Analysis and Reporting of Randomized Trials in Cleft Palate Surgery: Learning from the Timing of Primary Surgery (TOPS) Trial

被引：1

作者：

Fell, Matthew ^{[1
,2
,8
]}

Phippen, Ginette ^{[2
]}

van Eeden, Stephanie ^{[3
]}

Chong, David ^{[4
]}

Swan, Marc C. ^{[2
]}

van Eeden, Simon ^{[5
]}

Carlin, John B. ^{[6
,7
]}

机构：

[1] Univ Bristol, Cleft Collect, Bristol, England

[2] Oxford Univ Hosp NHS Fdn Trust, Salisbury NHS Fdn Trust, Spires Cleft Ctr, Oxford, Oxon, England

[3] Newcastle Univ, Sch Educ Commun & Language Sci, Newcastle Upon Tyne, Northumberland, England

[4] Royal Childrens Hosp, Plast & Maxillofacial Surg, Melbourne, Vic, Australia

[5] Alder Hey Childrens & Aintree Univ Hosp, Liverpool, Merseyside, England

[6] Murdoch Childrens Res Inst, Melbourne, Vic, Australia

[7] Univ Melbourne, Melbourne Sch Populat & Global Hlth, Melbourne, Vic, Australia

[8] Oxford Univ Hosp NHS Fdn Trust, Spires Cleft Ctr, Oxford, England

来源：

CLEFT PALATE CRANIOFACIAL JOURNAL | 2025年 / 62卷 / 08期

关键词：

assessment; cleft palate; evidence-based practice; OUTCOME MEASURES; SPEECH; LIP; 5-YEAR-OLDS; STANDARDS; COHORT; CARE;

D O I：

10.1177/10556656241253949

中图分类号：

R78 [口腔科学];

学科分类号：

1003 ;

摘要：

The Timing of Primary Surgery (TOPS) trial was published August 2023 in the New England Journal of Medicine and is a milestone achievement for a study focused on cleft palate. Due to the complexity of outcome reporting in cleft and the rarity of such comparative trials, TOPS presents a useful opportunity to critically review the design, analysis and reporting strategies utilised. This perspective article focused on the inclusion of participants, the choice of the primary outcome measure and the analysis of ordinal data within the trial. Considerations for future comparative studies in cleft care are discussed.

引用

页码：1436 / 1442

页数：7

共 44 条

[1] A Standard Set of Outcome Measures for the Comprehensive Appraisal of Cleft Care [J].

Allori, Alexander C. ;

Kelley, Thomas ;

Meara, John G. ;

Albert, Asteria ;

Bonanthaya, Krishnamurthy ;

Chapman, Kathy ;

Cunningham, Michael ;

Daskalogiannakis, John ;

de Gier, Henriette ;

Heggie, Andrew A. ;

Hernandez, Cristina ;

Jackson, Oksana ;

Jones, Yin ;

Kangesu, Loshan ;

Koudstaal, Maarten J. ;

Kuchhal, Rajiv ;

Lohmander, Anette ;

Long, Ross E., Jr. ;

Magee, Leanne ;

Monson, Laura ;

Rose, Elizabeth ;

Sitzman, Thomas J. ;

Taylor, Jesse A. ;

Thorburn, Guy ;

van Eeden, Simon ;

Williams, Chris ;

Wirthlin, John O. ;

Wong, Karen W. .

CLEFT PALATE CRANIOFACIAL JOURNAL, 2017, 54 (05) :540-554

[2]

Auslander A., 2022, CLEFT PALATE-CRAN J, V59, pS47

[3] Benchmarking Speech, Velopharyngeal Function Outcomes and Surgical Characteristics Following the Sommerlad Protocol and Palate Repair Technique [J].

Baillie, Lauren ;

Sell, Debbie .

CLEFT PALATE-CRANIOFACIAL JOURNAL, 2020, 57 (10) :1197-1215

[4] A Cross-Sectional Cohort Study of Speech in Five-Year-Olds With Cleft Palate ± Lip to Support Development of National Audit Standards BENCHMARKING SPEECH STANDARDS IN THE UNITED KINGDOM [J].

