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Blood, 1 August 2005, Vol. 106, No. 3, pp. 803-811.
Prepublished online as a Blood First Edition Paper on April 19, 2005; DOI 10.1182/blood-2004-10-3872.
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Submitted October 12, 2004
Accepted March 11, 2005
Long-term outcome of treatment of anemia in MDS with erythropoietin and G-CSF
Martin Jadersten, Scott M Montgomery, Ingunn Dybedal, Anna Porwit-MacDonald, and Eva Hellstrom-Lindberg*
Karolinska Institutet, Department of Medicine, Division of Hematology, Karolinska University Hospital Huddinge, Stockholm, Sweden
Department of Medicine, Clinical Epidemiology Unit, Karolinska Institutet, Stockholm, Sweden; Clinical Research Centre, Orebro University Hospital, Orebro, Sweden
Department of Medicine, St. Olavs Hospital, Trondheim University Hospital, Trondheim, Norway
Department of Pathology, Karolinska University Hospital Solna, Stockholm, Sweden
* Corresponding author; email: eva.hellstrom-lindberg{at}medhs.ki.se.
We report long-term results of treatment of myelodysplastic syndrome (MDS) with erythropoietin + granulocyte colony-stimulating factor (G-CSF). One-hundred-and-twenty-nine patients were followed up 45 months after last inclusion in the Nordic studies. Erythroid response rate was 39% and median response duration 23 months (3-116+). Complete responders showed longer response duration than partial responders (29 vs. 12 months, P=0.006). The International Prognostic Scoring System (IPSS) groups Low/Int-1 had longer response duration than Int-2/High (25 vs. 7 months, P=0.002). The time until 25% developed acute myeloid leukemia (AML) was longer in the good/intermediate predictive groups for erythroid response compared to the poor predictive group (52 vs. 13 months, P=0.008). Only one of 20 long-term responders developed AML. We assessed the effect on long-term outcome by comparing treated patients with untreated selected from the IPSS database using multivariate Cox regression, adjusting for major prognostic variables. There was no difference in survival, OR 0.9 (95% CI 0.7-1.2, P=0.55), or risk of AML evolution, OR 1.3 (95% CI 0.7-2.2, P=0.40), between treated and untreated patients. Patients with high/intermediate probability of response and with IPSS Low/Int-1 show frequent and durable responses without adverse effects on outcome, while other patients should not be considered candidates for this treatment.
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