Topotecan in Haematologic Malignancies

Topotecan is a water-soluble, semisynthetic camptothecin-derived drug which inhibits the DNA enzyme topoisomerase I, thereby leading to the induction of apoptosis. Until now, a large number of studies have tested this cytotoxic drug in solid tumours, but only few trials have been conducted on haematologic malignancies. This review gives an overview of the published studies. Topotecan has shown significant anti-tumour activity in leukaemia, lymphoma and multiple myeloma. Especially in myelodysplastic syndromes (MDS) including chronic myelomonocytic leukaemia, high response rates were detected. Topotecan exhibits moderate haematologic and non-haematologic toxicities. The combination of topotecan with other cytostatics can potentiate the efficacy of treatment and has shown promising results in MDSand leukaemia. Based on the encouraging experiences, topotecan will be further evaluated in large international trials.