Leukemia Research 35 (2011) 1472–1476 Contents lists available at ScienceDirect Leukemia Research jou rnal h omepa g e: www.elsevier.com/locate/leukres Weekly standard doses of rh-EPO are highly effective for the treatment of anemic patients with low-intermediate 1 risk myelodysplastic syndromes Enrico Balleari a,b,* , Marino Clavio a,b , Eleonora Arboscello a,b , Andrea Bellodi a,b , Andrea Bruzzone a,b , Lisette Del Corso a,b , Maria Vita Lucchetti a,b , Maurizio Miglino a,b , Caterina Passalia a,b , Ivana Pierri a,b , Irene Ponassi a,b , Caterina Oneto a,b , Omar Racchi a,b , Marco Scudeletti c , Luana Vignolo a,b , Gabriele Zoppoli a,b , Marco Gobbi a,b , Riccardo Ghio a,b a Department of Hematology and Oncology, Azienda Ospedaliera Universitaria San Martino di Genova, Genova, Italy b Department of Internal Medicine, University of Genova, Genoa, Italy c ASL 4, Sestri Levante, Genova, Italy a r t i c l e i n f o Article history: Received 26 February 2011 Received in revised form 18 May 2011 Accepted 20 May 2011 Available online 26 July 2011 Keywords: Low-risk myelodysplastic syndromes Erythropoietin Erythropoietic stimulating agents Anemia IPSS WPSS a b s t r a c t For more than 20 years erythropoietin (rHEPO) has largely been used to treat anemia in myelodysplastic syndromes (MDS). Early clinical trials showed erythroid responses in no more than 15–25% of patients. In the last decade, a better selection of MDS patients suitable for a therapeutic challenge with rHEPO, alone or in combination with G-CSF, allowed for an increased response-rate, averaging around 40%. More recently, an even higher percentage of responses have been obtained using higher-doses of rHEPO (up to 80,000 IU/weekly) in lower-risk MDS patients. This treatment however, especially at such high doses, is costly and not easily affordable for prolonged periods. The aim of this study was to verify if the use of “standard” doses of rHEPO could induce a satisfying response-rate with a less expensive treatment schedule in IPSS-defined “lower-risk” MDS anemic patients. From January 2005 to December 2009 a total of 55 consecutive anemic (Hb ≤ 10 g/dL) patients (29 males, 26 females, median age 78 years) with low-intermediate-1 risk MDS were treated after informed consent with rHEPO (40,000 IU once a week subcutaneously) for at least 3 months; at the end of this period, erythroid response was assessed, and responders were allowed to continue the treatment indefinitely, whereas non-responders were consid- ered “off study”. Both efficacy and safety of the treatment were recorded and evaluated. After 3 months of treatment, 36 out of 55 (65.5%) patients achieved an erythroid response to rHEPO according to IWG 2006 criteria. Higher response-rates to rHEPO were related with both lower IPSS and particularly WPSS scores. Treatment was safe, and only 1 patient had to discontinue the treatment because of unmanage- able side-effects. Among the 36 responders, 28 (77%) maintained the response after a median follow-up of 46 months. Our data indicate that standard doses of rHEPO are at least as effective as higher-doses for correcting anemia in lower-risk MDS patients; in this clinical scenario, this schedule allows for a consistent reduction of costs without precluding the achievement of a durable erythroid response. © 2011 Elsevier Ltd. All rights reserved. 1. Introduction On the basis of the International Prognostic Scoring System (IPSS) [1], patients affected by myelodysplastic syndromes (MDS) are usually at present grouped, for prognostic and therapeutic purposes, into two groups: lower-risk MDS (including low and intermediate-1 risk IPPS groups, who have a lower rate of leukemic * Corresponding author at: Department of Hematology and Oncology, University of Genoa, Viale Benedetto XV, n 6, 16132 Genova, Italy. Tel.: +39 010 5555785; fax: +39 010 5556788. E-mail address: balleari@unige.it (E. Balleari). transformation and a relatively longer survival) and higher-risk MDS (including intermediate-2 and high-risk IPPS groups, with high risk of progression to leukemia and short survival). Anemia is the main clinical problem for lower-risk MDS patients, because it is present at diagnosis in the large majority of them, eventually requiring chronic transfusion in up to 80% of them, with an often severe functional impairment that leads to a poor quality of life [2]. Recombinant human erythropoietin (rHEPO) has been largely used in MDS patients with anemia since the early nineties of the last century, with response rates usually inferior to 25% in older studies (reviewed in [3]). In the last decade, a discrete increase in the response-rate to rHEPO was observed with standard rHEPO 0145-2126/$ – see front matter © 2011 Elsevier Ltd. All rights reserved. doi:10.1016/j.leukres.2011.05.025