Congratulations go to lead author Dr. Gupta and lead biostatistician Dr. John Kairalla for winning the David Sackett Trial of the Year for 2024. A link to the New England Journal paper is below. This represents one of the most successful cancer trials ever conducted. It was stopped early, Kairalla was responsible for the design, including safety monitoring plans. The long-term event-free survival on the experimental arm was 96%, cutting relapse rates by 66%. No new randomized trial to improve event-free survival is feasible.
I would be interested in any ideas you might share with the group when the outcome of a cancer treatment is so curative that randomized trials are not feasible. One idea in children is to collect national death index identifiers in a new single arm study where everyone gets the newly established treatment so that 25 years into the future, you can assess truly long-term all-cause mortality. Another is to collect genetic material at the time of diagnosis to identify that small resistant subset.
Best wishes to all,
Jonathan J Shuster, PhD
Professor Emeritus
University of Florida
College of Medicine
Blinatumomab in standard risk pediatric B-acute lymphoblastic leukemia
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Jonathan Shuster
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