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# Uses of Epoetin for Anemia in Oncology

*Evidence Reports/Technology Assessments, No. 30*

Investigators: Jerome Seidenfeld, PhD, Principal Investigator, Naomi Aronson, PhD, Margaret Piper, PhD, MPH, Carole Redding Flamm, MD, MPH, Vic Hasselblad, PhD, and Kathleen M Ziegler, PharmD.

Agency for Healthcare Research and Quality (US)2001 Jun
Report No.: 01-E0091-58763-036-2

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## Structured Abstract

#### 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.

#### Search Strategy:

                The MEDLINE, CancerLit, and EMBASE databases were searched from 1985 through 1998                    and *Current Contents on Diskette* and *Medscape                        Oncology* through October 1999 for the terms: erythropoietin                    (Medical Subject Heading [MeSH®]); epoetin alfa (MeSH®);                    erythropoietin (tw); epoetin (tw); Epogen (tw); Procrit (tw); Eprex (tw);                    Marogen (tw); Recormon (tw); epo (tw); Anemia/drug therapy (MeSH®;                    included all subheadings); Anemia/therapy (MeSH®; included all                    subheadings); Anemia/diet therapy (MeSH®; included all subheadings).                    The search was then limited to "neoplasms" or "myelodysplastic syndromes" and                    studies on human subjects. The yield was 2,943 references.

#### Selection Criteria:

                This systematic review is limited to controlled trials comparing the outcomes of                    managing anemia with and without the use of epoetin in one of the four patient                    populations of interest. Uncontrolled trials were excluded.

#### Data Collection and Analysis:

                We used a prospectively designed protocol conducted by two independent reviewers,                    with disagreements resolved by consensus. The meta-analysis used a random                    effects model to combine data on odds of transfusion in patients with anemia due                    primarily to cancer therapy.

#### Main Results:

                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.

#### Conclusions:

                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.

This review identified common deficiencies in the design and reporting of trials                    on epoetin. Some methodologic deficiencies may result in overestimation of the                    effects of epoetin and inadequacy of reporting may limit the ability to                    interpret and generalize results. Future trials should maintain a higher                    standard of methodologic quality and completeness of reporting.

## Contents

                          - Expand All
                          - Collapse All

                        - Preface
                        - Summary
                        - Findings
                        - Future Research
                        - 1. Introduction                           - Scope and Objectives
                          - Prevalence and Severity of Anemia in Cancer Patients
                          - Differential Diagnosis Determines Treatment
                          - Red Blood Cell Transfusion and Epoetin in Anemia Treatment
                          - Anemia and Quality of Life in Cancer Patients
                          - Community-Based Trials of Epoetin Therapy
                          - Analyses Comparing Costs of Epoetin to Costs of Transfusion
                          - Background on Key Clinical Issues for Each Population Addressed in this Systematic Review
                        - 2. Methodology                           - Overview
                          - Search Strategy for the Identification of Articles
                          - Methods of the Review
                          - Meta-Analysis
                        - 3. Results and Conclusions I: Anemia Resulting Primarily From Cancer Therapy                           - Objective
                          - Key Questions
                          - Overview of the Evidence
                          - Results
                          - General Conclusions
                        - 4. Results and Conclusions II: Anemia Due Primarily to Malignant Disease                           - Objective
                          - Key Questions
                          - Overview of the Evidence
                          - Results
                          - Conclusions
                        - 5. Results and Conclusions III: Outcomes of Anemia Management Strategies in Patients Being Treated for Malignancy with Bone Marrow Ablation and Allogeneic Stem-Cell Support                           - Objective
                          - Key Questions
                          - Overview of the Evidence
                          - Study Quality
                          - Results
                          - Conclusions
                        - 6. Results and Conclusions IV: Outcomes of Anemia Management Strategies in Patients Being Treated for Malignancy with Bone Marrow Ablation and Autologous Stem-Cell Support                           - Objective
                          - Key Questions
                          - Overview of the Evidence
                          - Results
                          - Conclusions
                        - 7. Future Research Priorities
                        - Evidence Tables
                        - Appendixes                           - Appendix A. Technical Advisory Group External Reviewers Medical Advisory Panel
                          - Appendix B. Study Sponsorship
                          - Appendix C. Data Abstraction Form
                          - Appendix D. Acronyms
                        - References
                        - Bibliography

                          - Expand All
                          - Collapse All

Prepared for: Agency for Healthcare Research and Quality, U.S. Department                of Health and Human Services.1                 Contract No. 290-97-0015.                Prepared by: Blue Cross and Blue                    Shield Association, Technology Evaluation Center.

#### Suggested citation:

Seidenfeld J, Aronson N, Piper M, et al. Uses of                    Epoetin for Anemia in Oncology. Evidence Report/Technology Assessment No. 30.                    (Prepared by Blue Cross and Blue Shield Association Technology Evaluation Center                    under Contract No. 290-97-0015.) AHRQ Publication No. 01-E009. Rockville, MD:                    Agency for Healthcare Research and Quality. June 2001.

On December 6, 1999, under Public Law 106-129, the Agency for Health Care Policy and                Research (AHCPR) was reauthorized and renamed the Agency for Healthcare Research and                Quality (AHRQ). The law authorizes AHRQ to continue its research on the cost,                quality, and outcomes of health care and expands its role to improve patient safety                and address medical errors.

This report may be used, in whole or in part, as the basis for development of                clinical practice guidelines and other quality enhancement tools, or a basis for                reimbursement and coverage policies. AHRQ or U.S. Department of Health and Human                Services endorsement of such derivative products may not be stated or implied.

The authors of this report are responsible for its content. Statements in the report                should not be construed as endorsement by the Agency for Healthcare Research and                Quality or the U.S. Department of Health and Human Services of a particular drug,                device, test, treatment, or other clinical service.

                        - 1
                        - 
2101 East Jefferson Street, Rockville, MD 20852. www​.ahrq.gov

Copyright Notice

Bookshelf ID: NBK33589

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                    - Cite this Page Seidenfeld J, Aronson N, Piper M, et al. Uses of Epoetin for Anemia in Oncology. Rockville (MD): Agency for Healthcare Research and Quality (US); 2001 Jun. (Evidence Reports/Technology Assessments, No. 30.)

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