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Research output: Contribution to Journal/Magazine › Journal article › peer-review
Research output: Contribution to Journal/Magazine › Journal article › peer-review
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TY - JOUR
T1 - Elicitation of expert prior opinion
T2 - application to the MYPAN trial in childhood polyarteritis nodosa
AU - Hampson, Lisa
AU - Whitehead, John
AU - Eleftheriou, Despina
AU - Tudur-Smith, Catrin
AU - Jones, Rachel
AU - Jayne, David
AU - Hickey, Helen
AU - Beresford, Michael W.
AU - Bracaglia, Claudia
AU - Caldas, Alfonso
AU - Cimaz, Rolando
AU - Dehoorne, Joke
AU - Dolezalova, Pavla
AU - Friswell, Mark
AU - Jelusic, Marija
AU - Marks, Stephen D.
AU - Martin, Neil
AU - McMahon, Anne-Marie
AU - Peitz, Joachim
AU - van Royen-Kirkhof, Annet
AU - Soylemezoglu, Oguz
AU - Brogan, Paul
AU - MYPAN Vasculitis Expert Group
N1 - © 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
PY - 2015/3/30
Y1 - 2015/3/30
N2 - ObjectivesDefinitive 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).MethodsA 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.ResultsA 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.ConclusionsWe suggest that the methodological template we propose could be applied to trial design for other rare diseases.
AB - ObjectivesDefinitive 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).MethodsA 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.ResultsA 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.ConclusionsWe suggest that the methodological template we propose could be applied to trial design for other rare diseases.
U2 - 10.1371/journal.pone.0120981
DO - 10.1371/journal.pone.0120981
M3 - Journal article
VL - 10
JO - PLoS ONE
JF - PLoS ONE
SN - 1932-6203
IS - 3
M1 - e0120981
ER -