Department of Surgery, University of Southampton and University Hospital Southampton NHS Foundation Trust, Tremona Road, Southampton, Hampshire, SO16 6YD, UK.
Patient-Centered Outcomes, Adelphi Values, Adelphi Mill, Bollington, Cheshire, SK10 5JB, UK.
Patient. 2017 Oct;10(5):615-628. doi: 10.1007/s40271-017-0233-0.
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.
胰腺外分泌不足（PEI）影响慢性胰腺炎（CP）和囊性纤维化（CF）患者，这些患者产生的消化性胰腺酶不足。常见症状包括脂肪泻、腹泻和腹痛。
本研究旨在开发和测试一种评估 PEI 症状及其对健康相关生活质量影响的患者报告结局（PRO）工具，并检验其内容效度。
通过文献回顾、对 10 名专家医生（德国 4 名，英国 3 名，法国 3 名）的访谈，以及对 61 名患有 PEI 的 CF 和 CP 患者（英国 29 名，德国 18 名，法国 14 名）和专家医生（英国 10 名）的探索性、定性、概念启发式访谈，支持工具的开发。随后对 37 名患有 PEI 的患者（英国 24 名，德国 8 名，法国 5 名）进行了草案工具的认知性审阅，对 3 名医生（均来自英国）进行了可行性评估。对所有访谈，使用主题分析方法和 Atlas.ti 计算机化定性软件对逐字记录进行了定性分析。所有主题均来自数据而非先验。
患者访谈中出现了文献中未报告的症状和影响。出现了 6 个症状概念：疼痛、腹胀、肠道症状、恶心/呕吐、进食问题和疲劳/乏力。还确定了 6 个影响领域。开发了一份包含 45 个项目的英文、法文和德文工具，用于认知性审阅患者访谈。认知性审阅后，删除了 18 个项目。
严格的定性患者研究和专家临床投入支持了具有潜在潜力的 PEI 特异性 PRO 的开发，有助于管理和监测 PEI 患者的未满足需求。下一步是对由此产生的工具进行心理测量评估。
