Recommendations to Standardize the Conduct of Clinical Trials Evaluating Novel Therapies for Perianal Fistulizing Crohn’s Disease
Bibliographic record
Abstract
BACKGROUND & AIMS: Perianal fistulizing Crohn's disease (PFCD) is a potentially debilitating condition with few systematically evaluated treatment options. We aimed to develop expert recommendations to standardize study design and conduct of randomized controlled trials of medical therapy for patients with PFCD. METHODS: We utilized a RAND/UCLA appropriateness method to develop a framework for conducting randomized controlled trials of medical therapy in PFCD. An expert international panel of 15 gastroenterologists, radiologists, and colorectal surgeons rated the appropriateness of survey statements in 2 rounds of voting. Median scores of 1 to ≤3.5, >3.5 to <6.5 and ≥6.5 to 9 on survey statements reflected panel ratings of inappropriate, uncertain, and appropriate, respectively. RESULTS: A total of 292 statements were rated in the final survey, of which 156 (53.4%) were considered appropriate and formed the basis for recommendations. Trial eligibility should be decided based on both clinical and radiological criteria and patients should exhibit a minimum degree of clinical disease activity at screening. The primary efficacy outcome should be either a co-primary or composite endpoint of clinical and radiological assessments depending on treatment phase (induction vs maintenance), recognizing that clinical endpoints are meaningful for patients and radiological outcomes are essential to objectively capture the burden of disease. There was uncertainty regarding definition of radiological remission, but presence of a perianal fluid collection of any size was considered inconsistent with radiologic remission. CONCLUSIONS: These recommendations may help to standardize study design and conduct and foster clinical development of much needed therapies for this debilitating manifestation of Crohn's disease.
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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 | 0.542 | 0.673 |
| Meta-epidemiology (narrow) | 0.004 | 0.004 |
| Meta-epidemiology (broad) | 0.006 | 0.011 |
| Bibliometrics | 0.009 | 0.008 |
| Science and technology studies | 0.004 | 0.009 |
| Scholarly communication | 0.011 | 0.009 |
| Open science | 0.013 | 0.007 |
| Research integrity | 0.031 | 0.029 |
| Insufficient payload (model declined to judge) | 0.005 | 0.006 |
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; the direct Gemma label and the distilled Codex classifier agree on what is shown here.
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".