Comprehensive Treatment Strategy For

The clinical problems that MDS patients experience can be broken down into three basic categories. While anemia is the major problem for some patients, others have potentially life-threatening neutropenia or throm-bocytopenia. Patients in both categories may be at high risk for developing AML. These scenarios are discussed below.

1. Anemia only: These patients often have relatively good-risk disease, and are most suitable for low-intensity approaches or supportive care alone. rHuEPO should be tried for patients with symptomatic anemia; it works best in those with little or no transfusion requirements and relatively low serum erythropoietin levels. The doses required are higher than those used to treat chemotherapy-associated anemia. Patients with RARS, in particular, may benefit from combining G-CSF with rHuEPO, as there appears to be synergistic effects on erythropoiesis. Lenalidomide has shown significant activity in transfusion-dependent patients with a del(5q) cyto-genetic abnormality, and also in those with a normal karyotype. Thalidomide has modest stimula

Treatment strategy for patients with MDS

Treatment strategy for patients with MDS

Three clinical issues to consider if predominant problem is tory activity on red blood cell production, but its activity seems inferior to lenalidomide. 5-Azacytidine has received FDA approval for RBC-transfusion-dependent patients. Although it has greater side effects than rHuEPO, its use may forestall the development of iron overload and thus may benefit certain high-risk patient subsets. Heavily transfused patients are appropriate candidates for investiga-tional therapies and in rare instances, even stem cell transplantation. The timing for such treatment should be determined in consultation with a transplant physician.

2. Neutropenia/thrombocytopenia: Patients with mild to moderate, uncomplicated neutropenia and/or thrombocytopenia can often be observed; however, such patients should be evaluated on an individual basis. Patients with life-threatening neutropenia or throm-bocytopenia warrant active treatment and not simply supportive care (such as platelet transfusions and antibiotics), even though some such patients may be assigned to a "low-risk" IPSS subcategory. 5-Azacytidine can induce trilineage responses and along with decitabine (an investigational agent in phase III testing) can induce rapid and impressive improvements in peripheral blood counts. Patients with severe cytopenias (even without an increase in bone marrow (BM) blasts) should be considered for allo-geneic stem cell transplantation before they have life-threatening infectious or bleeding complications. Whether patients with cytopenias and no increase in BM blasts will have superior outcomes with reduced intensity stem cell transplantation is not known, but hopefully will be addressed in future clinical trials. Patients with severe cytopenias who are not eligible for a stem cell transplant should receive a series of

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