Qualitative Assessment of the Symptoms and Impact of Pancreatic Exocrine Insufficiency (PEI) to Inform the Development of a Patient-Reported Outcome (PRO) Instrument.
Pancreatic exocrine insufficiency (PEI) affects patients with chronic pancreatitis (CP) and cystic fibrosis (CF) who produce insufficient digestive pancreatic enzymes. Common symptoms include steatorrhoea, diarrhea, and abdominal pain.



The objective of the study was to develop and test the content validity of a patient-reported outcome (PRO) instrument assessing PEI symptoms and their impact on health-related quality of life.



Instrument development was supported by a literature review, expert physician interviews (n = 10: Germany 4, UK 3, France 3), and exploratory, qualitative, concept-elicitation interviews with patients with CF and CP with PEI (n = 61: UK 29, Germany 18, France 14) and expert physicians (n = 10). Cognitive debriefing of the draft instrument was then performed with patients with PEI (n = 37: UK 24, Germany 8, France 5), and feasibility was assessed with physicians (n = 3). For all interviews, verbatim transcripts were qualitatively analysed using thematic analysis methods and Atlas.ti computerized qualitative software. All themes were data driven rather than a priori.



Patient interviews elicited symptoms and impacts not reported in the literature. Six symptom concepts emerged: pain, bloating, bowel symptoms, nausea/vomiting, eating problems, and tiredness/fatigue. Six impact domains were also identified. A 45-item instrument was developed in English, French, and German for testing in cognitive debriefing patient interviews. Following cognitive debriefing, 18 items were deleted.



Rigorous qualitative patient research and expert clinical input supported development of a PEI-specific PRO with the potential to aid management and monitoring of unmet needs among patients with PEI. The next step is to perform psychometric evaluation of the resulting instrument.
Department of Surgery, University of Southampton and University Hospital Southampton NHS Foundation Trust, Tremona Road, Southampton, Hampshire, SO16 6YD, UK.
Colin D
CD
Johnson
Patient-Centered Outcomes, Adelphi Values, Adelphi Mill, Bollington, Cheshire, SK10 5JB, UK.
Rob
R
Arbuckle
Patient-Centered Outcomes, Adelphi Values, Adelphi Mill, Bollington, Cheshire, SK10 5JB, UK.
Nicola
N
Bonner
UK National Institute for Health Research Southampton Respiratory Biomedical Research Unit, Southampton Children's Hospital and University Hospital Southampton NHS Foundation Trust, Tremona Road, Southampton, Hampshire, SO16 6YD, UK.
Gary
G
Connett
Department of Gastroenterology, University Hospital of Santiago de Compostela, Santiago de Compostela, Spain.
Enrique
E
Dominguez-Munoz
Service de Pancréatologie-Gastroentérologie, Pôle des Maladies de l'Appareil Digestif, APHP DHU UNITY, Université Denis Diderot-Paris VII Hôpital Beaujon, 92118, Clichy Cedex, France.
Philippe
P
Levy
Cystic Fibrosis Department, Charité University Hospital, Augustenburger Platz 1, 13353, Berlin, Germany.
Doris
D
Staab
Patient-Centered Outcomes, Adelphi Values, Adelphi Mill, Bollington, Cheshire, SK10 5JB, UK. nicola.williamson@adelphivalues.com.
Nicola
N
Williamson
Department of Medicine A, University Medicine Greifswald, Sauerbruchstrasse, 17489, Greifswald, Germany.
Markus M
MM
Lerch
D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D002648:Child; D003550:Cystic Fibrosis / Q000150:complications; D005060:Europe; D010188:Exocrine Pancreatic Insufficiency / Q000209:etiology / Q000503:physiopathology* / Q000523:psychology*; D005260:Female; D006304:Health Status; D006801:Humans; D007407:Interviews as Topic; D008297:Male; D008603:Mental Health; D008875:Middle Aged; D050500:Pancreatitis, Chronic / Q000150:complications; D000071066:Patient Reported Outcome Measures; D011594:Psychometrics; D036301:Qualitative Research; D011788:Quality of Life; D012720:Severity of Illness Index; D011795:Surveys and Questionnaires / Q000592:standards*; D055815:Young Adult
Chronic Pancreatitis; Cognitive Debriefing; Cystic Fibrosis; Expert Physician; Pancreatic Exocrine Insufficiency