to develop an individual approach algorithm for enzyme replacement therapy in children with CF using a more correct calculation method - for the actually consumed fat with meal.

cystic fibrosis, children, malabsorption syndrome, enzyme replacement therapy, method for calculating dosage of pancreatin, doses of pancreatin