High-dose (40,000 IU twice/week) alpha recombinant human erythropoietin as single agent in low/intermediate risk myelodysplastic syndromes: A retrospective investigation on 133 patients treated in a single institution (pages 762–767)

摘要

We investigated the efficacy of alpha recombinant human erythropoietin (a-rHuEPO) administered as singlenagent to 133 patients affected by myelodysplastic syndromes referring to our Institution in the last 10 years.nWPSS score was ‘‘very low’’ in 67%, ‘‘low’’ in 19%, ‘‘intermediate’’ in 14%. The starting schedule was:n40,000 IU bi-weekly, with reduction or suspension, when necessary, in responsive patients. According tonnew IWG criteria, response rate (RR) was 75%, 66%, 59% after 8, 16, 24 weeks, respectively. Comparingn‘‘very low’’ and ‘‘low/intermediate’’ risk, RR was 81% vs. 43% (P < 0.001); 70% vs. 45% (P 5 0.040); 63% vs.n42% (P 5 NS) after 8, 16, 24 weeks. RR was significantly influenced by transfusion dependence (P 5 0.039)nand basal serum EPO level (P < 0.001). Mean Hb value was 94 ± 11 g/l before therapy; 114 ± 19 after 8nweeks (P < 0.001); 116 ± 18 after 16 weeks (P < 0.001); 114 ± 17 after 24 weeks (P < 0.001). Reduction ornsuspension of therapy significantly affected Hb level after 4 (P < 0.001) and 8 weeks (P < 0.01). Conversely,nrestart of full dosage significantly enhanced again Hb level after 4 (P < 0.01) and 8 weeks (P < 0.001). 65%npatients are alive (mean survival: 74 weeks). Seventy percent are alive in the ‘‘very low risk’’ group and 38%nin ‘‘low/intermediate risk’’ group (P < 0.001). Overall mean follow-up was 69 weeks (range, 8–376): it was 80nweeks in responsive patients (max 376) and 38 weeks in patients who progressively became unresponsiven(max 168) (P < 0.01). Median response was 36 weeks, with 33% of patients still responding after one year.nTreatment was well tolerated. Am. J. Hematol. 86:762–767, 2011. VVC 2011 Wiley-Liss, Inc.