Blood Journal
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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)

  1. Peter L. Greenberg1,
  2. Zhuoxin Sun2,
  3. Kenneth B. Miller3,
  4. John M. Bennett4,
  5. Martin S. Tallman5,
  6. Gordon Dewald6,
  7. Elisabeth Paietta7,
  8. Richard van der Jagt8,
  9. Jessie Houston6,9,
  10. Mary L. Thomas10,
  11. David Cella5, and
  12. Jacob M. Rowe11
  1. 1Stanford University Cancer Center, CA;
  2. 2Dana-Farber Cancer Institute, Boston, MA;
  3. 3Tufts-New England Medical Center, Boston, MA;
  4. 4Wilmot Cancer Center, University of Rochester Medical Center, NY;
  5. 5Northwestern University Feinberg School of Medicine, Chicago, IL;
  6. 6Mayo Clinic, Rochester, MN;
  7. 7Montefiore Medical Center, Bronx, NY;
  8. 8Ottawa Hospital, Ottawa, ON;
  9. 9Carle Clinic Association, Urbana, IL;
  10. 10Veterans Administration Palo Alto Health Care System, CA; and
  11. 11Rambam Medical Center and Technion, Haifa, Israel
  1. Presented in part at the 46th annual meeting of the American Society of Hematology, San Diego, CA, December 2004.


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.

  • Submitted March 18, 2009.
  • Accepted June 25, 2009.
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