BACKGROUND Most patients with cystic fibrosis (CF) suffer from pancreatic insufficiency (PI), leading to fat malabsorption, malnutrition, abdominal discomfort and impaired growth. Pancreatic enzyme replacement therapy (PERT) is effective, but evidence based guidelines for dose adjustment are lacking. A mobile app for self-management of PERT was developed in the context of the HORIZON 2020 project MyCyFAPP. It contains an algorithm to calculate individual PERT-doses for optimal fat digestion, based on in vitro and in vivo studies carried out in the same project. In addition, the app includes a symptoms diary, educational material, and it is linked to a web tool allowing health care professionals to evaluate patient's data and provide feedback. METHODS A 6-month open label prospective multicenter interventional clinical trial was performed to assess effects of using the app on gastro-intestinal related quality of life (GI QOL), measured by the CF-PedsQL-GI (shortened, CF specific version of the Pediatric Quality of Life Inventory, Gastrointestinal Symptoms Module). RESULTS One hundred and seventy-one patients with CF and PI between 2 and 18 years were recruited at 6 European CF centers. Self-reported CF-PedsQL-GI improved significantly from month 0 (M0) (84.3, 76.4-90.3) to month 6 (M6) (89.4, 80.35-93.5) (p< 0.0001). Similar improvements were reported by parents. Lower baseline CF-PedsQL-GI was associated with a greater improvement at M6 (p < 0.001). CONCLUSIONS The results suggest that the MyCyFAPP may improve GI QOL for children with CF. This tool may help patients to improve self-management of PERT, especially those with considerable GI symptoms.

中文翻译：

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使用移动应用程序进行胰酶替代疗法的自我管理与改善患有囊性纤维化的儿童的胃肠相关生活质量有关

背景大多数囊性纤维化（CF）患者患有胰腺功能不全（PI），导致脂肪吸收不良、营养不良、腹部不适和生长受损。胰酶替代疗法 (PERT) 是有效的，但缺乏基于证据的剂量调整指南。在 HORIZON 2020 项目 MyCyFAPP 的背景下开发了一个用于 PERT 自我管理的移动应用程序。它包含一种算法，可根据在同一项目中进行的体外和体内研究来计算最佳脂肪消化的单个 PERT 剂量。此外，该应用程序还包括症状日记、教育材料，并链接到一个网络工具，允许医疗保健专业人员评估患者的数据并提供反馈。方法 进行了一项为期 6 个月的开放标签前瞻性多中心介入临床试验，以评估使用该应用程序对胃肠相关生活质量 (GI QOL) 的影响，通过 CF-PedsQL-GI（缩短的、CF 特定版本的儿科生活质量量表，胃肠道症状模块）。结果 在 6 个欧洲 CF 中心招募了 171 名 2 至 18 岁的 CF 和 PI 患者。自我报告的 CF-PedsQL-GI 从第 0 个月 (M0) (84.3, 76.4-90.3) 到第 6 个月 (M6) (89.4, 80.35-93.5) 显着改善（p < 0.0001）。家长们也报告了类似的改善。较低的基线 CF-PedsQL-GI 与 M6 的更大改善相关（p < 0.001）。结论 结果表明，MyCyFAPP 可能会改善 CF 儿童的 GI QOL。