Core Outcome Set for Liver Transplant in Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE): the COLT-MNGIE Project- Protocol
Authors/Creators
- 1. IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Interaziendale Metropolitana (NeuroMet), - Neurologia AOU S.Orsola-Malpighi, Bologna, Italy
- 2. IRCCS Istituto delle Scienze Neurologiche di Bologna, Epidemiology and Biostatistics Unit, Bologna, Italy
- 3. Department of General Surgery and Transplantation, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy
- 4. Department of Neurology, Columbia University Irving Medical Center, New York, New York, USA
- 5. Digestive System Research Unit, University Hospital Vall d'Hebrón
- 6. Vall d'Hebron Research Institute, Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Autonomous University of Barcelona, Barcelona, Spain.
- 7. Mitocon ONLUS, Rome, Italy.
- 8. School of Medicine - Department of Medical and Surgical Sciences (DIMEC) University of Bologna, Bologna, Italy
- 9. Internal Medicine Unit for the Treatment of Severe Organ Failure, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy
- 10. Mitocon ONLUS, Rome, Italy
- 11. Departement de Neuropediatrie, CHU Gui de Chauliac, Institut des Neurosciences de Montpellier, Montpellier, France
Description
The purpose of this protocol is to report the methodology for the development of a Core Outcome Set (COS) for the mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). A COS represents the minimum that should be measured in a clinical trial for a particular condition. The aim of the COLT-MNGIE project is to develop a COS to be used in research on the efficacy, safety and value of liver transplantation for MNGIE and other permanent enzyme-replacement treatments. COS will be developed according to the Core Outcome Measures in Effectiveness Trials (COMET) Initiative standards and recommendations. The COLT-MNGIE project is promoted by MITOCON (https://www.mitocon.it/), the Italian advocacy group on mitochondrial diseases, and it will involve healthcare professionals (neurologists, paediatric neurologists, gastroenterologists, transplant surgeons, post-surgical ICU specialists, nutritionists), biochemists and other researchers/professionals, persons with MNGIE, their care givers and methodologists.
Files
MNGIE COS protocol v20230505.pdf
Files
(793.2 kB)
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