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Epipodophyllotoxin VM-26 in the treatment of childhood neuroblastoma.

Thirteen children with disseminated neuroblastoma that had become refractory to conventional chemotherapy were treated with the epipodophyllotoxin VM-26. Three patients developed partial responses (greater than 50% reductions in tumors and in the proportion of tumor cells in bone marrow). Acute nonhematologic toxicity after treatment was minimal. Hematologic toxicity was observed but could not be assessed accurately since most patients had abnormal hematopoiesis due to extensive tumor involvement of bone marrow. These results demonstrate that VM-26, as a single agent, can produce measurable tumor responses in children with neuroblastoma.