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| ID | Type | Description | Link |
|---|---|---|---|
| CFAbd-Score.kid | Other Identifier | Brandenburg Med.School (Univ.), Brandenb.a.d.Havel/Germany | |
| CFAbd-day2day | Other Identifier | Brandenburg Med.School (Univ.), Brandenb.a.d.Havel/Germany |
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| Name | Class |
|---|---|
| University of Jena | OTHER |
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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
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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients with CF | >2000 in Germany, France, Italy, Spain, Denmark, Belgium, Portugal, GB, Ireland, USA, Australia, Canada, Brazil, Argentina... |
| |
| Age-matched healthy controls | >100 in Germany >100 in each of the other participating countries |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Questionnaire Patient Reported Outcome Measure (PROM) | Diagnostic Test | non-interventional |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 7 years |
| Development and evaluation of a diary for assessment and quantification of abdominal symptoms, a CF-specific PROM following FDA recommendations | 7 |
| Measure | Description | Time Frame |
|---|---|---|
| Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings | relating CFAbd-Scores to history, clinical- and laboratory findings | 7 years |
| Assessing the Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients |
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Inclusion Criteria:
CF cohort:
Diagnosis of CF determined by
Healthy controls:
Exclusion Criteria:
CF cohort:
Healthy controls:
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Patients with CF of all ages
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jochen G Mainz, Prof. MD | Contact | +493381411803 | jochen.mainz@mhb-fontane.de | |
| Franziska Duckstein, MA | Contact | +493381411841 | f.duckstein@klinikum-brandenburg.de |
| Name | Affiliation | Role |
|---|---|---|
| Jochen G Mainz, Prof. MD | Medizinische Hochschule Brandenburg (MHB), University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Klinikum Westbrandenburg | Recruiting | Brandenburg an der Havel | 14770 | Germany |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28472055 | Result | Tabori H, Arnold C, Jaudszus A, Mentzel HJ, Renz DM, Reinsch S, Lorenz M, Michl R, Gerber A, Lehmann T, Mainz JG. Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings. PLoS One. 2017 May 4;12(5):e0174463. doi: 10.1371/journal.pone.0174463. eCollection 2017. | |
| 29234058 | Result |
| Label | URL |
|---|---|
| Validity and Reliability of a Novel Multimodal Questionnaire for the Assessment of Abdominal Symptoms in People with Cystic Fibrosis (CFAbd-Score). | View source |
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Stool
abdominal ultrasound |
| 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 | 7 years |
| Medizinische Hochschule Brandenburg (MHB), University | Recruiting | Brandenburg an der Havel | 14770 | Germany |
|
| Tabori H, Jaudszus A, Arnold C, Mentzel HJ, Lorenz M, Michl RK, Lehmann T, Renz DM, Mainz JG. Relation of Ultrasound Findings and Abdominal Symptoms obtained with the CFAbd-Score in Cystic Fibrosis Patients. Sci Rep. 2017 Dec 12;7(1):17465. doi: 10.1038/s41598-017-17302-4. |
| 30887269 | Result | Jaudszus A, Zeman E, Jans T, Pfeifer E, Tabori H, Arnold C, Michl RK, Lorenz M, Beiersdorf N, Mainz JG. Validity and Reliability of a Novel Multimodal Questionnaire for the Assessment of Abdominal Symptoms in People with Cystic Fibrosis (CFAbd-Score). Patient. 2019 Aug;12(4):419-428. doi: 10.1007/s40271-019-00361-2. |
| 34789668 | Result | Jaudszus A, Pfeifer E, Lorenz M, Beiersdorf N, Hipler UC, Zagoya C, Mainz JG. Abdominal Symptoms Assessed With the CFAbd-Score are Associated With Intestinal Inflammation in Patients With Cystic Fibrosis. J Pediatr Gastroenterol Nutr. 2022 Mar 1;74(3):355-360. doi: 10.1097/MPG.0000000000003357. |
| 35721187 | Result | Mainz JG, Zagoya C, Polte L, Naehrlich L, Sasse L, Eickmeier O, Smaczny C, Barucha A, Bechinger L, Duckstein F, Kurzidim L, Eschenhagen P, Caley L, Peckham D, Schwarz C. Elexacaftor-Tezacaftor-Ivacaftor Treatment Reduces Abdominal Symptoms in Cystic Fibrosis-Early results Obtained With the CF-Specific CFAbd-Score. Front Pharmacol. 2022 Jun 3;13:877118. doi: 10.3389/fphar.2022.877118. eCollection 2022. |
| 36710099 | Result | Caley LR, Zagoya C, Duckstein F, White H, Shimmin D, Jones AM, Barrett J, Whitehouse JL, Floto RA, Mainz JG, Peckham DG. Diabetes is associated with increased burden of gastrointestinal symptoms in adults with cystic fibrosis. J Cyst Fibros. 2023 Mar;22(2):275-281. doi: 10.1016/j.jcf.2023.01.010. Epub 2023 Jan 27. |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| D003920 | Diabetes Mellitus |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| D044882 | Glucose Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D004700 | Endocrine System Diseases |
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