Elicitation of Expert Prior Opinion: Application to the MYPAN Trial in Childhood Polyarteritis Nodosa

Lisa V. Hampson; John Whitehead; Despina Eleftheriou; Catrin Tudur-Smith; Rachel Jones; David Jayne; Helen Hickey; Michael W. Beresford; Claudia Bracaglia; Afonso Caldas; Rolando Cimaz; Joke Dehoorne; Pavla Dolezalova; Mark Friswell; Marija Jelusic; Stephen D. Marks; Neil Martin; Anne-Marie McMahon; Joachim Peitz; Annet  van Royen; Oguz Soylemezoglu; Paul A. Brogan

doi:10.1371/journal.pone.0120981

Elicitation of Expert Prior Opinion: Application to the MYPAN Trial in Childhood Polyarteritis Nodosa

Lisa V. Hampson^*
, John Whitehead
, Despina Eleftheriou
, Catrin Tudur-Smith
, Rachel Jones
, David Jayne
, Helen Hickey
, Michael W. Beresford
, Claudia Bracaglia
, Afonso Caldas
, Rolando Cimaz
, Joke Dehoorne
, Pavla Dolezalova
, Mark Friswell
, Marija Jelusic
, Stephen D. Marks
, Neil Martin
, Anne-Marie McMahon
, Joachim Peitz
, Annet van Royen

Oguz Soylemezoglu, Paul A. Brogan

^*Corresponding author for this work

Child Health

Research output: Contribution to journal › Article › Academic › peer-review

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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.

Original language	English
Article number	0120981
Number of pages	14
Journal	PLoS ONE [E]
Volume	10
Issue number	3
DOIs	https://doi.org/10.1371/journal.pone.0120981
Publication status	Published - 30 Mar 2015

Keywords

CHURG-STRAUSS-SYNDROME
POOR-PROGNOSIS FACTORS
RARE DISEASES
MICROSCOPIC POLYANGIITIS
PULSE CYCLOPHOSPHAMIDE
RANDOMIZED-TRIAL
CLINICAL-TRIALS
VASCULITIS
BELIEFS

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Hampson, L. V., Whitehead, J., Eleftheriou, D., Tudur-Smith, C., Jones, R., Jayne, D., Hickey, H., 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., ... Brogan, P. A. (2015). Elicitation of Expert Prior Opinion: Application to the MYPAN Trial in Childhood Polyarteritis Nodosa. PLoS ONE [E], 10(3), Article 0120981. https://doi.org/10.1371/journal.pone.0120981

@article{eba6624700b842918eb4c8c6d6c77e83,

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.",

keywords = "CHURG-STRAUSS-SYNDROME, POOR-PROGNOSIS FACTORS, RARE DISEASES, MICROSCOPIC POLYANGIITIS, PULSE CYCLOPHOSPHAMIDE, RANDOMIZED-TRIAL, CLINICAL-TRIALS, VASCULITIS, BELIEFS",

author = "Hampson, \{Lisa V.\} 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 Afonso 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\}, Annet and Oguz Soylemezoglu and Brogan, \{Paul A.\}",

year = "2015",

month = mar,

day = "30",

doi = "10.1371/journal.pone.0120981",

language = "English",

volume = "10",

journal = "PLoS ONE [E]",

issn = "1932-6203",

publisher = "Public Library of Science",

number = "3",

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Hampson, LV, Whitehead, J, Eleftheriou, D, Tudur-Smith, C, Jones, R, Jayne, D, Hickey, H, 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, A, Soylemezoglu, O & Brogan, PA 2015, 'Elicitation of Expert Prior Opinion: Application to the MYPAN Trial in Childhood Polyarteritis Nodosa', PLoS ONE [E], vol. 10, no. 3, 0120981. https://doi.org/10.1371/journal.pone.0120981

TY - JOUR

T1 - Elicitation of Expert Prior Opinion

T2 - Application to the MYPAN Trial in Childhood Polyarteritis Nodosa

AU - Hampson, Lisa V.

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, Afonso

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, Annet

AU - Soylemezoglu, Oguz

AU - Brogan, Paul A.

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.

KW - CHURG-STRAUSS-SYNDROME

KW - POOR-PROGNOSIS FACTORS

KW - RARE DISEASES

KW - MICROSCOPIC POLYANGIITIS

KW - PULSE CYCLOPHOSPHAMIDE

KW - RANDOMIZED-TRIAL

KW - CLINICAL-TRIALS

KW - VASCULITIS

KW - BELIEFS

U2 - 10.1371/journal.pone.0120981

DO - 10.1371/journal.pone.0120981

M3 - Article

SN - 1932-6203

VL - 10

JO - PLoS ONE [E]

JF - PLoS ONE [E]

IS - 3

M1 - 0120981

ER -

Elicitation of Expert Prior Opinion: Application to the MYPAN Trial in Childhood Polyarteritis Nodosa

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Keywords

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