Cystic Fibrosis
Conditions
Keywords
Cystic fibrosis, Abdomen, Pancreas, Liver, Gut, Symptom Score, Patient reported outcome measure (PROM), diabetes, CFTR-modulator, Tricafta, Kaftrio
Brief summary
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.
Detailed 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
Interventions
DIAGNOSTIC_TESTQuestionnaire Patient Reported Outcome Measure (PROM)
non-interventional
Sponsors
University of Jena
Medizinische Hochschule Brandenburg Theodor Fontane
Study design
Observational model
CASE_CONTROL
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
Yes
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.)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Development and evaluation of the CFAbd-Score (initially named JenAbdomen CF-Score), a CF-specific PROM following FDA recommendations | 7 years | 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 | 7 | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings | 7 years | 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 | 3 years | abdominal ultrasound |
| Effects of a new therapy with CFTR-modulator on abdominal symtoms, quantified with the CFAbd-Score | 7 years | Assessing the CFAbd-Score´s sensitivity to identify and quantify changes due to effective therapeutic interventions, following FDA-guidelines for validation of a PROM |
Countries
Germany
Contacts
Primary ContactJochen G Mainz, Prof. MD
Backup ContactFranziska Duckstein, MA
Outcome results
None listed