Improving Power and Sample Size Calculation in Rehabilitation Trial Reports: A Methodological Assessment

Castellini, Greta; Gianola, Silvia; Bonovas, Stefanos; Moja, Lorenzo

doi:10.1016/j.apmr.2016.02.013

Cited by 21 publications

(12 citation statements)

References 28 publications

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“…The minority (4.2%) of randomized questions explicitly identified the delta value as the MCID with justification. These findings are in line with the limited number of previous studies investigating clinical significance reporting, wherein under-reporting was found [ 13 , 19 , 20 , 28 , 29 ]. Chan et al [ 13 ] found that, among a random sample of 27 RCTs in major medical journals, 20 articles included sample size calculations, 90% of which reported a delta value but only 11% stated that the delta value was chosen to reflect the MCID of the intervention.…”

Section: Discussionsupporting

confidence: 91%

Clinical significance in pediatric oncology randomized controlled treatment trials: a systematic review

Howard

Goddard²,

Rassekh

et al. 2018

Trials

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BackgroundClinical significance in a randomized controlled trial (RCT) can be determined using the minimal clinically important difference (MCID), which should inform the delta value used to determine sample size. The primary objective was to assess clinical significance in the pediatric oncology randomized controlled trial (RCT) treatment literature by evaluating: (1) the relationship between the treatment effect and the delta value as reported in the sample size calculation, and (2) the concordance between statistical and clinical significance. The secondary objective was to evaluate the reporting of methodological attributes related to clinical significance.MethodsRCTs of pediatric cancer treatments, where a sample size calculation with a delta value was reported or could be calculated, were systematically reviewed. MEDLINE, EMBASE, and the Cochrane Childhood Cancer Group Specialized Register through CENTRAL were searched from inception to July 2016.ResultsRCTs (77 overall; 11 and 66), representing 95 (13 and 82) randomized questions were included for non-inferiority and superiority RCTs (herein, respectively). The minority (22.1% overall; 76.9 and 13.4%) of randomized questions reported conclusions based on clinical significance, and only 4.2% (15.4 and 2.4%) explicitly based the delta value on the MCID. Over half (67.4% overall; 92.3 and 63.4%) reported a confidence interval or standard error for the primary outcome experimental and control values and 12.6% (46.2 and 7.3%) reported the treatment effect, respectively. Of the 47 randomized questions in superiority trials that reported statistically non-significant findings, 25.5% were possibly clinically significant. Of the 24 randomized questions in superiority trials that were statistically significant, only 8.3% were definitely clinically significant.ConclusionsA minority of RCTs in the pediatric oncology literature reported methodological attributes related to clinical significance and a notable portion of statistically insignificant studies were possibly clinically significance.Electronic supplementary materialThe online version of this article (10.1186/s13063-018-2925-8) contains supplementary material, which is available to authorized users.

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Section: Discussionsupporting

confidence: 91%

Clinical significance in pediatric oncology randomized controlled treatment trials: a systematic review

Howard

Goddard²,

Rassekh

et al. 2018

Trials

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“…However, in comparison to Castellini et al , who reviewed back pain trials published after 1968 and included 222 trials, we judge our search to have been comprehensive. [ 51 ]…”

Section: Discussionmentioning

confidence: 99%

A Systematic Review of Outcome Measures Use, Analytical Approaches, Reporting Methods, and Publication Volume by Year in Low Back Pain Trials Published between 1980 and 2012: Respice, adspice, et prospice

