Pancreatic enzyme replacement therapy for young cystic fibrosis patients.
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).


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.


CONCLUSIONS
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.
Munck
Anne
A
Centre de Ressources et de Compétence pour la Mucoviscidose Hôpital Robert Debré, AP-HP, Paris, France. anne.munck@rdb.aphp.fr
Duhamel
Jean-Francois
JF
Lamireau
Thierry
T
Le Luyer
Bernard
B
Le Tallec
Claire
C
Bellon
Gabriel
G
Roussey
Michel
M
Foucaud
Pierre
P
Giniès
Jean Louis
JL
Houzel
Anne
A
Marguet
Christophe
C
Guillot
Marcel
M
David
Valerie
V
Kapel
Nathalie
N
Dyard
François
F
Henniges
Friederike
F
D000284:Administration, Oral; D002675:Child, Preschool; D003258:Consumer Behavior; D018592:Cross-Over Studies; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000378:metabolism; D005260:Female; D005765:Gastrointestinal Agents / Q000008:administration & dosage*; D006801:Humans; D007223:Infant; D050356:Lipid Metabolism / Q000187:drug effects; D008297:Male; D008863:Microspheres; D020799:Pancrelipase / Q000008:administration & dosage*; D010290:Parents; D016896:Treatment Outcome
D005765:Gastrointestinal Agents; D020799:Pancrelipase