Treatment with Erythropoietin and G-CSF Improves Survival in MDS Patients with Low Transfusion Need.

Erythropoietin (Epo) and G-CSF alleviates anemia and increases quality of life in myelodysplastic syndrome (MDS). In patients with low transfusion need and low S-Epo level the erythroid response rate reaches 70% with a median response duration of two years, while patients with high transfusion need and high S-Epo level respond poorly and do not benefit from treatment. A recent study comparing Epo and G-CSF treated MDS patients with untreated historical controls indicates that the treatment can be given safely as chronic administration. To investigate the affects of growth factor treatment on outcome in patients with different probability of response, we compared treated patients included in three Nordic MDS Group studies (n=123) with untreated patients from Pavia, Italy (n=240). In both cohorts data on level of transfusion need and S-Epo level was recorded at baseline. All patients were transfusion dependent (n=176) or anemic with a hemoglobin level below 10 g/dl (n=187). An erythroid response, defined as at least abolition of transfusion need, was observed in 41% of treated patients, with a median response duration of 23 months (range 3–116+). Multivariate Cox regression analysis showed that treated patients with transfusion need of less than 2 units of RBC per month had a survival benefit, with HR 0.57 (P=0.015), while no difference in survival was observed in patients with higher transfusion need (P=0.36). There was no significant impact on risk of leukemic transformation in patients with the low (P=0.75) or high (P=0.21) transfusion need. This is the first study implicating that treatment of anemia in MDS with Epo and G-CSF in selected patients has a positive impact on outcome. Whether this effect is mediated by limiting the iron overload or counteracting other negative effects chronic anemia remains to be investigated.