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Other Therapies
Source: Childhood Myelodysplastic Neoplasms Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: December 5, 2024 · Captured 2026-09-09.
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Context: Treatment of Childhood MDS
In general, the primary aim for children with newly diagnosed MDS is to rule out AML-associated somatic variants, which would indicate the need to treat according to AML guidelines. Thereafter, the objective should be to provide supportive care while looking for an appropriate donor for HSCT. During this time, close monitoring for the emergence of AML is imperative.[25] Therapies used in adult MDS have not been shown to be beneficial in childhood MDS, likely owing to differences in underlying variant etiologies.
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Other therapies for MDS that have been studied and may be applicable include the following:
Agents such as lenalidomide, an analogue of thalidomide, have been tested based on findings that demonstrated increased activity in the bone marrow of patients with MDS. Lenalidomide has shown the most efficacy in patients with 5q- syndrome, especially those with thrombocytosis. The U.S. Food and Drug Administration approved lenalidomide for use in adults with this finding.[26]
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Preserved source evidence · Not medical advice