title: The use of r-HuEpo in the treatment of anaemia related to myelodysplasia (MDS).
abstract: One hundred and sixteen (116) anaemic patients with myelodysplastic syndromes (MDS) were treated with recombinant human erythropoietin (r-HuEpo) in an open-label, multicentre, compassionate treatment trial; 100 patients received therapy for > or = 4 weeks and were evaluable for efficacy. The distribution of FAB subtypes was: 44 RA, 40 RARS, eight RAEB, two RAEB-t, one CMML, and five not specified. Mean baseline haematocrit was 24.5%, and the mean prestudy transfusion requirement in the 12 weeks immediately prior to study entry was 6.5 units. r-HuEpo treatment was initiated at a dose of 150 U/kg three times weekly, with dose escalations of 50 U/kg monthly (up to 300 U/kg 3x/week) permitted if the haematocrit failed to rise. Response to therapy was defined as either an increase in haematocrit of > or = 6 percentage points over baseline, unrelated to transfusion, or a > or = 50% decrease in transfusion requirement in the last 3 months of study treatment, compared to the baseline period (12 weeks). By these criteria, 28% (28/100) of patients responded to r-HuEpo treatment. Overall, 86% (24/28) of patients responding to therapy had baseline Epo levels < or = 100 mU/ml. Response rates by FAB subtype were: RA 39% (17/44), RARS 17.5% (7/40) and RAEB 12.5% (1/8). Additionally, a 54% (15/28) response rate was seen in RA patients with baseline Epo levels < or = 100 mU/ml. Responses to therapy were durable and generally occurred at r-HuEpo doses of 150-200 U/kg t.i.w. There were no reports of thrombosis, seizures or therapy-related hypertension. The data show that patients with MDS, especially those with the RA and RARS subtypes, can benefit from treatment with r-HuEpo. Those patients with baseline Epo levels < or = 100 mU/ml were most likely to respond to therapy.
authors.affiliation: R. W. Johnson Pharmaceutical Research Institute, Raritan, New Jersey 08869-0602, USA.
authors.forename: E H
authors.initials: EH
authors.lastname: Rose
authors.forename: R I
authors.initials: RI
authors.lastname: Abels
authors.forename: R A
authors.initials: RA
authors.lastname: Nelson
authors.forename: D M
authors.initials: DM
authors.lastname: McCullough
authors.forename: L
authors.initials: L
authors.lastname: Lessin
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000740:Anemia / Q000097:blood / Q000628:therapy*; D004921:Erythropoietin / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D006400:Hematocrit; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000097:blood / Q000150:complications*; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D004921:Erythropoietin