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.
a sweat chloride of >60 mEq/L and/or
detection of 2 disease causing CFTR mutations with evidence of organ involvement.
Age-matched
Lacking ability to cooperate of patients /their families, respectively
Other diseases causing abdominal symptoms (Inflammatory bowel disease (IBD), alpha 1 antitrypsin deficiency, celiac disease, food allergy, gastroenteritis, etc.)
Patients with CF of all ages
Case-control
Prospective
Questionnaire Patient Reported Outcome Measure (PROM)
Diagnostic testnon-interventional
Development 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
Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations
Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings
relating CFAbd-Scores to history, clinical- and laboratory findings
Assessing the Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients
abdominal ultrasound
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
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Medizinische Hochschule Brandenburg Theodor Fontane
Lead sponsor
University of Jena
Collaborator
