Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Myelodysplastic Syndromes
For patients with clinically significant cytopenias, supportive care, including transfusions and prophylactic antibiotics, can be considered, but have not been proven to be curative; however, it is important that supportive care be utilized in these patients awaiting transplant. In addition, the use of hematopoietic growth factors can improve the hematopoietic status, but there is some concern that such treatment could accelerate conversion to AML. Steroid therapy has also been used, including glucocorticoids and androgens, with mixed results. Treatments directed toward scavenging free oxygen radicals with amifostine [39,40] or the use of differentiation-promoting retinoids, DNA methylation inhibitors (e.g., azacytidine and decitabine), and histone deacetylase inhibitors, have all shown some positive responses. Azacytidine has been FDA-approved for the treatment of MDS in adults based on randomized studies. Agents, such as lenalidomide, an analog of thalidomide, have been tested based on findings that demonstrated increased activity in the bone marrow of patients with MDS. Lenalidomide has shown most efficacy in patients with 5q- syndrome and is now FDA-approved for use in this group. Immunosuppression with antithymocyte globulin and/or cyclosporine has also been reported.[43,44]
Treatment Options Under Clinical Evaluation
The following are examples of national and/or institutional clinical trials that are currently being conducted.
- The use of a variety of inhibitors of DNA methylation and histone deacetylase inhibitors, as well as other therapies designed to induce differentiation, are being studied in both young and older adults with MDS.[45,46,47]
Current Clinical Trials
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with childhood myelodysplastic syndromes. The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.
General information about clinical trials is also available from the NCI Web site.
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