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Response to hypomethylating agents improves long-term outcomes for lower-risk patients with myelodysplastic syndrome in case-matched cohorts

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Abstract

Predictive factors for initiating hypomethylating agents’ (HMAs) treatment and the survival benefit of HMAs for lower-risk myelodysplastic syndrome (LR-MDS) are still unknown. This study evaluated the factors affecting the use of HMAs and compared long-term outcomes between best supportive care (BSC) and HMA groups after matching baseline clinical factors. Data of 353 patients diagnosed with LR-MDS by International Prognostic Scoring System between October 1992 and July 2013 were retrospectively analyzed. HMAs were administered continuously until a clinical response or progression. HMAs were administered to 243 patients with median 45 days (range 0–7078 days) after diagnosis, while 110 patients were treated with BSC. HMAs were administered over a median of 5 cycles and overall response was achieved in 104 patients (42.8%). The cumulative incidence of HMA treatment increased in higher-risk groups by other risk scoring systems. Three-year overall survival (OS) rate was higher in BSC group (69.1%) than HMA responders (47.4%, p = 0.065) or HMA non-responders (46.3%, p = 0.005). Among 162 case-matched cohorts, 3-year OS rates were comparable between the BSC group (67.1%) and HMA responders (58.1%, p = 0.914), while that of HMA non-responder was low (32.2%, p < 0.001). In the case-matched cohorts, HMA non-responder were associated with inferior OS rate in the multivariate analysis (hazard ratio 3.01, p = 0.001). Higher-risk groups by other clinical risk scoring systems among IPSS lower-risk patients showed an increased incidence of using HMAs. The OS rate of HMA responders among case-matched cohorts showed an improved OS rate similar to the BSC group.

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Authors and Affiliations

  1. Department of Hematology/Oncology, Kyungpook National University Hospital, 130 Dongdeok-ro, Jung-gu, Daegu, 41944, South Korea

    Dong Won Baek, Yoo Jin Lee, Sang Kyun Sohn & Joon Ho Moon

  2. School of Medicine, Kyungpook National University Hospital, Daegu, South Korea

    Dong Won Baek, Yoo Jin Lee, Hyunjeong Kim, Sang Kyun Sohn & Joon Ho Moon

  3. Department of Internal Medicine, Kyungpook National University Hospital, Daegu, South Korea

    Hyunjeong Kim

  4. Department of Hematology/Oncology, Chonnam National University Hwasun Hospital, Hwasun, South Korea

    Seo Yeon Ahn, Jae Sook Ahn & Hyeoung Joon Kim

  5. Busan National University Hospital, Busan, South Korea

    Ho Jin Shin

  6. Inje University Busan Baik Hospital, Busan, South Korea

    Won Sik Lee & Sang Min Lee

  7. Chungnam National University Hospital, Daejeon, South Korea

    Ik Chan Song

  8. Kosin University Gosper Hospital, Busan, South Korea

    Ho Sup Lee

  9. Gyeongsang National University Hospital, Jinju, South Korea

    Sung Woo Park

  10. Ulsan University Hospital, Ulsan, South Korea

    Yunsuk Choi

  11. Daegu Catholic University Medical Center, Daegu, South Korea

    Yoon Young Cho & Sung Hwa Bae

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  1. Dong Won Baek
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Correspondence to Joon Ho Moon.

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This study was approved by the Institutional Review Board of the each participating centers. All procedures performed in studies involving human participants were in accordance with the ethical standards of institutional and/or national research committees and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.

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Baek, D.W., Lee, Y.J., Kim, H. et al. Response to hypomethylating agents improves long-term outcomes for lower-risk patients with myelodysplastic syndrome in case-matched cohorts. Ann Hematol 97, 2309–2317 (2018). https://doi.org/10.1007/s00277-018-3458-7

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