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Erythropoietin plus granulocyte colony-stimulating factor in the treatmentof myelodysplastic syndromes. Identification of a subgroup of responders
Remacha, AF; Arrizabalaga, B; Villegas, A; Manteiga, R; Calvo, T; Julia, A; Fuertes, IF; Gonzalez, FA; Font, L; Junca, J; Del Arco, A; Malcorra, JJ; Equiza, EP; De Mendiguren, BP; Romero, M;
Hosp Sant Pau, Dept Hematol, Barcelona 08025, Spain Hosp Sant Pau Barcelona Spain 08025 Dept Hematol, Barcelona 08025, Spain
Titolo Testata:
fascicolo: 12, volume: 84, anno: 1999,
pagine: 1058 - 1064
myelodysplastic syndromes; erythropoietin.; granulocyte-colony stimulating factor (G-CSF);
Tipo documento:
Settore Disciplinare:
Life Sciences
Indirizzi per estratti:
Indirizzo: Remacha, AF Hosp Sant Pau, Dept Hematol, Avda Padre Claret 167, Barcelona 08025, Spain Hosp Sant Pau Avda Padre Claret 167 Barcelona Spain 08025 ain
A.F. Remacha et al., "Erythropoietin plus granulocyte colony-stimulating factor in the treatmentof myelodysplastic syndromes. Identification of a subgroup of responders", HAEMATOLOG, 84(12), 1999, pp. 1058-1064


Background and Objectives. Anemia leading to transfusion is probably the most important problem in patients with myelodysplastic syndromes (MDS). Human recombinant erythropoietin (rHuEpo) and granulocyte colony-stimulating factor (G-CSF) have been used to treat patients with anemia of MDS, but fewer than 50% respond. The aim of this work was to evaluate the benefit of rHuEpo +/- G-CSF treatment and to isolate the response predictive variables ina group of selected patients with MDS. Design and Methods. A non-randomized multicenter trial was carried out in 32 patients with MDS. The Inclusion criteria were age greater than or equalto 18 years, refractory anemia (RA) or refractory anemia with ringed sideroblasts, Hb less than or equal to 100 g/L or receiving transfusions and serum erythropoietin less than or equal to 250 U/L. These patients were treated with subcutaneous rHuEpo (300 U/kg) three times a week for 8 weeks. In the case of partial response (PR) or no response (NR) subcutaneously administered G-CSF (1 mu g/kg) three times a week was added to the rHuEpo for 8 more weeks. If the patient achieved complete response (CR) or PR in the secondphase, he was included in a follow-up phase of 24 weeks in which the dose of growth factors was tapered down. Several variables, including the score published by the Scandinavian-American group, were used as possible predictive variables. Results. An erythroid response was observed in 16 patients (50%); in 12 itwas a CR and In 4 it was a PR. During the period of rHuEpo administration,7 OR and 4 PR (34.4%) were documented. Of the 14 patients in whom G-CSF was added to rHuEpo, 7 (50%) responded (3 CR and 4 PR). No major side-effectsassociated with growth factors were observed. The multivariate analysis showed that of the different variables evaluated only the Scandinavian-American response score was significant with a relative probability of response of 11.8 (95% confident intervals: 2.5-53) when this score was > +1 (77% of cases responded). In contrast, when this score was less than or equal to 1 only 15% of the cases responded. Interpretation and Conclusions. Use of the Scandinavian-American response score is to be recommended in a patient-oriented approach to treating MDS cases with the Epo and G-CSF. Treatment with rHuEpo and G-CSF is safe, its main drawback being its cost. However, a long-term study evaluating the regimen's cost-benefit ratio is warranted. (C) 1999, Ferrata Storti Foundation.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 31/03/20 alle ore 04:55:51