Ultrase MT12 and Ultrase MT20 in the treatment of exocrine pancreatic insufficiency in cystic fibrosis: safety and efficacy

Abstract

Summary Background : Cystic fibrosis causes exocrine pancreatic insufficiency, leading to malabsorption. Supplemental pancreatic enzyme therapy alleviates the concomitant malnutrition experienced by cystic fibrosis patients. It is recognized that patients experience variations in clinical response to different brands of enzymes. This has prompted the US Food and Drug Administration to require that enzyme supplements be subjected to New Drug Applications. Aim : To investigate the safety and efficacy of supplemental pancreatic enzyme therapy in cystic fibrosis subjects. Methods : We compared two doses of one formulation of enteric‐coated pancreatic enzymes: Ultrase MT12 (12 000 lipase units per capsule) and Ultrase MT20 (20 000 lipase units per capsule), to placebo in two separate safety and efficacy studies. Results : Mean total fat, protein and carbohydrate intake did not differ significantly between the groups. A significant difference in both fat and protein absorption occurred with the enzyme therapy groups. The Ultrase MT12 and Ultrase MT20 groups experienced a mean fat and protein absorption 79.4% and 83.8%, and 87.3% and 88.6%, respectively. No adverse events related to study drug were reported. Conclusions : This study further supports the use of enzymes to treat pancreatic insufficiency in cystic fibrosis. Excellent fat and protein absorption was achieved with minimal adverse events and safe doses.

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

“…A study of supplemental pancreatic enzyme therapy by Konstan et al compared two doses of pancrelipase (12,000 and 20,000 lipase units per capsule) to placebo [41]. Mean CFA and CNA was 79.4% and 83.8%, respectively, for the low-dose pancrelipase group, compared to 46.7% (p = 0.0002) and 58.4% (p = 0.0001), respectively, for placebo; mean CFA and CNA was 87.3% and 88.6%, respectively, for the high-dose pancrelipase group, versus 58.7% (p = 0.0001) and 62.9% (p = 0.0001), respectively, for placebo.…”

Dual core quantum dots for highly quantitative ratiometric detection of trypsin activity in cystic fibrosis patients

“…This observation is in good agreement with previous studies in the literature in which the CPA (coefficient of protein absorption) increased in the range 59-90% after supplementary pancreatic enzyme uptake. 44,45 Accordingly, the values obtained for these four particular patients can now be found in the CF homozygotic range (symbol (o) in Fig. 4).…”

Efficacy and safety of a unique enteric-coated bicarbonate-buffered pancreatic enzyme replacement therapy in children and adults with cystic fibrosis

“…Based on fat-balance studies measuring fat ingestion and fecal fat, the majority of people with CF and untreated EPI absorb <50% of ingested fat [9]. With EC-PERT, fat-balance studies that have not selected for responders demonstrate that the majority of people with CF and EPI can absorb >80% of ingested fat [10–13]. Healthy people without EPI absorb >90% [9].…”