RESUMEN
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.
Asunto(s)
Fibrosis Quística , Terapia de Reemplazo Enzimático/métodos , Insuficiencia Pancreática Exocrina , Enfermedades Gastrointestinales , Aplicaciones Móviles , Calidad de Vida , Automanejo/métodos , Dolor Abdominal/etiología , Dolor Abdominal/terapia , Niño , Fibrosis Quística/fisiopatología , Fibrosis Quística/psicología , Fibrosis Quística/terapia , Insuficiencia Pancreática Exocrina/etiología , Insuficiencia Pancreática Exocrina/terapia , Femenino , Enfermedades Gastrointestinales/etiología , Enfermedades Gastrointestinales/fisiopatología , Enfermedades Gastrointestinales/psicología , Enfermedades Gastrointestinales/terapia , Humanos , Síndromes de Malabsorción/etiología , Síndromes de Malabsorción/terapia , Masculino , Desnutrición/etiología , Desnutrición/terapia , Encuestas y CuestionariosRESUMEN
Participation in proficiency testing schemes (PT-schemes) is an important way to prove and to improve the skills of analytical chemical students. It gives valuable information on the professional capabilities of the students and is thus an important step in the training of laboratory engineers and technicians. In the programme described three types of samples are offered to the students: chemical, environmental and clinical samples. A guiding protocol presents instructions to the participating student groups. Standard operating procedures are available, but the students are also free to select their own method. The results are methodologically and statistically judged by professional PT-organisers and are presented in the customary way: the dataset, the mean (consensus) value of all participants and the Z-scores. The final presentation of the results takes place in an annual symposium where awards are presented to the best performing student groups, and a lecture programme is presented with subjects in the area of quality assurance and quality control. A selection of results from the KILO/PT/1998-1999 and the KILO/PT/1999-2000 programme is presented.