Fax +41 61 306 12 34 E-Mail karger@karger.ch www.karger.com Brief Communication Acta Haematol 2008;120:104–107 DOI: 10.1159/000166864 Twice-Weekly High-Dose rHuEpo for the Treatment of Anemia in Patients with Low-Risk Myelodysplastic Syndromes R. Latagliata a E.N. Oliva b P. Volpicelli a I. Carmosino a M. Breccia a I. Vincelli b C. Alati b L. Napoleone a F. Vozella a F. Nobile b G. Alimena a a Department of Cellular Biotechnology and Hematology, University La Sapienza, Rome, and b Department of Hematology, Bianchi-Melacrino-Morelli Hospital, Reggio Calabria, Italy sponse rates exceeding 30–35% with standard rHuEpo doses (10,000 IU 3 times weekly or 40,000 IU once a week). These findings were confirmed by Ross et al. [10] in a meta-analysis considering 150 studies. The addition of granulocyte colony-stimulating factor to standard rHuEpo doses has slightly increased the rate of erythroid response, but this association is expensive and no positive effect on quality of life has been estab- lished in a recent randomized study [11]. In order to im- prove these clinical results, an Italian cooperative group explored the efficacy of higher rHuEpo doses and showed a response rate of 150%, with improvement in quality of life in responders. However, no correlation between re- sponse to rHuEpo and biological characteristics could be found due to the lack of data regarding cytogenetics and serum Epo levels [12]. We performed this prospective non-randomized phase II study to confirm the efficacy of high-dose rHuEpo and to evaluate the presence of clinical and biological charac- teristics affecting quality of life and treatment outcome. Previously untreated MDS patients from two hemato- logical departments were considered eligible for this study in the presence of the following characteristics: low-risk de novo MDS, defined as low and Int-1 risk group according to the IPSS prognostic score [3] or mar- row blasts !10% if a karyotypic analysis failed or was not Myelodysplastic syndromes (MDS) are a heteroge- neous group of hemopoietic disorders with peripheral cytopenia due to ineffective hemopoiesis and a variable rate of transformation to overt acute myelogenous leuke- mia [1, 2]. However, patients with low-risk MDS (marrow blasts !10% or low and intermediate-1, Int-1, risk groups according to the International Prognostic Scoring Sys- tem, IPSS) have a relatively longer survival with a lower rate of leukemic transformation [3]; anemia is the main cause of symptoms referred by low-risk MDS patients, with a frequent functional impairment that leads to di- minished quality of life and survival [4, 5]. As a matter of fact, anemia occurs in up to 90% of patients at some point during the course of their disease, with 180% of patients requiring red blood cell (RBC) transfusion; moreover, transfusion requirement has recently been reported as a bad prognostic factor affecting overall survival in each WHO entity and it has been incorporated in a new WHO- based scoring system [6]. To manage anemia in these patients, human recombi- nant erythropoietin (rHuEpo) seemed to be a good op- tion and has been tested for 120 years. In a previous dou- ble-blind controlled study, rHuEpo was shown to be sig- nificantly superior to placebo in low-risk MDS patients, with an overall response rate of 36.8% [7]. However, this and many other trials [7–9] were unable to achieve re- Received: April 29, 2008 Accepted after revision: August 5, 2008 Published online: October 31, 2008 Roberto Latagliata, MD Department of Cellular Biotechnology and Hematology University La Sapienza Via Benevento 6 IT–00161 Rome (Italy) Tel. +39 06 857 951, Fax +39 06 4424 1984, E-Mail rob.lati@libero.it © 2008 S. Karger AG, Basel 0001–5792/08/1202–0104$24.50/0 Accessible online at: www.karger.com/aha