[Background] More than 90% of low-risk MDS face refractory anemia and blood transfusion dependence for a long time. At present, recombinant human erythropoietin (EPO) is the main treatment, but the effective rate of single agent is low, which seriously affects the quality of life. The anemia treatment in low-risk MDS remains a high unmet clinical need, and new treatment strategies are urgently needed.

[Methods] This study is a multicenter, single-arm clinical study of EPO combined with all-trans retinoic acid (ATRA) and testosterone undecanoate in the treatment of anemia in patients with low-risk MDS

[Results] As of June 2022, a total of 26 patients were enrolled, with a median age of 67 years, 15 males and 11 females, with median hemoglobin of 63 g/L, EPO concentration > 500 IU/L in 9 patients, and the overall hemoglobin response rate of 65.4% (17/26). The hemoglobin response rates were 55.6% (5/9) in patients with EPO concentrations greater than > 500 IU/L and 70.6% (12/17) in patients with EPO concentrations ≤ 500, which were not statistically different. Among them, 112 known or putative mutational gene targets in hematologic malignancy were examined for mutations in 16 patients using a custom targeted next-generation sequencing (NGS) gene panel. The frequency of SF3B1 mutation was 43.8% (7/16). The hemoglobin response rate was 85.7% (6/7) and 55.6% (5/9) in patients with or without SF3B1 mutation.

[Conclusion]: This study showed that EPO combined with tretinoin and testosterone undecanoate regimen can effectively improve the efficacy of patients with lower risk MDS, and is not affected by EPO concentration. It has the better efficacy in patients with SF3B1 mutation and is worthy of further randomized controlled studies to verify the efficacy.

No relevant conflicts of interest to declare.

Author notes

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Asterisk with author names denotes non-ASH members.

© 2022 by The American Society of Hematology
2022
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