Pancreatic Enzyme Replacement Therapy Formulation Developed Specifically for Infants and Young Children Is Safe and Effective

Study results published in the Journal of Cystic Fibrosis show that a capsule-free preparation of pancreatin gastro-resistant granules (Creon Micro) is a safe and effective treatment for young children with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF).

Researchers in Russia enrolled 40 children with a confirmed CF diagnosis and EPI (21 patients age 2 years or under and 19 patients age 2-3) to receive treatment with Creon Micro “at a dose of 5000 lipase units per 120 mL of formula or breast feed, or 1000 lipase units/kg body weight/meal, according to the Cystic Fibrosis Foundation guidelines.” The minimum dose was 5000 lipase units/meal, increased in increments of 5000 lipase units/meal.

Maximum permitted dose was 2500 units/kg body weight per feeding, or 4000 units/g fat intake, or 10,000 lipase units/kg body weight/day.

During the study, participants were prohibited from using other pancreatic enzyme replacement therapy preparations, narcotics, anti-diarrheals, antispasmodics, and laxatives. However, patients were permitted the use of “medications influencing duodenal pH, gastric emptying, and bile secretion” if taken at a stable dose.

Researchers monitored patients for frequency and severity of adverse events (AEs). Treatment efficacy was assessed by measuring height/length and body weight at baseline, month 1, and month 3. Caregivers also used daily diaries to record clinical symptoms of stool frequency and stool consistency.

The authors reported that “changes from baseline after 3 months were observed in height/length (mean ± SD change 0.03 ± 0.02 m), weight (0.75 ± 0.61 kg), height/length-for-age (2.78 ± 12.87 percentile points; z-score 0.13 ± 0.48), weight-for-age (4.14 ± 9.42 percentile points; z-score 0.20 ± 0.39) and BMI-for-age (7.71 ± 17.98 percentile points; z-score 0.29 ± 0.65).”

Mean ± SD daily stool frequency was “2.1 ± 0.8 in the overall population, 2.2 ± 0.6 in patients <2 years and 1.8 ± 0.9 in those 2—3 years. The mean percentage of days without soft or watery stools was 64.8% in the overall population.”

In their discussion of these results, the authors noted “Creon Micro was well tolerated with a favourable safety profile in young children with EPI due to CF. There were no serious AEs or AEs leading to discontinuation. Of the most frequent AEs, respiratory tract infection, frequent bowel movements, and rhinitis are known complications of CF.”