Korean J Intern Med.  2018 Nov;33(6):1194-1202. 10.3904/kjim.2016.426.

No benefit of hypomethylating agents compared to supportive care for higher risk myelodysplastic syndrome

Affiliations
  • 1Department of Hematology/Oncology, Kyungpook National University Hospital, Daegu, Korea. yoojinlee0605@gmail.com
  • 2Department of Hematology/Oncology, Chonnam National University Hwasun Hospital, Hwasun, Korea.
  • 3Department of Hematology/Oncology, Pusan National University Hospital, Busan, Korea.
  • 4Department of Hematology/Oncology, Inje University Busan Baik Hospital, Busan, Korea.
  • 5Department of Hematology/Oncology, Ulsan University Hospital, Ulsan, Korea.
  • 6Department of Hematology/Oncology, Kosin University Gospel Hospital, Busan, Korea.
  • 7Department of Hematology/Oncology, Daegu Catholic University Medical Center, Daegu, Korea.
  • 8Department of Hematology/Oncology, Dong-A University Hospital, Busan, Korea.
  • 9Department of Hematology/Oncology, Chungnam National University Hospital, Daejeon, Korea.
  • 10Department of Hematology/Oncology, Chungbuk National University Hospital, Cheongju, Korea.

Abstract

BACKGROUND/AIMS
This study evaluated the role of hypomethylating agents (HMA) compared to best supportive care (BSC) for patients with high or very-high (H/VH) risk myelodysplastic syndrome (MDS) according to the Revised International Prognostic Scoring System.
METHODS
A total of 279 H/VH risk MDS patients registered in the Korean MDS Working Party database were retrospectively analyzed.
RESULTS
HMA therapy was administered to 205 patients (73.5%), including 31 patients (11.1%) who then received allogeneic hematopoietic cell transplantation (allo-HCT), while 74 patients (26.5%) received BSC or allo-HCT without HMA. The 3-year overall survival (OS) rates were 53.1% ± 10.7% for allo-HCT with HMA, 75% ± 21.7% for allo-HCT without HMA, 17.3% ± 3.6% for HMA, and 20.8% ± 6.9% for BSC groups (p < 0.001). In the multivariate analysis, only allo-HCT was related with favorable OS (hazard ratio [HR], 0.356; p = 0.002), while very poor cytogenetic risk (HR, 5.696; p = 0.042), age ≥ 65 years (HR, 1.578; p = 0.022), Eastern Cooperative Oncology Group performance status (ECOG PS) 2 to 4 (HR, 2.837; p < 0.001), and transformation to acute myeloid leukemia (AML) (HR, 1.901; p = 0.001) all had an adverse effect on OS.
CONCLUSIONS
For the H/VH risk group, very poor cytogenetic risk, age ≥ 65 years, ECOG PS 2 to 4, and AML transformation were poor prognostic factors. HMA showed no benefit in terms of OS when compared to BSC. Allo-HCT was the only factor predicting a favorable long-term outcome. The use of HMA therapy did not seem to have an adverse effect on the transplantation outcomes. However, the conclusion of this study should be carefully interpreted and proven by large scale research in the future.

Keyword

Myelodysplasia; Higher risk; Revised International Prognostic Scoring System; Hypomethylating agent

MeSH Terms

Cell Transplantation
Cytogenetics
Humans
Leukemia, Myeloid, Acute
Multivariate Analysis
Myelodysplastic Syndromes*
Retrospective Studies
Transplants
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