title: Efficacy and Safety of a New Formulation of Pancrelipase (Ultrase MT20) in the Treatment of Malabsorption in Exocrine Pancreatic Insufficiency in Cystic Fibrosis.
abstract: Background. Pancreatic enzyme replacement therapy is the standard of care for treatment of malabsorption in patients with cystic fibrosis (CF) and exocrine pancreatic insufficiency (PI). Aim. To evaluate efficacy and safety of a new formulation of pancrelipase (Ultrase MT20) in patients with CF and PI. Coefficients of fat absorption (CFA%) and nitrogen absorption (CNA%) were the main efficacy parameters. Safety was evaluated by monitoring laboratory analyses, adverse events (AEs), and overall signs and symptoms. Methods. Patients (n = 31) were randomized in a crossover design comparing this pancrelipase with placebo during 2 inpatient evaluation periods (6-7 days each). Fat and protein/nitrogen ingestion and excretion were measured from food diaries and 72-hour stool collections. CFA% and CNA% were calculated for each period and compared. Results. Twenty-four patients provided analyzable data. This pancrelipase increased mean CFA% and CNA% (+34.7% and +25.7%, resp., P < .0001 for both), reduced stool frequency, and improved stool consistency compared with placebo. Placebo-treated patients reported more AEs, with gastrointestinal symptoms being the most frequently reported AE. Conclusions. This pancrelipase is a safe and effective treatment for malabsorption associated with exocrine PI in patients with CF.
authors.affiliation: Cystic Fibrosis Center, Rainbow Babies & Children's Hospital and Case Western Reserve University School of Medicine, 11100 Euclid Avenue, Cleveland, OH 44106, USA.
authors.forename: Michael W
authors.initials: MW
authors.lastname: Konstan
authors.forename: Theodore G
authors.initials: TG
authors.lastname: Liou
authors.forename: Steven D
authors.initials: SD
authors.lastname: Strausbaugh
authors.forename: Richard
authors.initials: R
authors.lastname: Ahrens
authors.forename: Jamshed F
authors.initials: JF
authors.lastname: Kanga
authors.forename: Gavin R
authors.initials: GR
authors.lastname: Graff
authors.forename: Kathryn
authors.initials: K
authors.lastname: Moffett
authors.forename: Susan L
authors.initials: SL
authors.lastname: Millard
authors.forename: Samya Z
authors.initials: SZ
authors.lastname: Nasr
authors.forename: Edith
authors.initials: E
authors.lastname: Siméon
authors.forename: Jean
authors.initials: J
authors.lastname: Spénard
authors.forename: Josée
authors.initials: J
authors.lastname: Grondin