C0007 - The Voice of the Patient With Exocrine Pancreatic Insufficiency Secondary to Chronic Pancreatitis: Preliminary Findings From a Patient-Driven Registry

Monday, October 24, 2022

3:00 PM – 5:00 PM ET

Location: Crown Ballroom

Presenting Author(s)

Jodie A. Barkin, MD

University of Miami, Miller School of Medicine
Miami, FL

Jodie A. Barkin, MD¹, Yasmin G. Hernandez-Barco, MD², Samer Al-Kaade, MD³, Rahul Pannala, MD⁴, Janine Twal, PharmD⁵, Valerie J. Powell, MS⁶, David C. Whitcomb, MD⁷
¹University of Miami, Miller School of Medicine, Miami, FL; ²Massachusetts General Hospital, Boston, MA; ³Mercy Clinic Gastroenterology, St. Louis, MO; ⁴Mayo Clinic Arizona, Phoenix, AZ; ⁵Aimmune Therapeutics, a Nestlé Health Science company, Bridgewater, NJ; ⁶CorEvitas, LLC, Arlington, VA; ⁷University of Pittsburgh, UPMC and Ariel Precision Medicine, Pittsburgh, PA

Introduction: Exocrine pancreatic insufficiency (EPI) remains underdiagnosed and undertreated despite its impact on quality of life (QoL) and increased morbidity/mortality. Chronic pancreatitis (CP) is the most common disease of the pancreas associated with EPI. The North American Pancreatitis Study 2 explored the natural history (including exocrine insufficiency) of recurrent acute pancreatitis (RAP) and CP; however, more data are needed on this subgroup. To better understand EPI, its impact on QoL/healthcare resources, and treatment effects, an innovative dual approach using a patient online community and patient-driven traditional registry was used to collect data from patients and their gastroenterologists on real-world experiences with EPI and pancreatic enzyme replacement therapy (PERT). Preliminary findings from the prospective registry are reported.

Methods: This is a longitudinal, noninterventional study in the US with a planned enrollment of 400 patients, spanning ~5 years. Adults with suspected/confirmed EPI, receiving PERT, and diagnosed with CP/RAP at enrollment are eligible. Assessments are collected at prespecified intervals. To understand impact of clinical practices and EPI clinical course, multivariate modeling will be used to analyze effects of medical history, comorbidities, and treatments on EPI history/progression, QoL, and healthcare resource use.

Results: As of February 22, 2022, 35 patients are enrolled. Median age is 62 years, and most patients are White (Table). Alcohol (susceptibility/progression) and diabetes mellitus were common etiologies. Abnormal fecal elastase-1 test, vitamin deficiency, and weight loss were frequently used evidence for EPI diagnosis. Steatorrhea was present some of the time in 14 patients; 12 never had steatorrhea. Patients reported PERT greatly (n=12) and somewhat (n=11) improved symptoms. Over the past 3 months, 10 patients reported ER visits; 7 needed hospitalizations.

Discussion: Enrolled patients with EPI are diverse in terms of demographics, etiologies, and clinical presentations. Initial findings include heterogeneous diagnostic methodologies, substantial but not overwhelming presence of steatorrhea, and improvement of symptoms with PERT in most patients. Early experience confirms feasibility of this data collection modality in those with CP; future analyses will help clarify areas of unmet needs in EPI management, patient experience, and patient/physician factors that may affect treatment success.

