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    Rights statement: © 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

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

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

In: PLoS ONE, Vol. 10, No. 3, e0120981, 30.03.2015.

Research output: Contribution to journalJournal articlepeer-review

Harvard

Beresford, MW, Bracaglia, C, Caldas, A, Cimaz, R, Dehoorne, J, Dolezalova, P, Friswell, M, Jelusic, M, Marks, SD, Martin, N, McMahon, A-M, Peitz, J, van Royen-Kirkhof, A, Soylemezoglu, O, Brogan, P & MYPAN Vasculitis Expert Group 2015, 'Elicitation of expert prior opinion: application to the MYPAN trial in childhood polyarteritis nodosa', PLoS ONE, vol. 10, no. 3, e0120981. https://doi.org/10.1371/journal.pone.0120981

APA

Beresford, M. W., Bracaglia, C., Caldas, A., Cimaz, R., Dehoorne, J., Dolezalova, P., Friswell, M., Jelusic, M., Marks, S. D., Martin, N., McMahon, A-M., Peitz, J., van Royen-Kirkhof, A., Soylemezoglu, O., Brogan, P., & MYPAN Vasculitis Expert Group (2015). Elicitation of expert prior opinion: application to the MYPAN trial in childhood polyarteritis nodosa. PLoS ONE, 10(3), [e0120981]. https://doi.org/10.1371/journal.pone.0120981

Vancouver

Beresford MW, Bracaglia C, Caldas A, Cimaz R, Dehoorne J, Dolezalova P et al. Elicitation of expert prior opinion: application to the MYPAN trial in childhood polyarteritis nodosa. PLoS ONE. 2015 Mar 30;10(3). e0120981. https://doi.org/10.1371/journal.pone.0120981

Author

Beresford, Michael W. ; Bracaglia, Claudia ; Caldas, Alfonso ; Cimaz, Rolando ; Dehoorne, Joke ; Dolezalova, Pavla ; Friswell, Mark ; Jelusic, Marija ; Marks, Stephen D. ; Martin, Neil ; McMahon, Anne-Marie ; Peitz, Joachim ; van Royen-Kirkhof, Annet ; Soylemezoglu, Oguz ; Brogan, Paul ; MYPAN Vasculitis Expert Group. / Elicitation of expert prior opinion : application to the MYPAN trial in childhood polyarteritis nodosa. In: PLoS ONE. 2015 ; Vol. 10, No. 3.

Bibtex

@article{138d957202a143e6a81d379a1b4297fe,
title = "Elicitation of expert prior opinion: application to the MYPAN trial in childhood polyarteritis nodosa",
abstract = "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.",
author = "Lisa Hampson and John Whitehead and Despina Eleftheriou and Catrin Tudur-Smith and Rachel Jones and David Jayne and Helen Hickey and Beresford, {Michael W.} and Claudia Bracaglia and Alfonso Caldas and Rolando Cimaz and Joke Dehoorne and Pavla Dolezalova and Mark Friswell and Marija Jelusic and Marks, {Stephen D.} and Neil Martin and Anne-Marie McMahon and Joachim Peitz and {van Royen-Kirkhof}, Annet and Oguz Soylemezoglu and Paul Brogan and {MYPAN Vasculitis Expert Group}",
note = " {\textcopyright} 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",
year = "2015",
month = mar,
day = "30",
doi = "10.1371/journal.pone.0120981",
language = "English",
volume = "10",
journal = "PLoS ONE",
issn = "1932-6203",
publisher = "Public Library of Science",
number = "3",

}

RIS

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 -