EPO and G-CSF for MDS: No Risk of Increased Progression
EPO and G-CSF for MDS: No Risk of Increased Progression
Abstract & Commentary
By Andrew S. Artz, MD, Section of Hematology/Oncology, University of Chicago. Dr. Artz reports no financial relationships with this field of study
Synopsis: MDS is a relatively common disorder managed by oncologists; but, until recently, few therapies have been available. In this observation study, patients with MDS who were treated with erythropoietin and G-CSF (EPO/G-CSF) for anemia with prolonged follow-up were assessed and then compared to another large cohort of MDS subjects. The response rate was 46% in the low-risk subjects according the IPSS score. Subjects treated with EPO/G-CSF demonstrated no increased risk of AML progression or death, providing reassurance that such treatment is unlikely to hasten disease progression.
Source: Jadersten M, et al. Long-term outcome of treatment of anemia in MDS with erythropoietin and G-CSF. Blood. 2005;106:803-811.
Myelodysplastic syndrome (MDS) represents a stem cell disorder with ineffective hematopoiesis and cytopenias. Although patients with MDS may become transfusion dependent and/or progress to AML, tremendous heterogeneity exists. The International Prognostic Scoring System (IPSS) stratifies patients into 4 risk groups: Low, Intermediate-1, Intermediate-2, and High. Although allogeneic stem cell transplant remains the only curative option, few patients are eligible for this approach. Anemia is a common problem, often treated with either red cell transfusions or growth factors (erythropoietin with or without G-CSF). While growth factors may enable reduced transfusion in a subset of patients with low serum endogenous EPO level, persistent administration holds a theoretical concern for promoting progression to AML, especially in a stem cell disorder.
Jadersten and colleagues present follow-up data combined from 3 Nordic MDS Group studies employing EPO/G-CSF for anemia. Of 141 treated patients, 129 were evaluable and median age was 72 years with 45 months of follow-up since the last enrolled patient. The EPO/G-CSF regimens varied. The authors used the study cohort (IPSS/IMRAW) of 816 untreated MDS patients from which the IPSS was derived for comparison. Response was defined as hemoglobin above 11.5 g/dL without need for transfusion.
For the 129 patients, 123 were evaluable for response. The response rate for the IPSS Low/Int-1 groups and Int-2/high was 46% and 27%, respectively. Compared to the IPSS/IMRAW cohort, no difference existed in survival or risk of AML progression for the EPO/G-CSF patients, in multi-variable models.
Commentary
This observational study determines response, progression to AML, and survival in patients with MDS treated with EPO/G-CSF. The response rate, particularly in the Low/Int-1 of 46%, is encouraging. Most importantly, the lack of a detrimental impact of these commonly employed growth factors on progression to AML or survival with long follow-up will be reassuring to oncologists. While bias may always exist in non-randomized comparisons, multivariable adjusting for important prognostic factors such as blast count, cytopenias, cytogenetics, and age, provides an excellent control group.
The findings must be placed in context however. With the availability of 5-azacytadine, promising results with new therapies such as lenalidomide,1 and increasing use of allogeneic transplantation in older patients, growth factors have an important but limited role for the subset with anemia, low-risk disease, lack of 5q-abnormality, and not immediately eligible for other therapies. However, the quality of life impact of enhanced hemoglobin and reduced use of red cell transfusions should not be minimized. The growing arsenal of therapies for MDS offers hope to patients and oncologists alike.
References
1. List A, et al. Efficacy of lenalidomide in myelodysplastic syndromes. N Engl J Med. 2005;352:549-57.
MDS is a relatively common disorder managed by oncologists; but, until recently, few therapies have been available. In this observation study, patients with MDS who were treated with erythropoietin and G-CSF (EPO/G-CSF) for anemia with prolonged follow-up were assessed and then compared to another large cohort of MDS subjects. The response rate was 46% in the low-risk subjects according the IPSS score. Subjects treated with EPO/G-CSF demonstrated no increased risk of AML progression or death, providing reassurance that such treatment is unlikely to hasten disease progression.Subscribe Now for Access
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