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.

Abstract

We investigated the efficacy of alpha recombinant human erythropoietin (α-rHuEPO) administered as single agent to 133 patients affected by myelodysplastic syndromes referring to our Institution in the last 10 years. WPSS score was "very low" in 67%, "low" in 19%, "intermediate" in 14%. The starting schedule was: 40,000 IU bi-weekly, with reduction or suspension, when necessary, in responsive patients. According to new IWG criteria, response rate (RR) was 75%, 66%, 59% after 8, 16, 24 weeks, respectively. Comparing "very low" and "low/intermediate" risk, RR was 81% vs. 43% (P < 0.001); 70% vs. 45% (P = 0.040); 63% vs. 42% (P = NS) after 8, 16, 24 weeks. RR was significantly influenced by transfusion dependence (P = 0.039) and basal serum EPO level (P < 0.001). Mean Hb value was 94 ± 11 g/l before therapy; 114 ± 19 after 8 weeks (P < 0.001); 116 ± 18 after 16 weeks (P < 0.001); 114 ± 17 after 24 weeks (P < 0.001). Reduction or suspension of therapy significantly affected Hb level after 4 (P < 0.001) and 8 weeks (P < 0.01). Conversely, restart of full dosage significantly enhanced again Hb level after 4 (P < 0.01) and 8 weeks (P < 0.001). 65% patients are alive (mean survival: 74 weeks). Seventy percent are alive in the "very low risk" group and 38% in "low/intermediate risk" group (P < 0.001). Overall mean follow-up was 69 weeks (range, 8-376): it was 80 weeks in responsive patients (max 376) and 38 weeks in patients who progressively became unresponsive (max 168) (P < 0.01). Median response was 36 weeks, with 33% of patients still responding after one year. Treatment was well tolerated.

• Authors: Antonio Azzarà, Giovanni Carulli, Sara Galimberti, Claudia Baratè, Rita Fazzi, Giulia Cervetti, Mario Petrini
• Journal: American journal of hematology (Am J Hematol) Vol. 86 Issue 9 Pg. 762-7 (Sep 2011) ISSN: 1096-8652 [Electronic] United States
• PMID: 21850658 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: Copyright © 2011 Wiley-Liss, Inc.

Chemical References

• EPO protein, human
• Hemoglobins
• Recombinant Proteins
• Erythropoietin

Topics

• Aged
• Aged, 80 and over
• Anemia (etiology, prevention & control, therapy)
• Blood Transfusion
• Cohort Studies
• Drug Monitoring
• Drug Resistance
• Erythropoietin (administration & dosage, adverse effects, blood, therapeutic use)
• Female
• Hemoglobins (analysis)
• Humans
• Male
• Middle Aged
• Myelodysplastic Syndromes (blood, diagnosis, drug therapy, physiopathology)
• Prognosis
• Recombinant Proteins
• Retrospective Studies
• Survival Analysis
• Time Factors