Researchers at the Research Centre for Medical Genetics in Russia and Abbott Laboratories in Russia and Germany have recently published in the Journal of Cystic Fibrosis their results on the efficacy and safety of pancreatic therapy in infants with exocrine pancreatic insufficiency (EPI) related to cystic fibrosis (CF). The study is entitled “Safety and efficacy of Creon® Micro in children with exocrine pancreatic insufficiency due to cystic fibrosis.”
CF is a life-threatening genetic disease in which a defective gene causes the body to form unusually thick, sticky mucus that is difficult to cough up and can result in serious respiratory and gastrointestinal manifestations. 80 to 90% of patients with CF develop EPI, which is a condition that results from a deficiency in the digestive enzymes produced by the pancreas, causing an inability to digest food properly (maldigestion) and malabsorption of nutrients. EPI can lead to malnutrition, poor growth and development, negatively affecting CF outcome and the survival rate. An early diagnosis of both CF and EPI and adequate therapy is therefore crucial.
EPI standard treatment is based on pancreatic enzyme replacement therapy (PERT) combined with nutritional management. One PERT indicated for EPI treatment in CF patients is Creon® (Abbot Laboratories). Creon® Micro corresponds to a capsule-free preparation especially developed for infants.
In this study, a prospective open-label, multicenter trial (NCT01747330) was conducted in eight centers in Russia to assess the efficacy, safety and ease-of-use of Creon Micro in children, aged between one month and less than four years, with EPI due to CF. The study was performed during a 3-month period and growth parameters like height/length and weight were assessed. In total, 40 infants (mean age 26.5 months) completed the trial.
Researchers did not encounter serious adverse side effects that led to discontinuation of the treatment, although 40% of the infants experienced mild adverse effects, namely respiratory tract infection, frequent bowel movements, stomatitis (inflammation of mouth and lips), rhinitis (inflammation of the mucous membrane inside the nose), nasopharyngitis (the common cold) and diarrhea. Overall, after the 3-month treatment, growth parameters like height/length-for-age, weight-for-age and body mass index (BMI)-for-age were all increased. 95% of the caregivers considered the treatment easy to administer and 90% reported a good or very good acceptance.
Researchers concluded that Creon Micro is an age-appropriate pediatric formulation for EPI that is well tolerated by infants with CF, leading to a general increase in growth development parameters. Treatment with Creon Micro was also found to be easy to use and well accepted by caregivers and children.