Introduction: There is limited understanding of the impact of pancrelipase on specific clinical symptoms and health-related quality of life (HrQoL) in real-world patients with exocrine pancreatic insufficiency (EPI). Here, we report results of our ongoing prospective, observational, multicenter cohort study.
Methods: Patients with probable, or definite chronic pancreatitis (CP) and EPI (fecal elastase-1 [FE1]<200 µg/g stool) and at least moderate severity of EPI symptoms based on the total symptoms score (TSS) of the Pancreatic Exocrine Insufficiency Questionnaire (PEI-Q), are being enrolled at 7 US pancreatology centers. The PEI-Q is an 18-item validated PRO for patients with CP or cystic fibrosis and who have EPI.¹ Study flow entails obtaining baseline assessments followed by initiating pancrelipase treatment (72k Lipase Units (LU)/meal and 36k LU/snack) and 3-month follow up information. Baseline information includes PROMIS fatigue and depression short form questionnaires as well as vital sign and serum nutritional markers. The primary endpoint is the difference between the mean PEI-Q TSS (13 items) at baseline and one month treatment with pancrelipase. Secondary outcomes include changes in HrQoL, stool frequency, and stool consistency (Bristol stool scale).
Results: As of November 2023, 26 and 21 patients were eligible for analyses at 1 month and 3 months follow-up, respectively. Characteristics for the n=26 patients: mean age of 59, 58% female, 54% white and median body weight of 158 lbs; 15% are current smokers, 19% current alcohol users. Diabetes was present in 59%, steatorrhea in 55%, and mean stool FE-1 was 90 µg/g stool. PEI-Q TSS improved from baseline to 1 month (mean ± SD): -0.8 ±0.9, p< 0.001, and baseline to 3 months: -1.1 ±0.6, p< 0.001, (Table 1). Pancrealipase treatment significantly improved HrQoL as measured by the PEI-Q impact domain (5 items) from baseline to 1 month -0.9 ±1.0, p<0.001 and baseline to 3 months -0.8 ±1.1, p<0.002, respectively (Table 1). Stool consistency improved towards normal , however mean stool frequency/day did not change at 1-month 0.2±1.6 p=0.552.
Conclusions: We demonstrate for the first-time significant and clinically meaningful improvements in EPI symptoms and HrQoL utilizing the validated PEI-Q in a real-world population of chronic pancreatitis patients with EPI who were treated with pancrelipase. Improvements in EPI symptoms and HrQoL as well as stool consistency were sustained through the 3-month observation period. The PEI-Q may represent a useful tool in the clinical management of EPI in patients with chronic pancreatitis and warrants further study.
¹Johnson 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.

PEI-Q = Pancreatic Exocrine Insufficiency Questionnaire; SD = standard deviation;<br /> 1. FAS 1: PEI-Q = Full Analytic Set of patients who have PEI-Q scores at baseline and at 1-month post-baseline.<br /> 2. FAS 2: PEI-Q = Full Analytic Set of patients who have PEI-Q scores at baseline and at 3 months post-baseline.<br /> 3. Baseline to month 1 difference and baseline to month 3 difference are the summary statistics of the patient-level paired difference of score between timepoints. A two-sided one sample t test was performed to assess if the mean difference (difference derived at the patient level) is significantly different from 0.

PEI-Q = Pancreatic Exocrine Insufficiency Questionnaire; SD = standard deviation;
1. FAS 1: PEI-Q = Full Analytic Set of patients who have PEI-Q scores at baseline and at 1-month post-baseline.
2. FAS 2: PEI-Q = Full Analytic Set of patients who have PEI-Q scores at baseline and at 3 months post-baseline.
3. Baseline to month 1 difference and baseline to month 3 difference are the summary statistics of the patient-level paired difference of score between timepoints. A two-sided one sample t test was performed to assess if the mean difference (difference derived at the patient level) is significantly different from 0.

Moming Li

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