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Clinical Trial
. 2025 Feb;206(2):551-555.
doi: 10.1111/bjh.19967. Epub 2024 Dec 25.

CPX-351 +/- gemtuzumab ozogamicin as induction therapy for adult patients with newly diagnosed, favourable-intermediate risk, FLT3-ITD negative, AML: A pilot study

Chezi Ganzel  1 , Avraham Frisch  2 , Ofir Wolach  3 , Yakir Moshe  4 , Baher Krayem  2 , Neta Dor  1 , Etti Broide  5 , Emmanuel Benayoun  5 , Jacob M Rowe  1   2 , Yishai Ofran  1
Affiliations
Clinical Trial

CPX-351 +/- gemtuzumab ozogamicin as induction therapy for adult patients with newly diagnosed, favourable-intermediate risk, FLT3-ITD negative, AML: A pilot study

Chezi Ganzel et al. Br J Haematol. 2025 Feb.

Abstract

This pilot study evaluated CPX-351 in adults with newly diagnosed, favourable-intermediate risk, FLT3-ITD-negative AML. Twenty patients received CPX-351 for induction, with six also receiving gemtuzumab ozogamicin (GO). The complete response rate was 95%, with 42% achieving flow-based minimal residual disease (MRD) negativity post-induction. The 18-month leukaemia-free and overall survival estimates were 80% and 95% respectively. Adding GO appeared safe without prolonged cytopenias. Subclinical cardiotoxicity was observed in 25% of patients. The study demonstrated CPX-351's feasibility, with response and MRD-negativity rates comparable to standard '7 + 3' induction.

Keywords: AML; CPX‐351; MRD; cardiotoxicity; gemtuzumab ozogamicin; vyxeos.

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Conflict of interest statement

OW and YO serve as advisors to Medison.

Figures

FIGURE 1
FIGURE 1
Relapse‐free survival (A) and overall survival (B) of the entire cohort. Relapse‐free survival stratified by minimal/measurable disease (MRD) (C).
See this image and copyright information in PMC

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