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BACKGROUND Thrombopoietin receptor agonists are emerging as a therapeutic option for patients with aplastic anemia (AA) and myelodysplastic syndrome (MDS). We report our experience of treating children with AA/MDS with romiplostim, thrombopoietin receptor agonist. OBSERVATIONS Three children (AA, 2; MDS, 1) received romiplostim treatment at a median dose of 10 μg/kg/week (starting dose 5 μg/kg/wk; 2.5 μg/kg/wk increment). Trilineage hematopoietic recovery occurred at a median of 13 weeks (range 13 to 16 wk) without adverse events. Hemato