Lessons learned from drug trials in neurofibromatosis: A systematic review

Authors :: Annette Bakker
Angela M. Kaindl
D. Gareth Evans
Marco Nievo
Charlotte Carton
Cornelia Potratz
Rianne Oostenbrink
Edwin van de Ketterij
Britt A.E. Dhaenens
Rosalie E. Ferner
Guenter Heimann
Geesje Hissink
Eric Legius
Pediatrics
Erasmus MC other
Source :: European Journal of Medical Genetics, 64(9):104281. Elsevier Masson, Dhaenens, B A E, Ferner, R E, Evans, D G, Heimann, G, Potratz, C, van de Ketterij, E, Kaindl, A M, Hissink, G, Carton, C, Bakker, A, Nievo, M, Legius, E & Oostenbrink, R 2021, ' Lessons learned from drug trials in neurofibromatosis: A systematic review ', European journal of medical genetics, vol. 64, no. 9, 104281 . https://doi.org/10.1016/j.ejmg.2021.104281
Publication Year :: 2021
Publisher :: Elsevier BV, 2021.
Abstract: Neurofibromatosis (NF) is the umbrella term for neurofibromatosis type 1 (NF1), neurofibromatosis type 2 (NF2) and schwannomatosis (SWN). EU-PEARL aims to create a framework for platform trials in NF. The aim of this systematic review is to create an overview of recent clinical drug trials in NF, to identify learning points to guide development of the framework. We searched Embase, Medline and Cochrane register of trials on October 1, 2020 for publications of clinical drug trials in NF patients. We excluded publications published before 2010, systematic reviews, secondary analyses and studies with

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