Characteristic	Total N=35
Patient demographics^a
Age (years), median (range)	62 (31–81)
Sex, n (%)
Male	16 (45.7)
Female	14 (40.0)
Race, n (%)
White	23 (65.7)
Black or African American	3 (8.6)
Asian	2 (5.7)
Other	2 (5.7)
Ethnicity, n (%)
Hispanic or Latino	6 (17.1)
Not Hispanic or Latino	24 (68.6)
Chronic pancreatic etiology^b,c, n (%)
Toxic-metabolic
Alcohol (susceptibility/progression)	13 (37.1)
Tobacco smoking	7 (20.0)
Hyperlipidemia (fasting >300 mg/dL, nonfasting >500 mg/dL)	5 (14.3)
Medications	3 (8.6)
Toxins, other	3 (8.6)
Toxins, other, not otherwise specified	3 (8.6)
Hypercalcemia (total calcium levels >12.0 mg/dL or 3 mmol/L)	2 (5.7)
Toxins, chronic kidney disease (stage 5, end-stage renal disease)	1 (2.9)
Not applicable	16 (45.7)
Metabolic, other
Diabetes mellitus	12 (34.3)
Not applicable	23 (65.7)
Idiopathic
Late ( >35 years of age)	12 (34.3)
Early (< 35 years of age)	2 (5.7)
Not applicable	21 (60.0)
Evidence of suspected/confirmed EPI diagnosis^c, n (%)
Abnormal fecal elastase-1 test	10 (28.6)
Vitamin deficiency	9 (25.7)
Weight loss	9 (25.7)
Clinical steatorrhea	7 (20.0)
Pancreatic function testing	3 (8.6)
Other	7 (20.0)
Not applicable	6 (17.1)
Clinical manifestations of EPI^c, n (%)
Gastrointestinal symptoms
Diarrhea/loose stool	22 (62.9)
Abdominal pain	22 (62.9)
Bloating	17 (48.6)
Flatulence	8 (22.9)
Steatorrhea	8 (22.9)
Not applicable	3 (8.6)
Nutritional
Vitamin D deficiency	15 (42.9)
Other vitamin/mineral deficiencies	8 (22.9)
Unintentional weight loss/difficulty gaining weight	8 (22.9)
Malnutrition	6 (17.1)
Other	1 (2.9)
Not applicable	17 (48.6)

^aData were unavailable for 5 patients at the time of data analysis.

^bSelected chronic pancreatic etiology; other categories not shown include genetic, autoimmune, recurrent and severe acute pancreatitis, obstructive, and other.

^cMultiple options may be selected for individual participants.

Abbreviation: EPI, exocrine pancreatic insufficiency.

Table: Table. Patient Demographics and Disease Characteristics at Baseline

Disclosures:

Jodie Barkin: AbbVie – Advisor or Review Panel Member. Aimmune Therapeutics, a Nestlé Health Science company – Consultant. CorEvitas, LLC – Advisor or Review Panel Member. Envara Health – Advisor or Review Panel Member. Medtronic – Advisor or Review Panel Member.

Yasmin Hernandez-Barco: Aimmune Therapeutics, a Nestlé Health Science company – Consultant. CorEvitas LLC – Advisory Committee/Board Member.

Samer Al-Kaade: AbbVie – Advisory Committee/Board Member. Abbvie – Speakers Bureau. Aimmune Therapeutics, a Nestlé Health Science company – Consultant, Medical Writing Support. HYDE, Love&Overby, LLP – Consultant.

Rahul Pannala: Aimmune Therapeutics, a Nestlé Health Science company, – Consultant.

Janine Twal: Aimmune Therapeutics, a Nestlé Health Science company – Employee.

Valerie Powell: Aimmune Therapeutics, a Nestlé Health Science company – Consultant. CorEvitas LLC – Employee.

David Whitcomb: Aimmune Therapeutics, a Nestlé Health Science company – Consultant. Ariel Precision Medicine – Stock Options. Collaborative Alliance for Pancreatic Education and Research – Leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid. Department of Defense – Grant/Research Support. National Pancreas Foundation – Leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid. NIH / NIDDK – Grant/Research Support. Novartis – Consultant. Provident Charter School for Dyslexia – Leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid. Regeneron – Consultant. U.S. Application No. 15/156,063 – Intellectual Property/Patents. U.S. Patent Application No. 16/700,014 – Intellectual Property/Patents. UptoDate – Royalties.

Jodie A. Barkin, MD¹, Yasmin G. Hernandez-Barco, MD², Samer Al-Kaade, MD³, Rahul Pannala, MD⁴, Janine Twal, PharmD⁵, Valerie J. Powell, MS⁶, David C. Whitcomb, MD⁷. C0007 - The Voice of the Patient With Exocrine Pancreatic Insufficiency Secondary to Chronic Pancreatitis: Preliminary Findings From a Patient-Driven Registry, ACG 2022 Annual Scientific Meeting Abstracts. Charlotte, NC: American College of Gastroenterology.