Erythropoiesis-Stimulating Agents Versus Rbc Transfusion In Mds: Comparison of Long-Term Outcomes

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.

An assessment of erythroid response to epoetin α as a single agent versus in combination with granulocyte– or granulocyte‐macrophage–colony‐stimulating factor in myelodysplastic syndromes using a meta‐analysis approach

“…Similarly, another recent meta‐analysis specifically in low‐risk to intermediate‐1–risk patients reported no adverse effect of hematopoietic growth factor treatment on the rate of either leukemic transformation or survival 33. In light of several deleterious effects of prolonged RBC transfusions in patients with MDS, as reviewed previously,34, 35 the objectives of treatment in patients who have low‐risk to intermediate‐1– risk disease are to delay transfusion dependence or avoid transfusions in those who require them regularly. On the basis of the current meta‐analysis, EPO‐based therapies appear to fulfill these therapeutic objectives.…”

Azacitidine: A Review of its Use in the Management of Myelodysplastic Syndromes

“…In low risk patients, erythropoietin used within established guidelines can provide durable responses and may even result in a small increase in survival. 5,6 G-CSF and GM-CSF have been used in neutropenic populations; however patients with MDS have a predictable poor response to these agents given their dysfunctional bone marrow environment. Antibiotics are often necessary to fend off bacterial infections, and along with RBC transfusions, are the mainstay of supportive care for many patients.…”