Defining consensus opinion to develop randomised controlled trials in rare diseases using Bayesian design: An example of a proposed trial of adalimumab versus pamidronate for children with CNO/CRMO

INTRODUCTION: Chronic nonbacterial osteomyelitis (CNO) is a rare autoinflammatory bone disorder primarily affecting children and adolescents. It can lead to chronic pain, bony deformities and fractures. The pathophysiology of CNO is incompletely understood. Scientific evidence suggests dysregulated...

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Detalhes bibliográficos
Publicado no:PLoS One
Main Authors: Ramanan, A. V., Hampson, L. V., Lythgoe, H, Jones, A. P., Hardwick, B, Hind, H, Jacobs, B, Vasileiou, D, Wadsworth, I, Ambrose, N, Davidson, J, Ferguson, P. J., Herlin, T, Kavirayani, A, Killeen, O. G., Compeyrot-Lacassagne, S, Laxer, R. M., Roderick, M, Swart, J. F., Hedrich, C. M., Beresford, M. W.
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2019
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6550371/
https://ncbi.nlm.nih.gov/pubmed/31166977
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0215739
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