WolverHeme Happy Hour LFGooo! The Rise, the Fall, and the Resurrection of Gemtuzumab in AML
Mar 11, 2025
Join Dr. Anand Patel, a hematology MD and leading educator, alongside Dr. Charlie Foucar, a brilliant hematologist, as they delve into the controversial journey of gemtuzumab in treating acute myeloid leukemia (AML). They discuss its dramatic rise and fall, backed by intriguing studies and data interpretations. Hear them analyze treatment outcomes and dosing strategies while unpacking biases in clinical research. Their engaging banter, peppered with humor and personal anecdotes, makes complex medical discussions entertaining and enlightening!
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Gemtuzumab's Initial Approval and Withdrawal
- Gemtuzumab ozogamycin is an antibody-drug conjugate targeting CD33 in AML, initially approved in 2000 based on phase 2 studies.
- It was withdrawn after a phase 3 SWOG study showed no survival benefit and higher toxicity including fatal induction deaths.
Hills Meta-analysis Revived Gemtuzumab
- The Hills meta-analysis pooled data from 5 trials and showed a 20% overall survival benefit with gemtuzumab in favorable risk AML.
- This subgroup analysis drove gemtuzumab's 2017 FDA re-approval despite limited core binding factor representation and trial heterogeneity.
Small Core Binding Factor Subgroups Bias Results
- Core binding factor patients composed a tiny fraction of gemtuzumab trials, often with as few as 9-12 patients per study.
- Outcomes in control arms were often subpar, raising concerns about subgroup imbalances inflating apparent benefits.