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

被引:26
作者
Hampson, Lisa V. [1 ]
Whitehead, John [1 ]
Eleftheriou, Despina [2 ]
Tudur-Smith, Catrin [3 ]
Jones, Rachel [4 ]
Jayne, David [5 ]
Hickey, Helen [6 ]
Beresford, Michael W. [7 ]
Bracaglia, Claudia [8 ]
Caldas, Afonso [9 ]
Cimaz, Rolando [10 ]
Dehoorne, Joke [11 ]
Dolezalova, Pavla [12 ,13 ]
Friswell, Mark [14 ]
Jelusic, Marija [15 ]
Marks, Stephen D. [16 ]
Martin, Neil [17 ]
McMahon, Anne-Marie [18 ]
Peitz, Joachim [19 ]
van Royen-Kerkhof, Annet [20 ]
Soylemezoglu, Oguz [21 ]
Brogan, Paul A. [2 ]
机构
[1] Univ Lancaster, Dept Math & Stat, Lancaster, England
[2] UCL Inst Child Hlth, Dept Rheumatol, London, England
[3] Univ Liverpool, MRC North West Hub Trials Methodol Res, Dept Biostat, Liverpool L69 3BX, Merseyside, England
[4] Addenbrookes Hosp, Dept Renal Med, Cambridge, England
[5] Univ Cambridge, Dept Med, Cambridge CB2 2QQ, England
[6] Univ Liverpool, Alder Hey Childrens NHS Fdn Trust, Medicines Children Res Network Clin Trials Unit, Liverpool L69 3BX, Merseyside, England
[7] Univ Liverpool, Inst Translat Med, Dept Womens & Childrens Hlth, Liverpool L69 3BX, Merseyside, England
[8] IRCCS Bambino Gesu Children Hosp, Dept Paediat Med, Div Rheumatol, I-00165 Rome, Italy
[9] Integrated Hosp S Joao, Oporto Med Sch, Oporto, Portugal
[10] AOU Meyer, I-50139 Florence, Italy
[11] Univ Hosp Ghent, Dept Pediat Rheumatol & Nephrol, Ghent, Belgium
[12] Charles Univ Prague, Fac Med 1, Dept Paediat & Adolescent Med, Prague, Czech Republic
[13] Gen Univ Hosp, Prague, Czech Republic
[14] Great North Childrens Hosp, Newcastle Upon Tyne, Tyne & Wear, England
[15] Univ Zagreb, Sch Med, Univ Hosp Ctr Zagreb, Dept Paediat Rheumatol, Zagreb 41001, Croatia
[16] Great Ormond St Hosp Children NHS Fdn Trust, Dept Paediat Nephrol, London WC1N 3JH, England
[17] Yorkhill Hosp, Dept Paediat Rheumatol, Glasgow, Lanark, Scotland
[18] Sheffield Childrens Hosp NHS Fdn Trust, Sheffield, S Yorkshire, England
[19] Univ Hosp Cologne, Hosp Children & Adolescents, Cologne, Germany
[20] Wilhelmina Childrens Hosp, Univ Med Ctr, Utrecht, Netherlands
[21] Gazi Univ Hosp, Dept Pediat Nephrol & Rheumatol, Ankara, Turkey
基金
英国医学研究理事会;
关键词
CHURG-STRAUSS-SYNDROME; POOR-PROGNOSIS FACTORS; MICROSCOPIC POLYANGIITIS; PULSE CYCLOPHOSPHAMIDE; CLINICAL-TRIALS; VASCULITIS; BELIEFS;
D O I
10.1371/journal.pone.0120981
中图分类号
O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];
学科分类号
07 ; 0710 ; 09 ;
摘要
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.
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