Molecular MRD is strongly prognostic in patients with NPM1-mutated AML receiving venetoclax-based non-intensive therapy
- Author(s)
- Othman, J; Tiong, IS; O'Nions, J; Dennis, M; Mokretar, K; Ivey, A; Austin, MJ; Latif, AL; Amer, M; Chan, WY; Crawley, CR; Crolla, F; Cross, JW; Dang, R; Elliot, J; Fong, CY; Galli, S; Gallipoli, P; Hogan, F; Kalkur, P; Khan, AB; Krishnamurthy, P; Laurie, J; Loo, S; Marshall, S; Mehta, P; Murthy, V; Nagumantry, S; Pillai, S; Potter, N; Sellar, RS; Taylor, T; Zhao, R; Russell, NH; Wei, AH; Dillon, R;
- Journal Title
- Blood
- Publication Type
- epub ahead of print
- Abstract
- Assessment of measurable residual disease (MRD) by RT-qPCR is strongly prognostic in patients with NPM1-mutated AML treated with intensive chemotherapy, however there are no data regarding its utility in venetoclax-based non-intensive therapy, despite high efficacy in this genotype. We analysed the prognostic impact of NPM1 MRD in an international real-world cohort of 76 previously untreated patients with NPM1-mutated AML who achieved CR/CRi following treatment with venetoclax and hypomethylating agents (HMA) or low dose cytarabine (LDAC). 44 patients (58%) achieved bone marrow (BM) MRD negativity and a further 14 (18%) a reduction of ≥4 log10 from baseline as their best response, with no difference between HMA and LDAC. The cumulative rate of BM MRD negativity by the end of cycles 2, 4 and 6 was 25%, 47% and 50%. Patients achieving BM MRD negativity by the end of cycle 4 had 2-year overall (OS) of 84% compared to 46% if MRD positive. On multivariable analyses MRD negativity was the strongest prognostic factor. 22 patients electively stopped therapy in BM MRD negative remission after a median of 8 cycles with 2-year treatment-free remission of 88%. In patients with NPM1-mutated AML attaining remission with venetoclax combination therapies, NPM1 MRD provides valuable prognostic information.
- Publisher
- ASH
- Research Division(s)
- Blood Cells And Blood Cancer
- PubMed ID
- 37647641
- Publisher's Version
- https://doi.org/10.1182/blood.2023021579
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- Refer to copyright notice on published article.
Creation Date: 2023-09-07 09:17:13
Last Modified: 2023-09-07 09:26:22