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)

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

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136 Scopus citations

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 ≥ 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.

Original languageEnglish (US)
Pages (from-to)2393-2400
Number of pages8
JournalBlood
Volume114
Issue number12
DOIs
StatePublished - Nov 20 2009

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

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    Greenberg, P. L., Sun, Z., Miller, K. B., Bennett, J. M., Tallman, M. S., Dewald, G., Paietta, E., Van Der Jagt, R., Houston, J., Thomas, M. L., Cella, D., & Rowe, J. M. (2009). 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). Blood, 114(12), 2393-2400. https://doi.org/10.1182/blood-2009-03-211797