M Boon1, J Calvo-Lerma2, I Claes3, T Havermans3, I Asseiceira4, A Bulfamante5, M Garriga6, E Masip7, B A M van Schijndel8, V Fornes2, C Barreto4, C Colombo5, P Crespo2, S Vicente6, H Janssens9, J Hulst8, P Witters3, R Nobili5, L Pereira4, M Ruperto6, E Van der Wiel9, J G Mainz10, K De Boeck3, C Ribes-Koninckx2. 1. Department of Pediatrics, Center for Cystic Fibrosis, University Hospital Leuven, Leuven, Belgium. Electronic address: mieke.boon@uzleuven.be. 2. Instituto de Investigación Sanitaria La Fe, Valencia, Spain. 3. Department of Pediatrics, Center for Cystic Fibrosis, University Hospital Leuven, Leuven, Belgium. 4. Associação para a Investigação e Desenvolvimento da Faculdade de Medicina, Lisbon, Portugal. 5. Università degli Studi di Milano, Fondazione IRCCS Ca" Granda, Ospedale Maggiore Policlinico, Milan, Italy. 6. Unidad de Fibrosis Quística, Hospital Universitario Ramón y Cajal, Madrid, Spain. 7. Gastroenterology and Pediatric cystic Fibrosis Unit, La Fe Hospital, Valencia, Spain. 8. Department of Pediatrics, div of Gastro-Enterology, Erasmus MC- Sophia Children's Hospital, University Hospital Rotterdam, the Netherlands. 9. Department of Pediatrics, div Respiratory Medicine and Allergology, Erasmus MC-Sophia Children's Hospital, University Hospital Rotterdam, the Netherlands. 10. Cystic Fibrosis Center for Children and Adults, Jena University Hospital, Germany; Cystic Fibrosis Center Brandenburg Medical School (MHB), University, Brandenburg an der Havel, Germany.
Abstract
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.
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.
Authors: Jochen G Mainz; Carlos Zagoya; Louise Polte; Lutz Naehrlich; Lenny Sasse; Olaf Eickmeier; Christina Smaczny; Anton Barucha; Lilith Bechinger; Franziska Duckstein; Ludwik Kurzidim; Patience Eschenhagen; Laura Caley; Daniel Peckham; Carsten Schwarz Journal: Front Pharmacol Date: 2022-06-03 Impact factor: 5.988
Authors: A Jay Freeman; Meghana Sathe; Enid Aliaj; Drucy Borowitz; Barbra Fogarty; Christopher H Goss; Steven Freedman; Sonya L Heltshe; Umer Khan; Dara Riva; Christina Roman; Melita Romasco; Sarah Jane Schwarzenberg; Carmen A Ufret-Vincenty; Baha Moshiree Journal: J Cyst Fibros Date: 2021-01-13 Impact factor: 5.527
Authors: Carolina Wannheden; Matilda Åberg-Wennerholm; Marie Dahlberg; Åsa Revenäs; Sara Tolf; Elena Eftimovska; Mats Brommels Journal: J Med Internet Res Date: 2022-08-01 Impact factor: 7.076
Authors: Joaquim Calvo-Lerma; Mieke Boon; Jessie Hulst; Carla Colombo; Inês Asseiceira; María Garriga; Etna Masip; Ine Claes; Anna Bulfamante; Hettie M Janssens; Maria Roca; Saioa Vicente; Victoria Fornés; Laura Zazzeron; Bo van Schijndel; Sandra Woodcock; Luisa Pereira; Kris de Boeck; Carmen Ribes-Koninckx Journal: Nutrients Date: 2021-05-26 Impact factor: 5.717
Authors: Meghana Sathe; Baha Moshiree; Phuong T Vu; Umer Khan; Sonya L Heltshe; Melita Romasco; Steven D Freedman; Sarah Jane Schwarzenberg; Christopher H Goss; A Jay Freeman Journal: J Cyst Fibros Date: 2021-07-22 Impact factor: 5.527
Authors: Rebecca J Calthorpe; Sherie J Smith; Nicola J Rowbotham; Paul A Leighton; Gwyneth Davies; Tracey Daniels; Katie Gathercole; Lorna Allen; Zoe C Elliott; Alan R Smyth Journal: BMJ Open Respir Res Date: 2020-08