Erythropoiesis-stimulating agents versus RBC transfusion in MDS: comparison of long-term outcomes.

OrthoBiotech Clinical Affairs LLC, Bridgewater, NJ, USA.
Future Oncology (Impact Factor: 2.61). 09/2007; 3(4):397-403. DOI: 10.2217/14796694.3.4.397
Source: PubMed

ABSTRACT Impaired erythropoiesis and refractory anemia are clinical hallmarks of the myelodysplastic syndromes (MDS). As the disease evolves, a steady decline in hemoglobin in these disorders invariably results in dependence on packed red blood cell (PRBC) transfusion. Such chronic transfusion dependence has been associated with iron overload causing cardio-hepatic toxicity and alloimmunization, and can result in reduced survival in these patients. The use of hematopoietic growth factors, particularly erythropoiesis-stimulating agents (ESAs), has been reported to reduce the need for PRBC transfusion, raise hemoglobin and improve quality of life, at least in patients responding to such a therapy. Importantly, the clinical benefits of ESA are well balanced, with an apparently favorable safety profile in MDS, thus providing an eminent therapeutic option to delay or avoid transfusion dependence in these patients. The present report provides a detailed comparative profile of long-term PRBC transfusions and the balance of clinical benefits versus risks associated with ESA therapy for MDS.

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    ABSTRACT: Epoetin alpha (EPO) continues to be the initial treatment of choice for most anemic patients with myelodysplastic syndromes (MDS). Over the years, different therapeutic strategies have been adopted to optimize the clinical benefits of EPO in this setting. In the current meta-analysis of published literature, erythroid response (ER) rates with EPO as a single agent versus its combination with granulocyte-colony-stimulating factor (G-CSF) or granulocyte-macrophage-colony-stimulating factor (GM-CSF) were compared. The assessment indicated that the ER rates were comparable between the 2 EPO-based therapeutic strategies. Furthermore, EPO monotherapy at a higher dose of 60,000 to 80,000 U weekly produced significantly higher ER rates (64.5%) compared with the standard oncology dose of 30,000 to 40,000 U weekly either as a single agent (49%; P < .001) or in combination with G-CSF/GM-CSF (50.6% P = .007). In addition, when transfusion-dependent patients were assessed separately, both EPO monotherapy and its combination with G-CSF/GM-CSF produced comparable and appreciable levels of transfusion independence (28.8% and 24.8%, respectively). In the current meta-analysis, higher doses of EPO demonstrated better ER rates compared with EPO at standard doses alone or in combination with G-CSF/GM-CSF. Furthermore, the authors concluded that prospective clinical studies are warranted to evaluate the use of higher doses of EPO in anemic patients with MDS.
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