Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia

RCT (n=431) found overall survival was longer (median 14.7 vs. 9.6 months; HR 0.66; 95% CI, 0.52-0.85; p<0.001) and incidence of remission was higher (36.7% vs. 17.9%; p<0.001) among patients who received azacitidine plus venetoclax than among those who received azacitidine alone

SPS commentary:

According to an editorial, although the therapeutic approach to AML has remained largely unchanged for past few decades, it is notable that more than 30% of children and young adults are cured after receiving chemotherapy alone (with variations in combination of an anthracycline and cytarabine — the “3 and 7” regimen), without stem-cell transplantation. It notes the outcome in adults older than 60 years of age is markedly inferior to that in younger persons, with a lower incidence of complete remission and long-term disease-free survival of approximately 10%. It discusses the trial findings, highlighting that although azacitidine–venetoclax is clearly of important benefit in older patients with AML, there appear to be persistent relapses despite continued monthly therapy (median, seven cycles), and some mechanisms of resistance have already been described. It suggests that at present, it remains unproven whether azacitidine–venetoclax should replace “3 and 7” in the larger group of older patients who are suitable candidates for cytotoxic chemotherapy. In addition, clinically significant myelosuppression in the range of what is seen with standard chemotherapy still occurs with azacitidine–venetoclax and insufficient data exist in patients with favourable-risk core-binding factor cytogenetic abnormalities, in whom high-dose cytarabine regimens are curative. There is also limited experience with more proliferative leukaemias.


New England Journal of Medicine

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