title: 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.affiliation: Division of Haematology, Department of Oncology, Transplants and Advanced Technologies in Medicine, University of Pisa-AOUP, Pisa, Italy. antonio.azzara@med.unipi.it
authors.forename: Antonio
authors.initials: A
authors.lastname: Azzarà
authors.forename: Giovanni
authors.initials: G
authors.lastname: Carulli
authors.forename: Sara
authors.initials: S
authors.lastname: Galimberti
authors.forename: Claudia
authors.initials: C
authors.lastname: Baratè
authors.forename: Rita
authors.initials: R
authors.lastname: Fazzi
authors.forename: Giulia
authors.initials: G
authors.lastname: Cervetti
authors.forename: Mario
authors.initials: M
authors.lastname: Petrini
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D000740:Anemia / Q000209:etiology / Q000517:prevention & control / Q000628:therapy; D001803:Blood Transfusion; D015331:Cohort Studies; D016903:Drug Monitoring; D004351:Drug Resistance; D004921:Erythropoietin / Q000008:administration & dosage* / Q000009:adverse effects / Q000097:blood / Q000627:therapeutic use*; D005260:Female; D006454:Hemoglobins / Q000032:analysis; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000097:blood / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D011379:Prognosis; D011994:Recombinant Proteins; D012189:Retrospective Studies; D016019:Survival Analysis; D013997:Time Factors
chemical_list: C554270:EPO protein, human; D006454:Hemoglobins; D011994:Recombinant Proteins; D004921:Erythropoietin