In patients with lower-risk myelodysplastic syndrome (MDS), androgen therapy for managing disease-related cytopenias can result in hematologic improvement, according to results from a retrospective study published in the British Journal of Haematology.

Treatment for patients with lower-risk MDS aims to reduce cytopenia-related complications, limit or eliminate the need for transfusions, and maintain patients’ quality of life. Androgens have been used in this patient population and assessed retrospectively for efficacy.

Researchers enrolled 137 patients who had received either danazol (56 patients) or oxymetholone (81 patients) as first-line (107 patients) or second-line (30 patients) treatment between 2001 and 2018 for MDS-related cytopenia. Patients were classified as having low or intermediate-1 risk. Patients with prior exposure to erythroid-stimulating agents or hypomethylating agents were excluded.

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At the time of analysis, androgen therapy was continued in 17.5% of patients (24/137) and discontinued in 80.3% (110/137), with 3 patients lost to follow-up. Treatment was discontinued in 9.1% of patients due to disease progression or lack of efficacy, in 9.1% of patients due to adverse events or intolerance, and in 28.1% of patients due to other reasons.

Of the discontinuations related to adverse events, 16 were due to liver function abnormalities, 1 was due to cerebral venous thrombosis, 1 was due to skin rash, and 1 was due to cerebrovascular accident.

A total of 43.8% (60/137) of patients achieved the primary endpoint of hematologic improvement, with 26.7% experiencing erythroid improvement, 40.8% experiencing platelet improvement, and 26.9% experiencing neutrophil improvement.

The median time to first hematologic improvement achievement was 19 months overall, 39 months for erythroid improvement, 16 months for platelet improvement, and 33 months for neutrophil improvement. In the 95 patients with multilineage cytopenias, 30.5% achieved single-lineage responses and 18.9% achieved multilineage responses.

Median duration of response was 50 months overall, 69 months for erythroid improvement, 52 months for platelet improvement, and 33 months for neutrophil improvement.

After median follow-up of 28.8 months, 23 patients progressed to higher-risk MDS, and 15 patients progressed to acute myeloid leukemia. Three patients had clonal evolution, and 34 patients died.

Estimated 5-year overall survival was 66.5%, and estimated 5-year progression-free survival was 48.6%. Achieving erythroid improvement was correlated with improved survival outcomes.

“Further prospective studies are warranted to identify the efficacy and the precise mechanism of androgens, as well as to establish optimal treatment strategies for [patients with] lower-risk MDS,” concluded the authors.


  1. Choi EJ, Lee JH1, Park HS, et al. Androgen therapy for patients with lower-risk myelodysplastic syndrome and significant cytopenia: a retrospective study [published online August 1, 2019]. Br J Haematol. doi:10.1111/bjh.16121

This article originally appeared on Hematology Advisor