Measurable/minimal residual disease (MRD) assessment by qPCR was strongly associated with clinical outcomes in patients with NPM1-mutated acute myeloid leukemia (AML) who achieved complete remission (CR)/complete remission with incomplete recovery count (CRi) on venetoclax combination therapies.


“Flow cytometric MRD assessment is prognostic of clinical outcomes in venetoclax-treated patients with various AML mutations,” stated Jad Othman (Guy’s and St Thomas’ NHS Foundation Trust, UK)1,2 during a presentation at the 2022 annual meeting of the American Society of Hematology. The presented study aimed to determine whether MRD by qPCR is prognostic of clinical outcomes in patients with NPM1-mutated AML who achieved CR/CRi in the real world on venetoclax plus either low-dose cytarabine or a hypomethylating agent.

In a cohort of 55 patients, the best MRD response rates in the first 6 months of therapy were as follows: MRD undetectable (46%), at least 4 log reduction (19%), less than 4 log reduction (35%). After a median follow-up of 24.3 months, the deepest MRD reduction within the first 6 months was strongly related to overall survival (OS; P=0.00027) and event-free survival (EFS; P<0.0001), favoring those with undetectable MRD over other subgroups. Othman added that an MRD cut-off of less than 0.005 NPM1 copies/100 ABL was the best discriminator for OS (P<0.0001) and EFS (P<0.0001). He noted that peripheral blood MRD, although less sensitive than bone marrow MRD, may be adequately sensitive to predict clinical outcomes.

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