The University of Southampton
University of Southampton Institutional Repository

Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme)

Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme)
Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme)
Background
MACRO (Defining best Management for Adults with Chronic RhinOsinusitis) is an NIHR-funded programme of work designed to establish best practice for adults with chronic rhinosinusitis (CRS). The 7-year programme comprises three consecutive workstreams, designed to explore NHS care pathways through analysis of primary and secondary data sources, and to undertake a randomised controlled trial to evaluate a longer-term course of macrolide antibiotics and endoscopic sinus surgery for patients with CRS. A number of outstanding elements still required clarification at the funding stage. This paper reports an expert panel review process designed to agree and finalise the MACRO trial design, ensuring relevance to patients and clinicians whilst maximising trial recruitment and retention.
Methods
An expert panel consisting of the MACRO programme management group, independent advisors, and patient contributors, was convened to review current evidence and the mixed method data collected as part of the programme, and reach agreement on MACRO trial design. Specifically, agreement was sought for selection of macrolide antibiotic, use of oral steroids, inclusion of CRS phenotypes (with/without nasal polyps), and overall trial design.
Results
A 12-week course of clarithromycin was agreed as the main trial comparator, due to its increasing use as a first and second line treatment for patients with CRS, and the perceived need to establish its role in CRS management. Oral steroids will be used as a rescue medication during the trial, rather than routinely either pre or post trial randomisation, to limit any potential effects on surgical outcomes and better reflect current UK prescribing habits. Both CRS phenotypes will be included in a single trial to ensure the MACRO trial is both pragmatic and generalisable to primary care. A modified 3-arm trial design was agreed after intense discussions and further exploratory work. Inclusion criteria were amended to ensure that patients recruited would be considered eligible for the treatment offered in the trial, due to having already received appropriate medical therapy as deemed suitable by their ENT surgeon. A proposed 6-week run-in period prior to randomisation was removed due to the new criteria prior to randomisation.
Conclusion
The expert panel review process resulted in agreement on key elements and an optimal design for the MACRO trial, considered most likely to be successful in terms of both recruitment potential and ability to establish best management of patients with CRS.
1745-6215
Blackshaw, Helen
89f97306-9bba-42b3-a09f-4574d08bd711
Vennik, Jane
6ee78166-5a7a-433b-87fc-018771f20b19
Philpott, Carl
3b9f988c-a61e-4736-9b69-6697fb2cdc5b
Thomas, Mike
997c78e0-3849-4ce8-b1bc-86ebbdee3953
Eyles, Caroline
f8518cbb-669f-4cf6-bacb-4a174e385483
Carpenter, James
f711dd60-8725-4ce8-8d27-04ad03fc6850
Clarke, Caroline S.
87187b5a-a6c7-43eb-b754-f306bd1f698c
Morris, Stephen
0abf603b-3b19-43a9-be25-a0f1e76c574c
Schilder, Anne G.M.
4efe172e-cbc9-4d1b-b089-f293f5c626ef
Lund, Valerie
19b0121c-1518-4cd8-8c2b-be8557320b0f
Little, Paul
1bf2d1f7-200c-47a5-ab16-fe5a8756a777
Durham, Stephen
fad2d5de-ec3c-4f7b-aebc-2e6218b727e9
Denaxas, Spiros
65af3da2-30fd-4d09-bdb2-81abfd3f430c
Williamson, Elizabeth
0ffaceb0-37fa-4b19-be1f-420bb880a565
Beard, David
7bb0be51-1b39-4c53-b0c1-9d9e8c9df7e6
Cook, Jonathan
eb29ac3d-490b-4845-8756-dd600d97468c
Le Conte, Steffi
6aac8cc9-f712-406f-a1bf-ddadab83f4a4
Airey, K
18c2f6d1-1180-49ff-bba0-e6fac3ae150c
Boardman, J
e7693d8d-533e-4dbd-adee-a820fbc74528
Hopkins, Claire
8ed0a5e3-92a3-4988-9c2b-b0e51a8a044c
Blackshaw, Helen
89f97306-9bba-42b3-a09f-4574d08bd711
Vennik, Jane
6ee78166-5a7a-433b-87fc-018771f20b19
Philpott, Carl
3b9f988c-a61e-4736-9b69-6697fb2cdc5b
Thomas, Mike
997c78e0-3849-4ce8-b1bc-86ebbdee3953
Eyles, Caroline
f8518cbb-669f-4cf6-bacb-4a174e385483
Carpenter, James
f711dd60-8725-4ce8-8d27-04ad03fc6850
Clarke, Caroline S.
87187b5a-a6c7-43eb-b754-f306bd1f698c
Morris, Stephen
0abf603b-3b19-43a9-be25-a0f1e76c574c
Schilder, Anne G.M.
4efe172e-cbc9-4d1b-b089-f293f5c626ef
Lund, Valerie
19b0121c-1518-4cd8-8c2b-be8557320b0f
Little, Paul
1bf2d1f7-200c-47a5-ab16-fe5a8756a777
Durham, Stephen
fad2d5de-ec3c-4f7b-aebc-2e6218b727e9
Denaxas, Spiros
65af3da2-30fd-4d09-bdb2-81abfd3f430c
Williamson, Elizabeth
0ffaceb0-37fa-4b19-be1f-420bb880a565
Beard, David
7bb0be51-1b39-4c53-b0c1-9d9e8c9df7e6
Cook, Jonathan
eb29ac3d-490b-4845-8756-dd600d97468c
Le Conte, Steffi
6aac8cc9-f712-406f-a1bf-ddadab83f4a4
Airey, K
18c2f6d1-1180-49ff-bba0-e6fac3ae150c
Boardman, J
e7693d8d-533e-4dbd-adee-a820fbc74528
Hopkins, Claire
8ed0a5e3-92a3-4988-9c2b-b0e51a8a044c

