Universität zu Köln

Hampson, Lisa V., Whitehead, John, Eleftheriou, Despina, Tudur-Smith, Catrin, Jones, Rachel ORCID: 0000-0003-4790-283X, Jayne, David, Hickey, Helen, Beresford, Michael W., Bracaglia, Claudia ORCID: 0000-0002-9834-9619, Caldas, Afonso, Cimaz, Rolando, Dehoorne, Joke, Dolezalova, Pavla ORCID: 0000-0001-8375-661X, Friswell, Mark, Jelusic, Marija, Marks, Stephen D., Martin, Neil, McMahon, Anne-Marie, Peitz, Joachim, van Royen-Kerkhof, Annet, Soylemezoglu, Oguz and Brogan, Paul A. (2015). Elicitation of Expert Prior Opinion: Application to the MYPAN Trial in Childhood Polyarteritis Nodosa. PLoS One, 10 (3). SAN FRANCISCO: PUBLIC LIBRARY SCIENCE. 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
Creators:	Creators Email ORCID ORCID Put Code Hampson, Lisa V. UNSPECIFIED UNSPECIFIED UNSPECIFIED Whitehead, John UNSPECIFIED UNSPECIFIED UNSPECIFIED Eleftheriou, Despina UNSPECIFIED UNSPECIFIED UNSPECIFIED Tudur-Smith, Catrin UNSPECIFIED UNSPECIFIED UNSPECIFIED Jones, Rachel UNSPECIFIED orcid.org/0000-0003-4790-283X UNSPECIFIED Jayne, David UNSPECIFIED UNSPECIFIED UNSPECIFIED Hickey, Helen UNSPECIFIED UNSPECIFIED UNSPECIFIED Beresford, Michael W. UNSPECIFIED UNSPECIFIED UNSPECIFIED Bracaglia, Claudia UNSPECIFIED orcid.org/0000-0002-9834-9619 UNSPECIFIED Caldas, Afonso UNSPECIFIED UNSPECIFIED UNSPECIFIED Cimaz, Rolando UNSPECIFIED UNSPECIFIED UNSPECIFIED Dehoorne, Joke UNSPECIFIED UNSPECIFIED UNSPECIFIED Dolezalova, Pavla UNSPECIFIED orcid.org/0000-0001-8375-661X UNSPECIFIED Friswell, Mark UNSPECIFIED UNSPECIFIED UNSPECIFIED Jelusic, Marija UNSPECIFIED UNSPECIFIED UNSPECIFIED Marks, Stephen D. UNSPECIFIED UNSPECIFIED UNSPECIFIED Martin, Neil UNSPECIFIED UNSPECIFIED UNSPECIFIED McMahon, Anne-Marie UNSPECIFIED UNSPECIFIED UNSPECIFIED Peitz, Joachim UNSPECIFIED UNSPECIFIED UNSPECIFIED van Royen-Kerkhof, Annet UNSPECIFIED UNSPECIFIED UNSPECIFIED Soylemezoglu, Oguz UNSPECIFIED UNSPECIFIED UNSPECIFIED Brogan, Paul A. UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-409440
DOI:	10.1371/journal.pone.0120981
Journal or Publication Title:	PLoS One
Volume:	10
Number:	3
Date:	2015
Publisher:	PUBLIC LIBRARY SCIENCE
Place of Publication:	SAN FRANCISCO
ISSN:	1932-6203
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language CHURG-STRAUSS-SYNDROME; POOR-PROGNOSIS FACTORS; RARE DISEASES; MICROSCOPIC POLYANGIITIS; PULSE CYCLOPHOSPHAMIDE; RANDOMIZED-TRIAL; CLINICAL-TRIALS; VASCULITIS; BELIEFS Multiple languages Multidisciplinary Sciences Multiple languages
URI:	http://kups.ub.uni-koeln.de/id/eprint/40944