Previously, the most frequent "treatment" given for patients with MDS was "best supportive care," meaning red blood cell or platelet transfusions and antibiotics. "Active therapy" was given only when the disease progressed to AML or resembled AML, in terms of severe cytopenias. Now, there are an "arsenal" of therapies available. Given the diverse forms of therapy available to MDS patients, it is important to decide what the goal of therapy is: is it to improve the anemia, thrombocytopenia, or neutropenia, render the patient transfusion independent, achieve a complete remission of the disease, or cure the disease?
The clinical approach that I find optimal is to ask 2 questions up front:
(1) does the patient have significant neutropenia or thrombocytopenia (or is it imminent); and
(2) does the patient have increased bone marrow blasts, and therefore is at risk for progression to AML?
If the answer to both is no, then one is treating the anemia of MDS, and there are many options, including blood transfusions, an erythropoietic agent (erythropoietin or darbepoetin) given alone or with granulocyte colony-stimulating factor, antithymocyteglobulin and/or cyclosporin A, lenalidomide (or thalidomide, if lenalidomide is unavailable) or a hypomethylating agent (5-azacytidine or 5-aza-2-deoxycytidine). If the answer to either question is yes, then one must try to alter the natural history of the disease (ie, to improve blood production and delay the progression to AML). Thus far, 5-azacytidine and decitabine appear to improve the outcome for patients with poor risk MDS, and both can ameliorate the cytopenias of MDS and decrease the percentage blasts in the bone marrow. Intensive, AML-like chemotherapy can also accomplish these 2 goals, but studies show that many MDS patients tolerate such treatment poorly. Blood or marrow cell transplantation (and in some patients intensive chemotherapy) can cure this disease, but again many MDS patients are not suitable candidates for this aggressive approach, either. Patients with significant cytopenias, without increased bone marrow blasts, may be candidates for immunomodulatory treatments. Lenalidomide is currently being evaluated for higher risk MDS patients (especially those with a 5q– abnormality), and MDS patients should be encouraged to participate in therapeutic clinical trials.
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