Elicitation of expert prior opinion:application to the MYPAN trial in childhood polyarteritis nodosa

Beresford, Michael W. and Bracaglia, Claudia and Caldas, Alfonso and Cimaz, Rolando and Dehoorne, Joke and Dolezalova, Pavla and Friswell, Mark and Jelusic, Marija and Marks, Stephen D. and Martin, Neil and McMahon, Anne-Marie and Peitz, Joachim and van Royen-Kirkhof, Annet and Soylemezoglu, Oguz and Brogan, Paul and , MYPAN Vasculitis Expert Group (2015) Elicitation of expert prior opinion:application to the MYPAN trial in childhood polyarteritis nodosa. PLoS ONE, 10 (3). ISSN 1932-6203

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Abstract

Objectives Definitive sample sizes for clinical trials in rare diseases are usually infeasible. Bayesian methodology can be used to maximise what is learnt from clinical trials in these circumstances. We elicited expert prior opinion for a future Bayesian randomised controlled trial for a rare inflammatory paediatric disease, polyarteritis nodosa (MYPAN, Mycophenolate mofetil for polyarteritis nodosa). Methods A Bayesian prior elicitation meeting was convened. Opinion was sought on the probability that a patient in the MYPAN trial treated with cyclophosphamide would achieve disease remission within 6-months, and on the relative efficacies of mycophenolate mofetil and cyclophosphamide. Expert opinion was combined with previously unseen data from a recently completed randomised controlled trial in ANCA associated vasculitis. Results A pan-European group of fifteen experts participated in the elicitation meeting. Consensus expert prior opinion was that the most likely rates of disease remission within 6 months on cyclophosphamide or mycophenolate mofetil were 74% and 71%, respectively. This prior opinion will now be taken forward and will be modified to formulate a Bayesian posterior opinion once the MYPAN trial data from 40 patients randomised 1:1 to either CYC or MMF become available. Conclusions We suggest that the methodological template we propose could be applied to trial design for other rare diseases.

Item Type:
Journal Article
Journal or Publication Title:
PLoS ONE
Additional Information:
© 2015 Hampson et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited
Uncontrolled Keywords:
/dk/atira/pure/subjectarea/asjc/2700
Subjects:
ID Code:
73853
Deposited By:
Deposited On:
18 Jun 2015 05:46
Refereed?:
Yes
Published?:
Published
Last Modified:
21 Oct 2020 03:18