Pancreatic enzyme replacement therapy for young cystic fibrosis patients
Anne Munck1, Jean-Francois Duhamel, Thierry Lamireau
1Centre de Ressources et de Compétence pour la Mucoviscidose Hôpital Robert Debré, AP-HP, Paris, France. anne.munck@rdb.aphp.fr
Insights
Parents preferred Creon for children (CfC) over Creon 10000 (C10) for treating maldigestion in cystic fibrosis infants. Both enzyme therapies improved fat absorption similarly, supporting CfC for improved daily care.
Area of Science:
- Pediatric Gastroenterology
- Cystic Fibrosis Research
- Pancreatic Enzyme Therapy
Background:
- Maldigestion affects 90% of cystic fibrosis (CF) patients, necessitating pancreatic enzyme supplementation.
- Early identification of pancreatic insufficiency leads to enzyme therapy, even in breastfed infants.
- Creon for children (CfC) is a specialized infant pancreatic enzyme preparation with smaller granules.
Purpose of the Study:
- To compare parent preference between CfC and C10 in infants and toddlers.
- To evaluate the efficacy and safety of CfC versus C10 in treating maldigestion.
- To assess the impact on fat absorption and clinical symptoms.
Main Methods:
- A prospective, randomized, multi-center, cross-over study involving 40 infants and toddlers.
- Participants received both CfC and Creon 10000 (C10) for two weeks each.
- Primary endpoint: parent treatment preference. Secondary endpoints: coefficient of fat absorption (CFA), clinical symptoms, and safety.
Main Results:
- 51% of parents preferred CfC, while 23% preferred C10; 26% had no preference.
- Mean CFA was similar for both treatments (77.8% vs. 78.7%).
- Gastrointestinal symptoms and malabsorption laboratory parameters were observed; safety and tolerability were comparable.
Conclusions:
- Parental preference favored CfC for treating maldigestion in young cystic fibrosis patients.
- Both enzyme preparations demonstrated similar improvements in malabsorption.
- The study supports CfC for enhancing daily care in infants with cystic fibrosis, particularly those identified via neonatal screening.
Unlabelled:
Maldigestion in cystic fibrosis (CF) affects approximately 90% of patients. As soon as pancreatic insufficiency is identified, enzyme supplementation is prescribed even with breast fed infants. A pancreatic enzyme preparation developed particularly for infants, Creon for children (CfC), contains smaller granules to be administered with a dosing spoon (5000 lipase units per scoop).
Patients And Methods:
In a prospective, randomised, multi-centre study, 40 infants and toddlers received both CfC and Creon 10000 (C10) for two weeks each in a cross-over design. Dosing of pancreatic enzymes was continued as applied before the study. The primary endpoint was the parents' treatment preference. Secondary endpoints included coefficient of fat absorption (CFA), clinical symptoms and safety parameters.
Results:
20 parents (51%) from the N=39 intent to treat sample preferred CfC, 9 (23%) preferred C10, and 10 (26%) had no preference The applied doses led to a mean CFA with similar results for both treatments (77.8% vs. 78.7%). Gastrointestinal symptoms were reported on a number of study days, and some children had abnormal results for laboratory parameters of malabsorption. Safety and tolerability of the preparations were good and all these parameters were comparable for both treatments.
Conclusion:
Those parents who had a preference favoured CfC over C10. Both enzyme preparations improved malabsorption to a similar degree, although the applied dosages could have been too low in some children reflected in a suboptimal CFA. These data support the use of CfC for young patients with cystic fibrosis improving the daily care of this cohort detected mainly now through neonatal screening programmes.
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