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Jennifer Elliott, PhD, Global Project Leader at Takeda Oncology, discusses the results of the phase 2 P2001 study presented at ASCO 2020. This clinical trial’s primary goal was to determine whether the combination of pevonedistat and azacitidine improves event-free survival (EFS) when compared with single-agent azacitidine in patients with high-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML), or low-blast acute myelogenous leukemia (LB AML). 

The results of the P2001 were promising. Significantly improved EFS was found for patients in the pevonedistat and azacitidine arm of the trial compared to the patients in azacitidine alone arm. As Dr. Elliott mentions, the complete response rate of the pevonedistat and azacitidine arm was almost double that of the azacitidine alone arm. The results also showed that 70% of patients who were transfusion dependent prior to the trial were transfusion independent after treatment with the combination of agents. As Dr. Elliott reinforces, not needing to go to a clinic frequently for transfusions improves patients’ overall quality of life. Furthermore, there was little to no toxicity increase with the addition of pevonedistat, which is a clinically significant finding as myelodysplastic syndrome, CMML, and LB-AML patients tend to be older.

For more information about myelodysplastic syndromes, CMML, AML, and other rare hematologic disorders, visit 



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