Clinical validation of an evidence-based method to adjust pancreatic enzyme replacement therapy through a prospective interventional study in paediatric patients with cystic fibrosis

on behalf of MyCyFAPP Project

Research output: Contribution to journalArticle

1 Citation (Scopus)

Abstract

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

Original languageEnglish
Article numbere0213216
JournalPLoS One
Volume14
Issue number3
DOIs
Publication statusPublished - Mar 1 2019

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Enzyme Replacement Therapy
Pediatrics
cystic fibrosis
prospective studies
Cystic Fibrosis
Fats
Prospective Studies
therapeutics
Enzymes
dosage
enzymes
lipids
Nutrition
Meals
PERT
Diet
methodology
gastrointestinal transit
in vitro digestion
Feces

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)
  • Agricultural and Biological Sciences(all)

Cite this

Clinical validation of an evidence-based method to adjust pancreatic enzyme replacement therapy through a prospective interventional study in paediatric patients with cystic fibrosis. / on behalf of MyCyFAPP Project.

In: PLoS One, Vol. 14, No. 3, e0213216, 01.03.2019.

Research output: Contribution to journalArticle

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abstract = "Background A method to adjust Pancreatic Enzyme Replacement Therapy in Cystic Fibrosis is not currently available. Objectives To assess the in vivo efficacy of a method to adjust the dose of enzymatic supplement in CF extrapolated from previous in vitro digestion studies (theoretical optimal dose, TOD). Secondly, to assess how individual patient characteristics influence the expected coefficient of fat absorption (CFA) and thus to identify an individual correction factor to improve TOD. Methods A prospective interventional study in 43 paediatric patients with CF from 5 European centres. They followed a 24h fixed diet with the theoretical optimal dose for each meal. Faecal collection was carried out between colorimetric markers in order to include all the faeces corresponding to the fixed diet. Beta regression models were applied to assess the associations of individual patient characteristics with the CFA. Results Median CFA was 90{\%} (84, 94{\%} 1 st , 3 rd Q.) with no significant differences among centres. Intestinal transit time was positively associated with CFA (p = 0.007), but no statistical associations were found with and age, gender, phenotype or BMI. Regression model showed no improvement of the in vitro predicted theoretical optimal dose when taking individual patient characteristics into account. Conclusion Strict adherence to the theoretical optimal dose of enzymatic supplement for a prescribed meal, led to median CFA levels at the clinical target of 90{\%} with a low variability between patients. The proposed method can be considered as a first approach for an evidence-based method in PERT dosing based on food characteristics. Results have to be confirmed in free dietary settings.",
author = "{on behalf of MyCyFAPP Project} and Joaquim Calvo-Lerma and Jessie Hulst and Mieke Boon and Carla Colombo and Etna Masip and Mar Ruperto and Victoria Forn{\'e}s-Ferrer and {Van Der Wiel}, Els and Ine Claes and Maria Garriga and Maria Roca and Paula Crespo-Escobar and Anna Bulfamante and Sandra Woodcock and Sandra Mart{\'i}nez-Barona and Ana Andr{\'e}s and {De Boeck}, Kris and Carmen Ribes-Koninckx",
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T1 - Clinical validation of an evidence-based method to adjust pancreatic enzyme replacement therapy through a prospective interventional study in paediatric patients with cystic fibrosis

AU - on behalf of MyCyFAPP Project

AU - Calvo-Lerma, Joaquim

AU - Hulst, Jessie

AU - Boon, Mieke

AU - Colombo, Carla

AU - Masip, Etna

AU - Ruperto, Mar

AU - Fornés-Ferrer, Victoria

AU - Van Der Wiel, Els

AU - Claes, Ine

AU - Garriga, Maria

AU - Roca, Maria

AU - Crespo-Escobar, Paula

AU - Bulfamante, Anna

AU - Woodcock, Sandra

AU - Martínez-Barona, Sandra

AU - Andrés, Ana

AU - De Boeck, Kris

AU - Ribes-Koninckx, Carmen

PY - 2019/3/1

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N2 - Background A method to adjust Pancreatic Enzyme Replacement Therapy in Cystic Fibrosis is not currently available. Objectives To assess the in vivo efficacy of a method to adjust the dose of enzymatic supplement in CF extrapolated from previous in vitro digestion studies (theoretical optimal dose, TOD). Secondly, to assess how individual patient characteristics influence the expected coefficient of fat absorption (CFA) and thus to identify an individual correction factor to improve TOD. Methods A prospective interventional study in 43 paediatric patients with CF from 5 European centres. They followed a 24h fixed diet with the theoretical optimal dose for each meal. Faecal collection was carried out between colorimetric markers in order to include all the faeces corresponding to the fixed diet. Beta regression models were applied to assess the associations of individual patient characteristics with the CFA. Results Median CFA was 90% (84, 94% 1 st , 3 rd Q.) with no significant differences among centres. Intestinal transit time was positively associated with CFA (p = 0.007), but no statistical associations were found with and age, gender, phenotype or BMI. Regression model showed no improvement of the in vitro predicted theoretical optimal dose when taking individual patient characteristics into account. Conclusion Strict adherence to the theoretical optimal dose of enzymatic supplement for a prescribed meal, led to median CFA levels at the clinical target of 90% with a low variability between patients. The proposed method can be considered as a first approach for an evidence-based method in PERT dosing based on food characteristics. Results have to be confirmed in free dietary settings.

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