Summary.Four patients with refractory anaemia with excess blasts in transformation (RAEB‐t) and seven patients with acute leukaemia (ALL transformed from myelodysplastic syndromes (MDS) were treated with etoposide (50 mg, 2 h infusion, two to seven times per week) for at least 4 weeks. Of 10 assessable patients, three RAEB‐t patients achieved partial response and one AL patient achieved complete remission. Three of the four responders were resistant to prior repeated low‐dose cytarabine therapy. The responders did not require transfusions for 2–9 months while continuing on etoposide therapy. The side‐effects were mild and well tolerated. Three possible mechanisms, i.e. a cytotoxic effect, differentiation‐induction of malignant cells, and prolongation of blood cell survival by destroying the reticuloendotheliai system, may explain the effects of etoposide. We conclude that low‐dose etoposide is a potential therapy for MDS and atypi
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