Treatment of myelodysplastic syndrome patients with erythropoietin with or without granulocyte colony-stimulating factor: results of a prospective randomized phase 3 trial by the Eastern Cooperative Oncology Group (E1996).

Abstract

This phase 3 prospective randomized trial evaluated the efficacy and long-term safety of erythropoietin (EPO) with or without granulocyte colony-stimulating factor plus supportive care (SC; n = 53) versus SC alone (n = 57) for the treatment of anemic patients with lower-risk myelodysplastic syndromes. The response rates in the EPO versus SC alone arms were 36% versus 9.6%, respectively, at the initial treatment step, 47% in the EPO arm, including subsequent steps. Responding patients had significantly lower serum EPO levels (45% vs 5% responses for levels < 200 mU/mL vs > or = 200 mU/mL) and improvement in multiple quality-of-life domains. With prolonged follow-up (median, 5.8 years), no differences were found in overall survival of patients in the EPO versus SC arms (median, 3.1 vs 2.6 years) or in the incidence of transformation to acute myeloid leukemia (7.5% and 10.5% patients, respectively). Increased survival was demonstrated for erythroid responders versus nonresponders (median, 5.5 vs 2.3 years). Flow cytometric analysis showed that the percentage of P-glycoprotein(+) CD34(+) marrow blasts was positively correlated with longer overall survival. In comparison with SC alone, patients receiving EPO with or without granulocyte colony-stimulating factor plus SC had improved erythroid responses, similar survival, and incidence of acute myeloid leukemia transformation.

• Authors: Peter L Greenberg, Zhuoxin Sun, Kenneth B Miller, John M Bennett, Martin S Tallman, Gordon Dewald, Elisabeth Paietta, Richard van der Jagt, Jessie Houston, Mary L Thomas, David Cella, Jacob M Rowe
• Journal: Blood (Blood) Vol. 114 Issue 12 Pg. 2393-400 (Sep 17 2009) ISSN: 1528-0020 [Electronic] United States
• PMID: 19564636 (Publication Type: Clinical Trial, Phase III, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, U.S. Gov't, P.H.S.)

Chemical References

• Recombinant Proteins
• Erythropoietin
• Granulocyte Colony-Stimulating Factor

Topics

• Aged
• Drug Therapy, Combination
• Erythropoietin (administration & dosage)
• Female
• Flow Cytometry
• Granulocyte Colony-Stimulating Factor (administration & dosage)
• Humans
• Injections, Subcutaneous
• Male
• Myelodysplastic Syndromes (drug therapy)
• Prognosis
• Prospective Studies
• Quality of Life
• Recombinant Proteins
• Risk Factors
• Survival Rate
• Treatment Outcome