et al. 2016

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BackgroundIncreasing patient-reported outcome measures in the 1980s and 1990s led to the development of recommendations at the turn of the millennium for standardising outcome measures in non-specific low back pain (LBP) trials. Whether these recommendations impacted use is unclear. Previous work has examined citation counts, but actual use and change over time, has not been explored. Since 2011, there has been some consensus on the optimal methods for reporting back pain trial outcomes. We explored reporting practice, outcome measure use, and publications over time.MethodsWe performed a systematic review of LBP trials, searching the European Guidelines for the management of LBP, extending the search to 2012. We abstracted data on publications by year, outcome measure use, analytical approach, and approaches taken to reporting trials outcomes. Data were analysed using descriptive statistics and regression analyses.ResultsWe included 401 trials. The number of published trials per year has increased by a factor of 4.5 from 5.4 (1980–1999) to 24.4 (2000–2012). The most commonly used outcome measures have been the Visual Analogue Scale for pain intensity, which has slowly increased in use since 1980/81 from 20% to 60% of trials by 2012, and the Roland-Morris Disability Questionnaire, which rose to 55% in 2002/2003, and then fell back to 28% by 2012. Most trialists (85%) report between-group mean differences. Few (8%) report individual improvements, and some (4%) report only within-group analyses. Student’s t test, ANOVA, and ANCOVA regression, or mixed models, were the most common approaches to analysis.ConclusionsRecommendations for standardising outcomes may have had a limited or inconsistent effect on practice. Since the research community is again considering outcome measures and modifying recommendations, groups offering recommendations should be cognisant that better ways of generating trialist buy-in may be required in order for their recommendations to have impact.

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“…The respective differences between the reported and replicated sample sizes were then found to range from −237.5% to 84.2%8 and −93.3% to 60.6% 9. Furthermore, there was also a discrepancy between the planned and the actually recruited number of patients (recruited sample size smaller than planned sample size: 23.6%, recruited sample size larger than planned sample size: 58.4%) 11…”

Section: Discussionmentioning

confidence: 92%

Validity of sample sizes in publications of randomised controlled trials on the treatment of age-related macular degeneration: cross-sectional evaluation

et al. 2019

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ObjectiveThe aim of this cross-sectional study was to examine the completeness and accuracy of the reporting of sample size calculations in randomised controlled trial (RCT) publications on the treatment of age-related macular degeneration (AMD).MethodsA sample of 97 RCTs published between 2004 and 2014 was reviewed for the calculation of their sample size. It was examined whether a (complete) description of the sample size calculation was presented. Furthermore, the sample size was recalculated, whenever possible based on the published details, in order to verify the reported number of patients.Primary outcome measureThe primary endpoint of this cross-sectional investigation was a described sample size calculation that was reproducible, complete and correct (maximum tolerated deviation between reported and replicated sample size ±2 participants per trial arm).ResultsA total of 50 publications (52%) did not provide any information on the justification of the number of patients included. Only 17 publications (18%) provided all the necessary parameters for recalculation; 8 of 97 (8%, 95%-CI: 4% to 16%) publications achieved the primary endpoint. The median relative deviation between reported and recalculated sample sizes was 1%, with a range from −43% to +66%.ConclusionAlthough a transparent sample size legitimation is a crucial determinant of an RCT’s methodological validity, more than half of the RCT publications considered failed to report them. Furthermore, reported sample size legitimations were often incomplete or incorrect. In summary, clinical authors should pay more attention to the transparent reporting of sample size calculation, and clinical journal reviewers may opt to reproduce reported sample size calculations.SynopsisMore than half of the analysed RCT publications on the treatment of AMD did not report a transparent sample size calculation. Only 8% reported a complete and correct sample size calculation.

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Improving Power and Sample Size Calculation in Rehabilitation Trial Reports: A Methodological Assessment

Cited by 21 publications

References 28 publications

Clinical significance in pediatric oncology randomized controlled treatment trials: a systematic review

Clinical significance in pediatric oncology randomized controlled treatment trials: a systematic review

A Systematic Review of Outcome Measures Use, Analytical Approaches, Reporting Methods, and Publication Volume by Year in Low Back Pain Trials Published between 1980 and 2012: Respice, adspice, et prospice

Validity of sample sizes in publications of randomised controlled trials on the treatment of age-related macular degeneration: cross-sectional evaluation

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