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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2019
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6550371/
https://www.ncbi.nlm.nih.gov/pubmed/31166977
http://dx.doi.org/10.1371/journal.pone.0215739

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