Britton, Lorraine ;

Albery, Liz ;

Bowden, Melanie ;

Harding-Bell, Anne ;

Phippen, Ginette ;

Sell, Debbie .

CLEFT PALATE-CRANIOFACIAL JOURNAL, 2014, 51 (04) :431-451

[5] Estimands: bringing clarity and focus to research questions in clinical trials [J].

Clark, Timothy Peter ;

Kahan, Brennan C. ;

Phillips, Alan ;

White, Ian ;

Carpenter, James R. .

BMJ OPEN, 2022, 12 (01)

[6] A randomised controlled trial comparing palate surgery at 6 months versus 12 months of age (the TOPS trial): a statistical analysis plan [J].

Conroy, Elizabeth J. ;

Cooper, Rachael ;

Shaw, William ;

Persson, Christina ;

Willadsen, Elisabeth ;

Munro, Kevin J. ;

Williamson, Paula R. ;

Semb, Gunvor ;

Walsh, Tanya ;

Gamble, Carrol .

TRIALS, 2021, 22 (01)

[7] Current Surgical Practice for Children Born with a Cleft lip and/or Palate in the United Kingdom [J].

Fell, Matthew ;

Davies, Alex ;

Davies, Amy ;

Chummun, Shaheel ;

Cobb, Alistair R. M. ;

Moar, Kanwalraj ;

Wren, Yvonne .

CLEFT PALATE-CRANIOFACIAL JOURNAL, 2022, :679-688

[8] Range and Frequency of Congenital Malformations Among Children With Cleft Lip and/or Palate [J].

Fitzsimons, K. J. ;

Hamilton, M. J. ;

van der Meulen, J. ;

Medina, J. ;

Wahedally, M. A. H. ;

Park, M. H. ;

Russell, C. J. H. .

CLEFT PALATE CRANIOFACIAL JOURNAL, 2023, 60 (08) :917-927

[9] Timing of Primary Surgery for Cleft Palate [J].

Gamble, Carrol ;

Persson, Christina ;

Willadsen, Elisabeth ;

Albery, Liz ;

Andersen, Helene Soegaard ;

Antoneli, Melissa Zattoni ;

Appelqvist, Malin ;

Aukner, Ragnhild ;

Bodling, Pia ;

Bowden, Melanie ;

Brunnegard, Karin ;

Cairns, Gillian ;

Calladine, Samantha ;

Campbell, Linsay ;

Clayton-Smith, Jill ;

Cooper, Rachael ;

Conroy, Elizabeth ;

El-Angbawi, Ahmed ;

Emborg, Berit Kildegaard ;

Wikman, Josefin Enfalt ;

Fitzpatrick, Beth ;

Fukushiro, Ana Paula ;

de Azevedo Bento Goncalves, Cristina Guedes ;

Havstam, Christina ;

Hvistendahl, Anne Katherine ;

Jorgensen, Line Dahl ;

Klinto, Kristina ;

Kvinnsland, Marit Berntsen ;

Larham, Catriona ;

Lemvik, Jorunn ;

Leturgie, Louise ;

Liljerehn, Eva ;

Lodge, Natalie ;

Lohmander, Anette ;

McMahon, Siobhan ;

Mehendale, Felicity ;

Miguel, Haline Coracine ;

Moe, Marianne ;

Nielsen, Joan Bogh ;

Nyberg, Jill ;

Pedersen, Nina-Helen ;

Phippen, Ginette ;

Alvarez Piazentin-Penna, Silvia Helena ;

Patrick, Kathryn ;

Pliskin, Lindsay ;

Rigby, Lucy ;

Semb, Gunvor ;

Southby, Lucy ;

Sporre, Maria ;

Taleman, Ann-Sofie Bjorkman .

NEW ENGLAND JOURNAL OF MEDICINE, 2023, 389 (09) :795-807

[10] Objective Randomised Blinded Investigation With Optimal Medical Therapy of Angioplasty in Stable Angina (ORBITA) and coronary stents: A case study in the analysis and reporting of clinical trials [J].

Gelman, Andrew ;

Carlin, John B. ;

Nallamothu, Brahmajee K. .

AMERICAN HEART JOURNAL, 2019, 214 :54-59

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