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A novel prognostic risk model for patients with R/R AML receiving venetoclax plus hypomethylating agents
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Off-label hypomethylating agents (HMA) and venetoclax (Ven) are frequently used to treat patients with R/R AML. However, predicting outcomes with these agents remains challenging. Shahswar et al. conducted a retrospective observational multicenter study of patients with R/R AML who had been treated with HMA + Ven and who reported to the Ven registry (NCT03662724; N = 240). The study aimed to assess the efficacy of HMA + Ven in a large patient cohort; determine genetic and clinical predictors of response, EFS, and OS; and develop an integrative model to support treatment decisions. The model was validated in 189 patients with AML treated with HMA + Ven in the first-line setting. Findings were published in Leukemia. |
| Key learnings |
| The ORR was 44%, and mOS was 7.9 months. The mortality rate was 75% at a median follow-up of 31.5 months. |
| Analysis of a subgroup with molecular information (n = 174) revealed risk factors for inferior survival, including the presence of extramedullary disease, HMA pretreatment, and NF1, PTPN11, FLT3, and TP53 mutations. SF3B1 mutation was identified as a favorable risk factor. |
| The identified risk factors were used to construct an HR-weighted risk model which allocated patients to risk groups with significantly differing survival outcomes; favorable (n = 46; mOS 21.4 months), intermediate (n = 75; mOS 7.5 months), and adverse (n = 53; mOS 4.6 months; p < 0.001). |
| This clinical-molecular, 3-tiered prognostic risk score (Ven-PRS) for outcomes in patients with R/R AML treated with HMA + Ven will support appropriate treatment selection in this high-risk population. |
Abbreviations: AML, acute myeloid leukemia; EFS, event-free survival; HMA, hypomethylating agent; HR, hazard ratio; m, median; ORR, overall response rate; OS, overall survival; R/R, relapsed/refractory; Ven, venetoclax.
- Shahswar R, et al. A novel prognostic risk model for patients with refractory/relapsed acute myeloid leukemia receiving venetoclax plus hypomethylating agents. Leukemia. Online ahead of print. DOI: 10.1038/s41375-024-02501-6.
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