ePancrelipase for the Treatment of Exocrine Pancreatic Insufficiency Due to Chronic Pancreatitis: Rationale and Methodology of a Prospective, Observational, Multicenter, Cohort Study

Introduction: There is limited understanding what impact pancrelipase has on specific clinical symptoms and health-related quality of life (HrQoL) in patients with exocrine pancreatic insufficiency (EPI). In 2 randomized, placebo-controlled studies of patients with chronic pancreatitis (CP) and EPI treatment with pancrelipase consistently reduced stool frequency while changes in other symptoms were not consistent. Here we describe the methodology of a prospective, observational, multicenter cohort study with the objective to quantitate EPI symptom response and HrQoL changes to pancrelipase treatment in patients with chronic pancreatitis and EPI. Methods: Patients with probable, or definite chronic pancreatitis and EPI (FE1< 200 µg/g), and at least moderate severity of EPI symptoms based on the symptoms score of the PEI-Q are being enrolled at 7 pancreatology centers. The PEI-Q is a validated PRO for patients with CP or cystic fibrosis and who have EPI.1 After additional baseline assessments including PROMIS fatigue and depression short form questionnaires, vitals, and blood draw for nutrition markers, patients initiate pancrelipase treatment and are followed for 3 months (Figure 1). The primary endpoint is the mean change in the PEI-Q Total Symptom Score (SS) from baseline to 1 month of treatment with pancrelipase (Visit 2). Results: As of May 2023, 30 of up to 90 patients have enrolled and completed the baseline assessment. Most subjects are 60 years of age or older (range 37-78), female (59%), White (59%) and non-hispanic (94%). Median body weight was 150 lbs (range 120-202), and less than 1/3rd are current alcohol users (29%) and/or current smokers (29%). Diabetes was present in 29%, steatorrhea in 56%, and mean stool FE-1 was 111 µg/g stool. Conclusion: The study aims to expand our understanding of the real-world impact of pancrelipase on EPI-symptoms, HrQoL and nutritional state in patients with chronic pancreatitis. 1 Johnson CD, Williamson N, Janssen-van Solingen G, et al. Psychometric evaluation of a patient-reported outcome measure in pancreatic exocrine insufficiency (PEI). Pancreatology. 2019;19(1):182-190.Figure 1.: Study Design.