Erythropoietin-stimulating agents (ESAs) appear to consistently improve clinical outcomes and quality of life (QoL) in patients with myelodysplastic syndrome (MDS)-associated anemia, according to a report published in the British Journal of Haematology.

The authors conducted a systematic literature review to evaluate the available evidence on the use of ESAs, including epoetin and darbepoetin alfa, for anemia in adult patients with lower-risk MDS who lacked the 5q deletion. The review included randomized and nonrandomized prospective studies reporting on clinical efficacy and effectiveness, patient-reported QoL, and safety.

In total, 53 articles reporting data from 35 studies were assessed. Overall, the studies demonstrated clinical benefits from ESAs. ESAs resulted in consistent improvements in erythroid response rates (ESA-naive patients, 45%-73%; previous ESA exposure, 25%-75%) and duration of response. Several studies showed improved QoL; however, the variety of methods used and inconsistencies in reporting limited comparisons across studies.

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Similar rates of progression to acute myeloid leukemia were reported across studies. For example, in a randomized controlled trial of darbepoetin alfa compared with placebo, rates were 2.2% and 2.1%, respectively, and in studies comparing epoetin alfa with placebo, rates ranged from 2.6% to 3.5% and 2.7% to 4.4%, respectively.

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Adverse events were not consistently reported. However, for darbepoetin alfa, the most frequently reported adverse events were hypertension, thromboembolism, pulmonary embolism, and stroke.

The authors concluded that ESA therapy should remain the first-line treatment for anemia in patients with lower-risk MDS. However, they noted additional studies are needed to directly compare subgroups of ESAs.


1. Park S, Greenberg P, Yucel A, et al. Clinical effectiveness and safety of erythropoietin‐stimulating agents for the treatment of low‐ and intermediate‐1−risk myelodysplastic syndrome: a systematic literature review [published online December 13, 2018]. Br J Haematol. doi: 10.1111/bjh.15707