Blackshaw, Helen, Vennik, Jane, Philpott, Carl, Thomas, Mike, Eyles, Caroline, Carpenter, James, Clarke, Caroline S., Morris, Stephen, Schilder, Anne G.M., Lund, Valerie, Little, Paul, Durham, Stephen, Denaxas, Spiros, Williamson, Elizabeth, Beard, David, Cook, Jonathan, Le Conte, Steffi, Airey, K, Boardman, J and Hopkins, Claire (2019) Expert panel process to optimise the design of a randomised controlled trial in chronic rhinosinusitis (the MACRO programme). Trials, 20, [230]. (doi:10.1186/s13063-019-3318-3).

Record type: Article

Abstract

Background
MACRO (Defining best Management for Adults with Chronic RhinOsinusitis) is an NIHR-funded programme of work designed to establish best practice for adults with chronic rhinosinusitis (CRS). The 7-year programme comprises three consecutive workstreams, designed to explore NHS care pathways through analysis of primary and secondary data sources, and to undertake a randomised controlled trial to evaluate a longer-term course of macrolide antibiotics and endoscopic sinus surgery for patients with CRS. A number of outstanding elements still required clarification at the funding stage. This paper reports an expert panel review process designed to agree and finalise the MACRO trial design, ensuring relevance to patients and clinicians whilst maximising trial recruitment and retention.
Methods
An expert panel consisting of the MACRO programme management group, independent advisors, and patient contributors, was convened to review current evidence and the mixed method data collected as part of the programme, and reach agreement on MACRO trial design. Specifically, agreement was sought for selection of macrolide antibiotic, use of oral steroids, inclusion of CRS phenotypes (with/without nasal polyps), and overall trial design.
Results
A 12-week course of clarithromycin was agreed as the main trial comparator, due to its increasing use as a first and second line treatment for patients with CRS, and the perceived need to establish its role in CRS management. Oral steroids will be used as a rescue medication during the trial, rather than routinely either pre or post trial randomisation, to limit any potential effects on surgical outcomes and better reflect current UK prescribing habits. Both CRS phenotypes will be included in a single trial to ensure the MACRO trial is both pragmatic and generalisable to primary care. A modified 3-arm trial design was agreed after intense discussions and further exploratory work. Inclusion criteria were amended to ensure that patients recruited would be considered eligible for the treatment offered in the trial, due to having already received appropriate medical therapy as deemed suitable by their ENT surgeon. A proposed 6-week run-in period prior to randomisation was removed due to the new criteria prior to randomisation.
Conclusion
The expert panel review process resulted in agreement on key elements and an optimal design for the MACRO trial, considered most likely to be successful in terms of both recruitment potential and ability to establish best management of patients with CRS.

Text
Optimising MACRO trial design v2 22-02-19 Clean - Accepted Manuscript
Download (75kB)

More information

Accepted/In Press date: 21 March 2019
e-pub ahead of print date: 23 April 2019

Identifiers

Local EPrints ID: 430470
URI: http://eprints.soton.ac.uk/id/eprint/430470
ISSN: 1745-6215
PURE UUID: 1b5153a9-29cd-4992-86da-84696efeb008
ORCID for Jane Vennik: ORCID iD orcid.org/0000-0003-4602-9805
ORCID for Paul Little: ORCID iD orcid.org/0000-0003-3664-1873

Catalogue record

Date deposited: 01 May 2019 16:30
Last modified: 12 Jul 2024 01:57

Export record

Altmetrics

Contributors

Author: Helen Blackshaw
Author: Jane Vennik ORCID iD
Author: Carl Philpott
Author: Mike Thomas
Author: Caroline Eyles
Author: James Carpenter
Author: Caroline S. Clarke
Author: Stephen Morris
Author: Anne G.M. Schilder
Author: Valerie Lund
Author: Paul Little ORCID iD
Author: Stephen Durham
Author: Spiros Denaxas
Author: Elizabeth Williamson
Author: David Beard
Author: Jonathan Cook
Author: Steffi Le Conte
Author: K Airey
Author: J Boardman
Author: Claire Hopkins

Download statistics

Downloads from ePrints over the past year. Other digital versions may also be available to download e.g. from the publisher's website.

View more statistics

Atom RSS 1.0 RSS 2.0

Contact ePrints Soton: eprints@soton.ac.uk

ePrints Soton supports OAI 2.0 with a base URL of http://eprints.soton.ac.uk/cgi/oai2

This repository has been built using EPrints software, developed at the University of Southampton, but available to everyone to use.

We use cookies to ensure that we give you the best experience on our website. If you continue without changing your settings, we will assume that you are happy to receive cookies on the University of Southampton website.

×