Development, validation and evaluation of a new multimodal questionnaire to assess and quantify the abdominal involvement in patients with the inherited life shortening multi-organ disease Cystic fibrosis. In the stepwhise development of the patient reported outcome measures (PROM) repeadedly, CF-patients, their families as well as professionals working in different fields of CF-care will be included.
Development of a multimodal questionnaire considering the FDA guidelines for development of a PROM, the CFAbd-Score * Evaluation of reliability of the questionnaire by examining internal consistency and construct validity * Evaluation of reproducibility of the questionnaire by re-testing of patients * Assessment of cross-generational applicability of the questionnaire by sub-scoring of age groups * Evaluation of the responsiveness of the score by comparison with age-matched healthy controls * Assessment of putative relationships of abdominal symptoms with faecal inflammatory markers * Assessment of applicability of the questionnaire in other CF centers * Transfer the CFAbd-Score to other countries and languages Development and validation of a CF-specific tool for daily assessment of abdominal symptoms (patient diary), the CFAbd-day2day Development and validation of a CF-specific tool for assessment of abdominal symptoms in children \< 12 years of age, the CFAbd-kid
Study Type
OBSERVATIONAL
Enrollment
2,000
non-interventional
Klinikum Westbrandenburg
Brandenburg an der Havel, Germany
RECRUITINGMedizinische Hochschule Brandenburg (MHB), University
Brandenburg an der Havel, Germany
RECRUITINGDevelopment and evaluation of the CFAbd-Score (initially named JenAbdomen CF-Score), a CF-specific PROM following FDA recommendations
Evaluation and validation of the CFAbd-Score based on a CF patient-reported outcome measure (PROM) that includes all relevant gastrointestinal symptoms and their impact on subjective quality of life
Time frame: 7 years
Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations
Time frame: 7
Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings
relating CFAbd-Scores to history, clinical- and laboratory findings
Time frame: 7 years
Assessing the Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients
abdominal ultrasound
Time frame: 3 years
Effects of a new therapy with CFTR-modulator on abdominal symtoms, quantified with the CFAbd-Score
Assessing the CFAbd-Score´s sensitivity to identify and quantify changes due to effective therapeutic interventions, following FDA-guidelines for validation of a PROM
Time frame: 7 years
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