Background

Patients with cystic fibrosis (CF) and pancreatic insufficiency need pancreatic enzyme replacement therapy (PERT) for dietary lipids digestion. There is limited evidence for recommending the adequate PERT dose for every meal, and controlling steatorrhea remains a challenge. This study aimed to evaluate a new PERT dosing method supported by a self-management mobile-app.

Methods

Children with CF recruited from 6 European centres were instructed to use the app, including an algorithm for optimal PERT dosing based on in vitro digestion studies for every type of food. At baseline, a 24h self-selected diet was registered in the app, and usual PERT doses were taken by the patient. After 1 month, the same diet was followed, but PERT doses were indicated by the app. Change in faecal fat and coefficient of fat absorption (CFA) were determined.

Results

58 patients (median age 8.1 years) participated. Baseline fat absorption was high: median CFA 96.9%, median 2.4g faecal fat). After intervention CFA did not significantly change, but range of PERT doses was reduced: interquartile ranges narrowing from 1447-3070 at baseline to 1783-2495 LU/g fat when using the app. Patients with a low baseline fat absorption (CFA<90%, n=12) experienced significant improvement in CFA after adhering to the recommended PERT dose (from 86.3 to 94.0%, p=0.031).

Conclusion

the use of a novel evidence-based PERT dosing method, based on in vitro fat digestion studies incorporating food characteristics, was effective in increasing CFA in patients with poor baseline fat absorption and could safely be implemented in clinical practice.

中文翻译：

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基于食物特性的循证方法调整胰酶补充剂治疗囊性纤维化剂量的临床评价

背景

患有囊性纤维化 (CF) 和胰腺功能不全的患者需要胰酶替代疗法 (PERT) 来消化膳食脂质。推荐每餐摄入足够的 PERT 剂量的证据有限，控制脂肪泻仍然是一个挑战。本研究旨在评估一种由自我管理移动应用程序支持的新 PERT 给药方法。

方法

从 6 个欧洲中心招募的患有 CF 的儿童被指示使用该应用程序，其中包括基于每种食物的体外消化研究的最佳 PERT 剂量算法。在基线时，在应用程序中注册了 24 小时自选饮食，患者服用了常用的 PERT 剂量。1 个月后，遵循相同的饮食，但应用程序显示了 PERT 剂量。测定粪便脂肪和脂肪吸收系数 (CFA) 的变化。

结果

58 名患者（中位年龄 8.1 岁）参加。基线脂肪吸收高：中位 CFA 96.9%，中位 2.4g 粪便脂肪）。干预后 CFA 没有显着变化，但 PERT 剂量范围缩小：四分位间距从基线的 1447-3070 缩小到使用应用程序时的 1783-2495 LU/g 脂肪。基线脂肪吸收低的患者（CFA<90%，n=12）在坚持推荐的 PERT 剂量后 CFA 显着改善（从 86.3% 到 94.0%，p=0.031）。

结论

使用一种新的基于证据的PERT给药方法，基于结合食物特性的体外脂肪消化研究，可有效增加基线脂肪吸收不良患者的CFA，并可在临床实践中安全实施。