Overview
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.
Description
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
Eligibility
Inclusion Criteria:
CF cohort:
Diagnosis of CF determined by
- a sweat chloride of >60 mEq/L and/or
- detection of 2 disease causing CFTR mutations with evidence of organ involvement.
Healthy controls:
- Age-matched
Exclusion Criteria:
CF cohort:
- Lacking ability to cooperate of patients /their families, respectively
Healthy controls:
- Other diseases causing abdominal symptoms (Inflammatory bowel disease (IBD), alpha 1 antitrypsin deficiency, celiac disease, food allergy, gastroenteritis, etc.)