Many new HR-MDS therapies are likely to deliver incremental survival gains rather than large overall survival improvements, particularly given an apparent survival plateau around 2 years. Future confirmatory programs should, therefore, plan for incremental improvements, prespecify handling of transplantation and post-protocol therapies, and integrate molecular stratification/enrichment to reduce variance. Adaptive designs should be used to stop futile programs earlier and to expand adequately powered trials when emerging data support clinically meaningful benefit.
Zeidan et al. (Sat,) studied this question.