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Hypomethylating agents (HMAs) show benefit in AML rather than in intermediate/high-risk MDS based on genetic mutations in epigenetic modification (EMMs): from a retrospective study

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Abstract

Since HMAs were recommended for treatments in AML and MDS, we wondered whether HMAs could provide similar benefit to AML and intermediate/high-risk MDS under the direction of next-generation sequencing. Here we retrospectively analyzed the prognosis of 176 AML and 128 intermediate/high-risk MDS patients treated with HMAs or non-HMA regimens. For AML, HMAs regimen was related to better CR rate compared with non-HMA regimen in elder cohort, while the situation was the opposite in younger cohort. In consolidation phase, EMM (+) patients could benefit from HMAs regimen. Relapsed AML patients receiving HMAs regimen rather than non-HMA regimen had better post-relapse survival. Multivariate analysis identified HMA regimen as an independent prognostic factor for OS in EMM (+) cohort. For intermediate/high-risk MDS patients not undergoing HSCT, however, HMA regimen showed no survival advantage in EMM (+) cohort and was conversely associated with shorter survival in EMM (-) cohort compared with non-HMA regimen. And among those undergoing HSCT, HMA prior to HSCT predicted poor prognosis compared with upfront HSCT regardless of the existence of EMMs. Therefore, HMAs had better therapeutic value in AML rather than in intermediate/high-risk MDS based on EMMs.

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Data availability

The data that support the findings of this study are available on request from the corresponding author. The data are not publicly available due to privacy.

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Funding

This work was partially supported by grants from the Chinese National Major Project for New Drug Innovation (2019ZX09201002003), National Natural Science Foundation of China (82030076, 82070161, 81970151, 81670162 and 81870134), Shenzhen Science and Technology Foundation (JCYJ20190808163601776, JCYJ20200109113810154), Shenzhen Key Laboratory Foundation (ZDSYS20200811143757022), Sanming Project of Shenzhen (SZSM202111004).

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Authors and Affiliations

  1. Medicine School, Nankai University, 94 Weijin Road, Tianjin, 300071, China

    Ruiqi Wang, Hong Wang, Mengzhen Wang & Li Yu

  2. Department of Hematology, Chinese PLA General Hospital, Medical School of Chinese PLA, 28 Fuxing Road, Beijing, 100853, China

    Ruiqi Wang, Bianhong Wang, Hong Wang, Mengzhen Wang, Yu Jing, Xiaoning Gao & Li Yu

  3. Department of Hematology-Oncology, Carson International Cancer Center, Shenzhen University General Hospital, Shenzhen University Health Science Center, 1098 Xueyuan Avenue, Shenzhen, 518060, China

    Yuanyuan Xu & Li Yu

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  1. Ruiqi Wang
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  3. Bianhong Wang
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  4. Hong Wang
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  7. Xiaoning Gao
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Correspondence to Xiaoning Gao or Li Yu.

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Wang, R., Xu, Y., Wang, B. et al. Hypomethylating agents (HMAs) show benefit in AML rather than in intermediate/high-risk MDS based on genetic mutations in epigenetic modification (EMMs): from a retrospective study. Ann Hematol 103, 61–71 (2024). https://doi.org/10.1007/s00277-023-05438-5

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  • DOI: https://doi.org/10.1007/s00277-023-05438-5

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