• Sang Kyun Sohn, Joon Ho Moon, In Hee Lee, Jae Sook Ahn, Hyeoung Joon Kim, Joo Seop Chung, Ho Jin Shin, Sung Woo Park, Won Sik Lee, Sang Min Lee, Hawk Kim, Ho Sup Lee, Yang Soo Kim, Yoon Young Cho, Sung Hwa Bae, Ji Hyun Lee, Sung Hyun Kim, Ik Chan Song, Ji Hyun Kwon, and Yoo Jin Lee.
• Department of Hematology/Oncology, Kyungpook National University Hospital, Daegu, Korea.
• Korean J. Intern. Med. 2018 Nov 1; 33 (6): 1194-1202.

Background/AimsThis 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.MethodsA total of 279 H/VH risk MDS patients registered in the Korean MDS Working Party database were retrospectively analyzed.ResultsHMA 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.ConclusionFor 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.

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