Liprotamase Long‐term Safety and Support of Nutritional Status in Pancreatic‐insufficient Cystic Fibrosis

Abstract

Treatment with a mean of 5.5 capsules of liprotamase per day, during meals and snacks, for up to 12 months was safe, well tolerated, and associated with age-appropriate growth and weight gain or weight maintenance in subjects with CF-related EPI.

Clinical, anthropometric and laboratory nutritional markers of pancreatic exocrine insufficiency: Prevalence and diagnostic use

“…Changes in other nutritional parameters such as triglycerides were not seen. In clinical intervention studies in patients with CF and PEI, 1 showed improvements in total serum cholesterol, HDL and serum vitamins A and E after~30e44 days on PERT [51], whereas another found no clinically significant changes in albumin, prealbumin, fasting triglycerides, cholesterol or fat-soluble vitamins over 1 year of PERT [74]. However, it has to be taken into account that the patients participating in these trials were not required to be PERT-naïve and had received PERT prior to study entry.…”

“…Some have reported significantly lower mean concentrations versus healthy controls [73] whereas others reported values within normal ranges in patients on PERT [74] and higher than normal values in 48% of patients on PERT and vitamin supplements [75].…”

“…One group found no significant difference in the prevalence of vitamin K deficiency between PEI and non-PEI patients with and without PERT [58]. Vitamin K levels have also been studied in patients with CF and PEI; 1 study reported levels within the normal range [74] but several have reported low vitamin K levels despite concomitant PERT in the majority of cases [67,76e80] and vitamin K supplementation appears to have been only partially successful [67,76,79].…”

New and Emerging Treatments for Cystic Fibrosis

“…Liprotamase is a non-porcine pancreatic enzyme-replacement product that contains three different microbially derived and biotechnology-produced enzymes. Liprotamase appeared to be well tolerated in phase III studies, and the coefficient of fat absorption improved by 10 % compared with placebo [85,86]. However, in 2011, the US FDA questioned the evidence for efficacy of liprotamase and asked for further clinical studies before an approval for use could be granted; a further phase III study is now planned (NCT02279498).…”

New insights into gene-specific management in cystic fibrosis from the 2012 European Cystic Fibrosis Conference

“…Although long-term benefits of ivacftor in children (6-11 years of age), adolescent and adult patients with G551D mutations were presented at the conference (up to 96 weeks) [4][5][6], many questions remain including its role and safety in very young children (<6 years) and its role in the treatment of patients with other related ('gating' or class III) mutations. Studies to address these questions are about to commence in the USA and Europe.…”