Clinical validation of an evidence-based method to adjust Pancreatic Enzyme Replacement Therapy through a prospective interventional study in paediatric patients with Cystic Fibrosis

• DOI: 10.1371/journal.pone.0213216
• Publication Date: 2019-03-12T17:31:21Z
• Authors: Joaquim Calvo-Lerma; Jessie Hulst; Mieke Boon; Carla Colombo; Etna Masip; Mar Ruperto; Victoria Fornés-F...; Els van der Wiel; Ine Claes; Maria Garriga; Maria Roca; Paula Crespo-Escobar; Anna Bulfamante; Sandra Woodcock; Sandra Martínez-B...; Ana Andrés; Kris de Boeck; Carmen Ribes-Koni...
• Keywords: Male; Adolescent; Cystic Fibrosis; PH; Science; CHILDREN; Pilot Projects; Body Mass Index; Feces; 03 medical and health sciences; Sex Factors; 0302 clinical medicine; Humans; Enzyme Replacement Therapy; Prospective Studies; Child; Pancreas; Science & Technology; Evidence-Based Medicine; Q; R; Age Factors; IN-VITRO; Lipase; Dietary Fats; Diet; 3. Good health; Multidisciplinary Sciences; EMC MM-04-54-07; Phenotype; Science & Technology - Other Topics; Medicine; Regression Analysis; Female; COEFFICIENT; Research Article

Background A method to adjust Pancreatic Enzyme Replacement Therapy in Cystic Fibrosis is not currently available. Objectives To assess the vivo efficacy of a dose enzymatic supplement CF extrapolated from previous vitro digestion studies (theoretical optimal dose, TOD). Secondly, how individual patient characteristics influence expected coefficient fat absorption (CFA) and thus identify an correction factor improve TOD. Methods prospective interventional study 43 paediatric patients with 5 European centres. They followed 24h fixed diet theoretical for each meal. Faecal collection was carried out between colorimetric markers order include all faeces corresponding diet. Beta regression models were applied associations CFA. Results Median CFA 90% (84, 94% 1st, 3rd Q.) no significant differences among Intestinal transit time positively associated (p = 0.007), but statistical found age, gender, phenotype or BMI. Regression model showed improvement predicted when taking into account. Conclusion Strict adherence prescribed meal, led median levels at clinical target low variability patients. The proposed can be considered as first approach evidence-based PERT dosing based on food characteristics. have confirmed free dietary settings.