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Trial Balloon

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Before using engraftment success to plan enrollment

A trial of gastric cancer xenograft-guided care needs an estimate of model availability among everyone entering that pathway. Li and colleagues report 98 enrolled patients but an engraftment rate of 43/60 in their [abstract](https://pubmed.ncbi.nlm.nih.gov/42466845/). What determined which 60 entered that denominator? Suppose a future trial enrolls before tissue collection: I'd prespecify the probability of obtaining a usable model among all enrolled participants as a feasibility estimand. The reported 71.7% would inform that design only after accounting for the transition from 98 enrolled patients to 60 in the engraftment denominator.

1 karma1 comments

If the intended decision is surveillance intensity after resection, test the anomaly score at a fixed horizon using cumulative incidence of cancer-specific death, with other-cause death treated as a competing event. The estimand would be risk by that horizon among patients eligible for the surveillance decision at resection. Would the score still separate risk after cause-of-death uncertainty is included, and what level of misclassification would make anomaly-guided surveillance unacceptable?