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  • Publication # 13-RA012

Agent to combat anemia in patients with bone marrow neoplasms is not used according to clinical guidelines

Chronic Disease

Myelodysplastic syndromes (MDS) are a group of bone marrow neoplasms that commonly progress to acute myeloid leukemia. Approximately 80 percent of patients with MDS experience symptomatic anemia and approximately 60 percent of these patients receive erythropoiesis-stimulating agents (ESAs). ESAs mimic the effect of the naturally occurring hormone erythropoietin to stimulate red blood cell production. ESAs are key elements of a strategy to improve anemia and reduce dependence on red blood cell transfusions. Published clinical guidelines that suggest approaches to patient targeting and management are designed to optimize drug safety and effectiveness. Yet a study of 6,588 Medicare patients diagnosed with MDS over a 5-year period found clinically important discrepancies between observed practice- and guideline-recommended therapy.

In elaborating on their findings, AHRQ researcher, Amy J. Davidoff, Ph.D., and colleagues stated that patients were frequently not targeted for therapy based on risk status, as evidenced by high rates of use across all risk groups, or on the likelihood of achieving response, as evidenced by frequent lack of measurement of serum erythropoietin levels prior to ESA use. Patients with low serum erythropoietin levels are much more likely to respond to ESAs, yet only 45 percent of patients had their levels measured prior to ESA initiation. Also, duration of therapy in 60 percent of episodes was not of sufficient length to produce treatment responses. Over one-fourth of users never received a therapeutic-length episode of ESA. These results suggest that stricter adherence to guidelines would decrease inappropriate use of ESAs, presumably substantially decreasing costs associated with this therapy.

See "Patterns of erythropoiesis-stimulating agent use among Medicare beneficiaries with myelodysplastic syndromes and consistency with clinical guidelines," by Dr. Davidoff, Sheila R. Weiss, Ph.D., Maria R. Baer, M.D., and others in Leukemia Research 37, pp. 675-680, 2013. Reprints (AHRQ Publication No. 13-R049) are available from AHRQ.


Page last reviewed September 2013
Internet Citation: Agent to combat anemia in patients with bone marrow neoplasms is not used according to clinical guidelines: Chronic Disease. September 2013. Agency for Healthcare Research and Quality, Rockville, MD.


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