Article (Scientific journals)
Treatment of anemia in myelodysplastic syndromes with granulocyte colony-stimulating factor plus erythropoietin: results from a randomized phase II study and long-term follow-up of 71 patients.
Hellstrom-Lindberg, E.; Ahlgren, T.; Beguin, Yves et al.
1998In Blood, 92 (1), p. 68-75
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Keywords :
Aged; Aged, 80 and over; Anemia/drug therapy/physiopathology; Drug Therapy, Combination; Erythropoietin/administration & dosage; Female; Follow-Up Studies; Granulocyte Colony-Stimulating Factor/administration & dosage; Humans; Male; Middle Aged; Myelodysplastic Syndromes/physiopathology; Treatment Outcome
Abstract :
[en] Treatment with erythropoietin (epo) may improve the anemia of myelodysplastic syndromes (MDS) in approximately 20% of patients. Previous studies have suggested that treatment with the combination of granulocyte colony-stimulating factor (G-CSF) and epo may increase this response rate. In the present phase II study, patients with MDS and anemia were randomized to treatment with G-CSF + epo according to one of two alternatives; arm A starting with G-CSF for 4 weeks followed by the combination for 12 weeks, and arm B starting with epo for 8 weeks followed by the combination for 10 weeks. Fifty evaluable patients (10 refractory anemia [RA], 13 refractory anemia with ring sideroblasts [RARS], and 27 refractory anemia with excess blasts [RAEB]) were included in the study, three were evaluable only for epo as monotherapy and 47 for the combined treatment. The overall response rate to G-CSF + epo was 38%, which is identical to that in our previous study. The response rates for patients with RA, RARS, and RAEB were 20%, 46%, and 37%, respectively. Response rates were identical in the two treatment groups indicating that an initial treatment with G-CSF was not neccessary for a response to the combination. Nine patients in arm B showed a response to the combined treatment, but only three of these responded to epo alone. This suggests a synergistic effect in vivo by G-CSF + epo. A long-term follow-up was made on 71 evaluable patients from both the present and the preceding Scandinavian study on G-CSF + epo. Median survival was 26 months, and the overall risk of leukemic transformation during a median follow-up of 43 months was 28%. Twenty patients entered long-term maintenance treatment and showed a median duration of response of 24 months.The international prognostic scoring system (IPSS) was effective to predict survival, leukemic transformation, and to a lesser extent, duration of response, but had no impact on primary response rates.
Disciplines :
Hematology
Author, co-author :
Hellstrom-Lindberg, E.
Ahlgren, T.
Beguin, Yves  ;  Centre Hospitalier Universitaire de Liège - CHU > Hématologie clinique
Carlsson, M.
Carneskog, J.
Dahl, I. M.
Dybedal, I.
Grimfors, G.
Kanter-Lewensohn, L.
Linder, O.
Luthman, M.
Lofvenberg, E.
Nilsson-Ehle, H.
Samuelsson, J.
Tangen, J. M.
Winqvist, I.
Oberg, G.
Osterborg, A.
Ost, A.
More authors (9 more) Less
Language :
English
Title :
Treatment of anemia in myelodysplastic syndromes with granulocyte colony-stimulating factor plus erythropoietin: results from a randomized phase II study and long-term follow-up of 71 patients.
Publication date :
1998
Journal title :
Blood
ISSN :
0006-4971
eISSN :
1528-0020
Publisher :
American Society of Hematology, Washington, United States - District of Columbia
Volume :
92
Issue :
1
Pages :
68-75
Peer reviewed :
Peer Reviewed verified by ORBi
Available on ORBi :
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