Premature Discontinuation of Pediatric Randomized Controlled Trials: A Retrospective Cohort Study

Stefan Schandelmaier, Yuki Tomonaga, Dirk Bassler, Joerg J. Meerpohl, Erik Von Elm, John You, Anette Bluemle, Francois Lamontagne, Ramon Saccilotto, Alain Amstutz, Theresa Bengough, Mihaela Stegert, Kelechi K. Olu, Kari A O Tikkinen, Ignacio Neumann, Alonso Carrasco-Labra, Markus Faulhaber, Sohail M. Mulla, Dominik Mertz, Elie A. AklXin Sun, Jason W. Busse, Ignacio Ferreira-González, Alain Nordmann, Viktoria Gloy, Heike Raatz, Lorenzo Moja, Rachel Rosenthal, Shanil Ebrahim, Per O. Vandvik, Bradley C. Johnston, Martin A. Walter, Bernard Burnand, Matthias Schwenkglenks, Lars G. Hemkens, Gordon Guyatt, Heiner C. Bucher, Benjamin Kasenda, Matthias Briel

Research output: Contribution to journalArticlepeer-review

Abstract

Objectives: To determine the proportion of pediatric randomized controlled trials (RCTs) that are prematurely discontinued, examine the reasons for discontinuation, and compare the risk for recruitment failure in pediatric and adult RCTs. Study design: A retrospective cohort study of RCTs approved by 1 of 6 Research Ethics Committees (RECs) in Switzerland, Germany, and Canada between 2000 and 2003. We recorded trial characteristics, trial discontinuation, and reasons for discontinuation from protocols, corresponding publications, REC files, and a survey of trialists. Results: We included 894 RCTs, of which 86 enrolled children and 808 enrolled adults. Forty percent of the pediatric RCTs and 29% of the adult RCTs were discontinued. Slow recruitment accounted for 56% of pediatric RCT discontinuations and 43% of adult RCT discontinuations. Multivariable logistic regression analyses suggested that pediatric RCT was not an independent risk factor for recruitment failure after adjustment for other potential risk factors (aOR, 1.22; 95% CI, 0.57-2.63). Independent risk factors were acute care setting (aOR, 4.00; 95% CI, 1.72-9.31), nonindustry sponsorship (aOR, 4.45; 95% CI, 2.59-7.65), and smaller planned sample size (aOR, 1.05; 95% CI 1.01-1.09, in decrements of 100 participants). Conclusion: Forty percent of pediatric RCTs were discontinued prematurely, owing predominately to slow recruitment. Enrollment of children was not an independent risk factor for recruitment failure.

Original languageEnglish
Pages (from-to)209-214.e1
JournalJournal of Pediatrics
Volume184
DOIs
Publication statusPublished - 2017

Keywords

  • Early termination of clinical trials (MeSH)
  • Pediatrics (MeSH)
  • Randomized controlled trials as a topic (MeSH)
  • Risk factors (MeSH)

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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