Pancreatic Enzyme Supplementation as Acid‐Resistant Microspheres versus Enteric‐Coated Granules in Cystic Fibrosis

Abstract

In order to compare the efficacy of pancreatic enzyme supplementation as pH-sensitive enteric-coated microspheres Pancrease to that of conventional supplementation with enteric-coated Pancreatin in cystic fibrosis, a double blind cross-over study was conducted. Eleven patients under 12 years of age received each of the enzyme preparations for four weeks. Treatment efficacy was evaluated by means of a symptom score card recording stool frequency, consistency, colour, odour, abdominal cramps and appetite as well as a 3 days fat absorption test. Weight increments were recorded 3 months before the study when patients were on Pancreatin, and 3 months after the study when patients were on Pancrease. In eight of the patients fat absorption was improved on Pancrease, but the difference did not reach statistical significance. However, the patients experienced significantly less dyspeptic symptoms, decreased stool frequency, better appetite and increments in weight were significantly higher on Pancrease compared to Pancreatin.

EUR-1008 pancreatic enzyme replacement is safe and effective in patients with cystic fibrosis and pancreatic insufficiency

“…There were no drug-related SAEs or discontinuations, nor were there any incidences of hyperuricemia or unexpected adverse trends in laboratory analyses following treatment with EUR-1008. As with previous studies of PEPs in young children [22][23][24][25][26], no age-related issues were noted with EUR-1008. However, these safety data may be limited by the short length of the study period (two weeks), which is too brief to detect AEs such as fibrosing colonopathy (not observed in this study).…”

“…This is significant, as weight loss during infancy or childhood correlates with poor adult pulmonary status in CF patients [5][6][7][8][9][10][11]. The decision to not use a placebo treatment or washout period in the design of the supplemental study is consistent with the practice reported in other published trials involving young CF patients [22][23][24][25][26].…”

“…Guidelines from the FDA now require that new PEPs be formulated to meet the label-claimed enzyme content and demonstrate safety and efficacy in human clinical trials [21], including trials in young children. Although there appear to be no age-related issues with PEP treatment in children b7 years of age [22][23][24][25][26], little safety data has been published in this patient group. The majority of literature on PEP usage in cystic fibrosis has focused on adolescents and adults.…”

Pancreatic enzyme replacement therapy for young cystic fibrosis patients

“…Young children achieve sufficient growth and weight gain more easily when only small amounts of fat and energy are lost in the stool. Increases in weight were larger during treatment with an acid resistant enzyme preparation than after an uncoated pancreatin formulation [18]. The present study with 4 weeks duration demonstrated no loss of body weight with either treatment but was too short to evaluate body weight development for clinical relevance.…”

Neonatal screening for cystic fibrosis is beneficial even in the context of modern treatment

“…Here we compare our cross-sectional data for CF populations aged 1 to 9 years in 2002 and discuss their outcomes in the context of NBS studies performed elsewhere. [9][10][11][12] We focused on patients in whom CF was diagnosed after 1994 who were treated with acid resistant enzymes 19 and determined whether NBS afforded any significant clinical benefit.…”