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high dose cytarabine exposure does not appear to pre- clude a response to this combination therapy. With regards to patients with KMT2A PTD mutations, as noted, 2 patients were able to obtain response to this combina- tion therapy while the other 2 were refractory to this treatment with no definitive features to explain difference in outcomes. In conclusion, we successfully determined the recommended phase 2 dose for this novel treatment regimen. The regimen had modest toxicities beyond uncomplicated (though prolonged) myelosuppression, and we propose that the study provides a framework for larger
efficacy studies for AML patients with the uncommon but biologically distinct molecular feature of KMT2A PTD.
Acknowledgments
The authors thank all of the patients who participated in this trial as well as the dedicated teams who cared for them in the James Cancer Hospital inpatient/outpatient Leukemia Units.
Funding
This work was supported by: U01 CA76576-05, P30 CA016058 and SPORE P50-CA140158.
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haematologica | 2018; 103(6)
A novel regimen for relapsed/refractory adult acute myeloid leukemia
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