Mobile PERT App Helps Improve Quality of Life for Children With CF, Study Shows

Mobile PERT App Helps Improve Quality of Life for Children With CF, Study Shows
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MyCyFAPP, a mobile app designed to help patients with cystic fibrosis (CF) manage their pancreatic enzyme replacement therapy (PERT) regimen, can improve the quality of life of children with the disease, particularly those with severe gastrointestinal (GI) symptoms, study says.

The study, “Use of a mobile application for self-management of pancreatic enzyme replacement therapy is associated with improved gastro-intestinal related quality of life in children with Cystic Fibrosis,” was published in the Journal of Cystic Fibrosis.

Statistics indicate that most CF patients (80–90%) develop pancreatic insufficiency, a condition in which the pancreas is unable to produce enough enzymes to help digest food. This leads to several GI problems, including malnutrition, fat malabsorption, abdominal discomfort, and impaired growth.

PERT is a form of treatment in which artificial enzymes are used to compensate for those lacking in CF patients and alleviate GI symptoms. Although PERT is known to be effective, guidelines for adjusting therapy doses are inadequate.

“The lack of an evidence-based tool to improve PERT, leads to low treatment efficacy: patients often base their PERT on habitual doses. If the dose is not properly adjusted, every single meal can contribute to discomfort and ultimately to malnutrition,” the researchers wrote.

This led to the development of a mobile app called MyCyFAPP intended to provide patients with a tailor-made tool that could determine the optimal PERT dose they should take after each meal in real time.

The app, which was developed as part of the HORIZON 2020 project, contains a mathematical algorithm to calculate PERT doses for optimal fat digestion based on data from several preclinical studies. MyCyFAPP also includes educational materials and a symptoms diary, and it also is linked to a web tool that allows physicians to evaluate patient data and provide feedback.

Researchers are now reporting the findings of the final part of the MyCyFAPP project aimed at evaluating the impact of the mobile app on GI symptoms, nutritional status, overall well-being, and quality of life (QOL) of children with CF.

“This investigation is essential before implementation of the app in clinical practice can be considered. We hypothesize that QOL, general well-being and nutritional status improve after using the app for at least 3 months,” the investigators wrote.

A total of 171 children with CF and pancreatic insufficiency, ages 2–18, were recruited from six European CF centers to participate in a six-month, open-label clinical trial. Their quality of life was assessed based on the CF-specific version of the Pediatric Quality of Life Inventory, Gastrointestinal Symptoms Module (CF-PedsQL-GI), a questionnaire containing 54 questions to be answered by children ages 5–18, or with the help of parents if needed for younger children.

Findings revealed that CF-PedsQL-GI scores reported by children improved significantly from the beginning of the study (median 84.3) to the sixth month of the trial (89.4). Higher scores indicate a better quality of life. Although self-reported CF-PedsQL-GI scores were higher at the third month (85.2) of the study compared with initial scores, the differences were insufficient to be considered statistically significant.

Similar analyses in parents revealed statistically significant improvements in CF-PedsQL-GI scores from the start of the study (80.1) to three months (84.7). These improvements lasted up to six months (85.7).

Additional analyses also revealed that lower CF-PedsQL-GI scores at the start were associated with greater improvements at six months.

The use of the mobile app was generally well tolerated by children participating in the study. A total of 20 adverse events occurred over the course of the study that led to trial discontinuation. These included GI symptoms possibly linked to excessively high PERT doses (hard stools, painful bowel movements), others associated with excessively low therapy doses (loose stools), and respiratory exacerbations (flare-ups) that required hospitalization.

“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,” the researchers wrote.

“Further steps will include further confirmation of the positive effect of using the APP on QOL, and hopefully obtaining a CE Mark certification for the MyCyFAPP as well as its implementation in regular clinical practice,” they added.

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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