Erythropoietin and granulocyte-colony stimulating factor treatment associated with improved survival in myelodysplastic syndrome.
OBJECTIVE
To assess the effect of erythropoietin (EPO) plus granulocyte-colony stimulating factor (G-CSF) treatment on survival and leukemic transformation in myelodysplastic syndrome (MDS).


METHODS
We compared the long-term outcome of patients with MDS treated with EPO plus G-CSF (n = 121) with untreated patients (n = 237) with MDS using multivariate Cox regression with delayed entry, for the first time adjusting for all major prognostic variables (WHO classification, karyotype, cytopenias, level of transfusion-need, age, and sex).


RESULTS
The erythroid response rate to EPO plus G-CSF was 39%, and the median response duration 23 months (range, 3 to 116+). In the multivariate analysis, treatment was associated with improved overall survival (hazard ratio, 0.61; 95% CI, 0.44 to 0.83; P = .002). Interestingly, this positive association was primarily observed in patients requiring fewer than 2 units of RBCs per month. Treatment was not linked to the rate of acute myeloid leukemia in any defined subgroup, including patients with an increase of marrow blasts or an unfavorable karyotype.


CONCLUSIONS
The inherent risk of leukemic evolution in MDS makes the current investigation highly relevant, in light of the recent reports of potential negative effects of EPO treatment on outcome in patients with cancer. We conclude that treatment of anemia in MDS with EPO plus G-CSF may have a positive impact on outcome in patients with no or low transfusion need, while not affecting the risk of leukemic transformation.
Jädersten
Martin
M
Karolinska Institutet, Department of Medicine, Division of Hematology, Stockholm, Sweden. martin.jadersten@ki.se
Malcovati
Luca
L
Dybedal
Ingunn
I
Della Porta
Matteo Giovanni
MG
Invernizzi
Rosangela
R
Montgomery
Scott M
SM
Pascutto
Cristiana
C
Porwit
Anna
A
Cazzola
Mario
M
Hellström-Lindberg
Eva
E
D000368:Aged; D000740:Anemia / Q000188:drug therapy / Q000209:etiology; D001803:Blood Transfusion; D002471:Cell Transformation, Neoplastic / Q000187:drug effects*; D017322:Clinical Trials, Phase II as Topic; D004921:Erythropoietin / Q000627:therapeutic use*; D005260:Female; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000627:therapeutic use*; D006801:Humans; D053208:Kaplan-Meier Estimate; D015470:Leukemia, Myeloid, Acute / Q000453:epidemiology; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000150:complications / Q000188:drug therapy* / Q000401:mortality; D011230:Precancerous Conditions / Q000188:drug therapy*
D004921:Erythropoietin; D016178:Granulocyte-Macrophage Colony-Stimulating Factor