Treatment of Myelodysplastic Syndrome with Azacitidine and Erythropoietin.

PURPOSE: Myelodysplastic syndrome (MDS) is a group of clonal bone marrow disorders leading to bone marrow failure and acute myeloid leukemia. With better understanding of the biology of MDS, newer treatment options like therapy with the hypomethylating agents, azacitidine and decitabine have proven beneficial. Major randomized controlled trials by CALGB and others have demonstrated the efficacy of azacitidine. Growth factor use was prohibited in these trials. However, in the community, most patients have continued to get growth factor support along with azacitidine therapy. Since there is lack of evidence backing such clinical practice, we attempted to review response to therapy in patients receiving combination therapy with azacitidine and erythropoietin.

METHODS: We identified MDS patients treated with concomitant azacitidine and erythropoietin at our institution over the last five years. Data collected from retrospective chart review of these patients was statistically analyzed.

RESULTS: Sixteen patients with concomitant azacitidine and erythropoietin therapy were identified. Complete remissions were seen in 31%(5/16) of the patients. Induction of partial remissions in our study was 25% (4/16). Thirty one percent (5/16) of patients had hematologic improvement not meeting criteria for partial remission. Thirteen percent of patients (2/16) did not benefit at all from azacitidine and erythropoietin support. The median number of cycles received for adequate response was six. A total response (complete remission, partial remission and hematological improvement) of 87%(14/16,) was seen with combination therapy, which is significantly higher than previously published total response of 44–60%.

CONCLUSION: Azacitidine with erythropoietin support provides important clinical benefits for patients with MDS. There was a trend towards a higher rate of complete and partial responses in these patients than reported earlier in patients who received azacitidine alone. Randomized controlled studies will be needed to validate this further.