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Amar Kelkar: Despite no difference OS/EFS, authors conclude young patients benefit due to lower relapse rates. We’re skeptical
Sep 5, 2023, 17:40

Amar Kelkar: Despite no difference OS/EFS, authors conclude young patients benefit due to lower relapse rates. We’re skeptical

Amar Kelkar, Physician at the Dana-Farber Cancer Institute and an Instructor in Medicine at Harvard Medical School, shared on Twitter:

“Out in The Lancet Haemotology, our Correspondence on AMLSG 09–09 by Döhner et al. The AMLSG09-09 study added gemtuzumab ozagamicin (GO) to chemo in patients w/ NPM1-mutated AML. Despite no difference OS/EFS, authors conclude young patients benefit due to lower relapse rates. We’re skeptical

The goal for lethal diseases like AML is cure. Benefit from treatment = survival. OS wasn’t even reported for young patients said to benefit from GO. Key point from the study protocol: ‘patient benefit can be shown by survival through an OS endpoint.’

Reduced cumulative incidence of relapse (CIR) is noted by authors, but we can’t ignore non-relapse mortality (NRM). Both form components of OS. CIR is helpful in some contexts but its real-world value for patients is arguable.

Post-hoc subgroup analyses have limitations, and should only be hypothesis-generating. They shouldn’t flip negative trial conclusions to positive.

AMLSG 09-09 joins trials showing mixed results on GO’s utility in AML. GO gives limited efficacy, with increased NRM offsetting any gains.

Our take? GO’s use should be confined to favourable risk, non-NPM1-mutated AML. Use cautiously.”

Source: Amar Kelkar/Twitter