Contributions of a survey and retrospective cohort study to the planning of a randomised controlled trial of corticosteroids in the treatment of paediatric septic shock
Bibliographic record
Abstract
BACKGROUND: Randomised controlled trials (RCTs) are challenging to conduct in a paediatric critical-care environment. Background work, including surveys and observational studies, is often used to determine disease estimates, sample sizes and design protocols when planning such RCTs. Our objective was to determine the necessity of performing a survey or a retrospective chart review or both when planning an RCT on corticosteroids in the treatment of paediatric septic shock. METHODS: We compared information on corticosteroid use for moderate to severe paediatric septic shock obtained from a survey of physician beliefs and stated practices with that obtained from a retrospective cohort study. The survey was conducted between February and March 2012 and the retrospective study included children from birth to 17 years of age admitted from January 2010 to June 2011. The survey and the retrospective study were conducted at four academic tertiary care centres in Canada. RESULTS: Survey responses from 23 physicians and retrospective data from 81 septic shock patients were included. The survey identified time to discontinuation of vasoactive infusions as the most feasible and clinically important outcome for an RCT on corticosteroids for paediatric septic shock. The retrospective chart review provided means and standard deviations for the suggested primary outcome, from which we could estimate sample sizes and justify the minimal clinically important difference. The survey found that physicians believe that patients with severe septic shock were most likely to benefit from corticosteroid administration but the majority stated they would be unwilling to randomise such patients, suggesting a lack of individual physician equipoise. The combined information from the survey and retrospective study suggested that enrolment of patients with moderate septic shock would be more feasible but that strategies would still have to be implemented to prevent open-label corticosteroid use. CONCLUSIONS: The survey provided valuable information on the choice of primary outcome, target population and physician equipoise. The retrospective study provided estimates of patient numbers, the minimal clinically important difference, evidence for community equipoise and physician practice patterns. Strong consideration should be given to performing both types of studies prior to conducting RCTs in paediatric critical-care environments.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.690 | 0.808 |
| Meta-epidemiology (narrow) | 0.002 | 0.002 |
| Meta-epidemiology (broad) | 0.004 | 0.004 |
| Bibliometrics | 0.006 | 0.006 |
| Science and technology studies | 0.002 | 0.003 |
| Scholarly communication | 0.007 | 0.006 |
| Open science | 0.002 | 0.005 |
| Research integrity | 0.004 | 0.004 |
| Insufficient payload (model declined to judge) | 0.003 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".