Authors' objectives:

Anemia is relatively common in patients with either hematologic or solid tissue malignancies. When cancer treatment or the disease itself decreases production of or impairs response to endogenous erythropoietin, epoetin treatment may correct the resulting anemia. This systematic review compares outcomes of managing anemia with epoetin (and red blood cell [RBC]transfusion used as necessary) with using RBC transfusion alone. Four groups of patients with malignancy are included: (1) patients with anemia or at risk of anemia resulting primarily from cancer therapy; (2) patients with anemia resulting primarily from their malignant disease and who may also be receiving cancer therapy; and patients who are anemic as a result of bone marrow ablation prior to (3) allogeneic or (4) autologous stem-cell transplantation.

Authors' results and conclusions: For patients with anemia resulting primarily from cancer therapy, epoetin reduces the odds of transfusion. The overall number needed to treat (NNT) is 4.4 (95 percent confidence interval [CI], 3.6 to 6.1), which suggests four to five patients must be treated to spare one patient from transfusion. Sensitivity analysis found a smaller magnitude of risk reduction for double-blinded compared with unblinded studies. A large, double-blinded randomized trial, not yet published, found improvement in quality-of-life scores with epoetin. Assessment of the study methodology and clinical significance of the findings awaits publication of the full report. The most robust evidence that epoetin improves outcomes is from trials in patient groups with baseline hemoglobin (Hb) at or below 10 g/dL. The evidence is not adequate to determine whether outcomes are superior when epoetin treatment is initiated at higher hemoglobin thresholds. As many as one-half of all patients did not achieve a hematologic response to epoetin. Thus, nonresponding patients may account for much of the transfusion use in the epoetin arms of these trials. To achieve the most efficient use of epoetin, more systematic evidence is needed on patient characteristics that predict responsiveness and on early indicators of response. Anemia primarily a result of malignancy included patients with multiple myeloma, non-Hodgkin's lymphoma, chronic lymphocytic leukemia, and myelodysplastic syndromes. Epoetin increases Hb levels and achieves statistically significant hematologic response rates in these patients. The evidence on transfusion outcomes is sparse but suggests a favorable effect of epoetin. Hematologic response rates appear to be lower for patients with myelodysplastic syndrome; higher doses of epoetin may be necessary to achieve response. For patients undergoing allogeneic stem-cell transplantation, epoetin decreased time to RBC engraftment by 1 to 2 weeks and may decrease the number of RBC units transfused. No reduction in length of hospitalization was reported. The evidence does not support a beneficial effect of epoetin for patients undergoing autologous stem-cell transplantation.

Authors' recommendations: For patients undergoing cancer therapy, evidence demonstrates that epoetin reduces transfusion if treatment is initiated when declining Hb levels near 10 g/dL. Randomized controlled trials, adequately powered, are needed to determine whether initiating treatment at higher baseline Hb levels yields additional benefits in reducing transfusion use or improving quality of life.

Authors' methods: Systematic review

Project Status: Completed

URL for project: http://www.ahrq.gov/clinic/epcsums/epoetsum.htm

Year Published: 2001

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: United States

Organisation Name: Agency for Healthcare Research and Quality

Contact Address: Center for Outcomes and Evidence Technology Assessment Program, 540 Gaither Road, Rockville, MD 20850, USA. Tel: +1 301 427 1610; Fax: +1 301 427 1639;

Contact Name: martin.erlichman@ahrq.hhs.gov

Contact Email: martin.erlichman@ahrq.hhs.gov

Copyright: Agency for Healthcare Research and Quality (AHRQ)