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Blood, 1 August 2005, Vol. 106, No. 3, pp. 768-769.
Erythropoietin for the anemia of low-risk myelodysplastic syndromesHÔPITAL AVICENNE; PARIS 13 UNIVERSITY
Recombinant EPO + G-CSF is an effective treatment of anemia in 40% of low-risk MDS, often with prolonged response and without increasing the risk of progression to AML.
In this issue, Jädersten and colleagues report the long term outcome of 129 MDS patients treated between 1990 and 1999 with EPO + G-CSF. The response rate was 39%. In the 41 responders (out of 48) who received maintenance treatment, median response duration was 23 months (range, 3-116+ months). Responses were more durable in patients with no excess of marrow blasts (RA and RARS), with an IPSS of low or intermediate 1 and in complete responders. Many patients required only low-maintenance doses of EPO and G-CSF (the latter could be stopped in several cases). Relapses that occurred on treatment were associated with disease progression in 30% of the cases, but remained unexplained in the other patients. Responders to EPO + G-CSF had better survival rates than nonresponders, and only 1 of the 20 patients with a response duration greater than 2 years progressed to AML. The authors also compared their 129 patients with 334 well-matched untreated MDS patients selected from the cohort that served to establish the IPSS.3 They found that the incidence of progression to more aggressive disease, especially AML, and survival were similar in the 2 patient populations (Figure 3 of Jädersten et al). Although the comparison did not result from a randomized study, these findings are reassuring. Indeed, EPO, with or without G-CSF, is largely administered worldwide to improve anemia in MDS.
The authors finally confirm that, in high- and intermediate-2–risk MDS (according to IPSS), EPO, with or without G-CSF, is of limited use. In low- and intermediate-1–risk MDS patients, on the contrary, EPO remains a strong contender for new investigational treatments of anemia (with the probable exception of MDS with the 5q–syndrome, where responses to lenalidomide appear clearly superior).4 Whether novel erythropoiesis-stimulating proteins with prolonged half-life, like darbepoietin alpha, will further improve the results obtained with human recombinant EPO, as recently suggested,5 will have to be confirmed. References
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| Copyright © 2005 by American Society of Hematology Online ISSN: 1528-0020 | |||||||||
2 RBC units/month) were favorable prognostic factors of response to EPO + G-CSF, whereas patients classified in the low or intermediate-1 groups according to the MDS International prognostic scoring system (IPSS; based on percentage of marrow blasts, number of cytopenias, and karyotype) also had better response.
