TY - JOUR
T1 - Premature Discontinuation of Pediatric Randomized Controlled Trials
T2 - A Retrospective Cohort Study
AU - Schandelmaier, Stefan
AU - Tomonaga, Yuki
AU - Bassler, Dirk
AU - Meerpohl, Joerg J.
AU - Von Elm, Erik
AU - You, John
AU - Bluemle, Anette
AU - Lamontagne, Francois
AU - Saccilotto, Ramon
AU - Amstutz, Alain
AU - Bengough, Theresa
AU - Stegert, Mihaela
AU - Olu, Kelechi K.
AU - Tikkinen, Kari A O
AU - Neumann, Ignacio
AU - Carrasco-Labra, Alonso
AU - Faulhaber, Markus
AU - Mulla, Sohail M.
AU - Mertz, Dominik
AU - Akl, Elie A.
AU - Sun, Xin
AU - Busse, Jason W.
AU - Ferreira-González, Ignacio
AU - Nordmann, Alain
AU - Gloy, Viktoria
AU - Raatz, Heike
AU - Moja, Lorenzo
AU - Rosenthal, Rachel
AU - Ebrahim, Shanil
AU - Vandvik, Per O.
AU - Johnston, Bradley C.
AU - Walter, Martin A.
AU - Burnand, Bernard
AU - Schwenkglenks, Matthias
AU - Hemkens, Lars G.
AU - Guyatt, Gordon
AU - Bucher, Heiner C.
AU - Kasenda, Benjamin
AU - Briel, Matthias
PY - 2017
Y1 - 2017
N2 - 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.
AB - 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.
KW - Early termination of clinical trials (MeSH)
KW - Pediatrics (MeSH)
KW - Randomized controlled trials as a topic (MeSH)
KW - Risk factors (MeSH)
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U2 - 10.1016/j.jpeds.2017.01.071
DO - 10.1016/j.jpeds.2017.01.071
M3 - Article
AN - SCOPUS:85014140876
VL - 184
SP - 209-214.e1
JO - Journal of Pediatrics
JF - Journal of Pediatrics
SN - 0022-3476
ER -