Few randomized trials in preterm birth prevention meet predefined usefulness criteria

Janneke van 't Hooft; Charlotte E van Dijk; Cathrine Axfors; Zarko Alfirevic; Martijn A Oudijk; Ben W J Mol; Patrick M Bossuyt; John P A Ioannidis

doi:10.1016/j.jclinepi.2023.08.016

Few randomized trials in preterm birth prevention meet predefined usefulness criteria

J Clin Epidemiol. 2023 Oct:162:107-117. doi: 10.1016/j.jclinepi.2023.08.016. Epub 2023 Aug 30.

Authors

Janneke van 't Hooft¹, Charlotte E van Dijk², Cathrine Axfors³, Zarko Alfirevic⁴, Martijn A Oudijk⁵, Ben W J Mol⁶, Patrick M Bossuyt⁷, John P A Ioannidis⁸

Affiliations

¹ Meta-Research Innovation Center at Stanford (METRICS), Stanford University, Palo Alto, CA, USA; Department of Obstetrics and Gynecology, Amsterdam UMC, University of Amsterdam, Vrije Universiteit Amsterdam, Amsterdam, Netherlands. Electronic address: [email protected].
² Department of Obstetrics and Gynecology, Amsterdam UMC, University of Amsterdam, Vrije Universiteit Amsterdam, Amsterdam, Netherlands.
³ Meta-Research Innovation Center at Stanford (METRICS), Stanford University, Palo Alto, CA, USA; Department for Women's and Children's Health, Uppsala University, Uppsala, Sweden.
⁴ Center for Women's Health Research, Liverpool Women's Hospital, Liverpool, United Kingdom.
⁵ Department of Obstetrics and Gynecology, Amsterdam UMC, University of Amsterdam, Vrije Universiteit Amsterdam, Amsterdam, Netherlands; Amsterdam Reproduction and Development Institute, Amsterdam, The Netherlands.
⁶ Department of Obstetrics and Gynecology, Monash University, Melbourne, Victoria, Australia; Aberdeen Centre for Women's Health Research, School of Medicine, University of Aberdeen, Aberdeen, UK.
⁷ Department of Clinical Epidemiology, Biostatistics and Bioinformatics, Amsterdam AUMC, University of Amsterdam, Amsterdam, The Netherlands.
⁸ Meta-Research Innovation Center at Stanford (METRICS), Stanford University, Palo Alto, CA, USA; Departments of Medicine, of Epidemiology and Population Health of Biomedical Data Science, and of Statistics, Stanford University, Palo Alto, CA, USA.

PMID: 37657614
DOI: 10.1016/j.jclinepi.2023.08.016

Abstract

Objectives: We operationalized a research usefulness tool identified through literature searches and consensus and examined if randomized controlled trials (RCTs) addressing preterm birth prevention met predefined criteria for usefulness.

Study design and setting: The usefulness tool included eight criteria combining 13 items. RCTs were evaluated for compliance with each item by multiple assessors (reviewer agreement 95-98%). Proportions of compliances with 95% confidence interval (CI) were calculated and change over time was assessed using ≧ 2010 as a cutoff.

Results: Among 347 selected RCTs, published within 56 preterm birth Cochrane reviews, only 36 (10%, 95% CI = 7-14%) met more than half of the usefulness criteria. Compared to trials before 2010, recent trials used composite or surrogate (less informative) outcomes more often (13% vs. 25%, relative risk 1.91, 95% CI = 1.21-3.00). Only 16 trials reflected real practice (pragmatism) in design (5%, 95% CI = 3-7%), with no improvements over time. No trials reported involvement of mothers to reflect patients' research priorities and outcomes selection. Recent trials were more transparent.

Conclusion: Few preterm birth prevention RCTs met more than half of the usefulness criteria but most of usefulness criteria are improving after 2010. Use of informative outcomes, patient centeredness, pragmatism and transparency should be key targets for future research planning.

Keywords: Clinical research; Pragmatism; Preterm birth; Randomized controlled trials; Transparency; Usefulness.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Female
Humans
Infant, Newborn
Premature Birth* / prevention & control
Randomized Controlled Trials as Topic