Phase II study of gemcitabine in children with solid tumors of mesenchymal and embryonic origin.

摘要: The therapeutic benefit and side-effect profile of gemcitabine in adults with relapsed solid tumors is well known. So far, few data are available about its significance pediatric tumors. To determine the efficacy tolerability children, drug was administered by intravenous short-term infusion over 30 min at a dose 1200 mg/m2 weekly for 3 weeks as one cycle children tumor embryonic or mesenchymal origin. From May 2003 to September 2004, 14 male six female patients (2-23, median 15.8 years) were recruited this prospective open-label phase II study (two-step Simon design). suffered from rhabdomyosarcoma (n=8), Ewing's sarcoma (n=4), osteosarcoma (n=2), neuroblastoma (n=3), hepatoblastoma (n=2) nephroblastoma (n=1). Median duration therapy 27.5 days (7-99), corresponding 4.0 (2-11) infusions gemcitabine. Two (neuroblastoma Ewing) had stable disease documented 69 70 days, whereas no objective responses observed. In 34/94 infusions; doses be reduced omitted grade 3-4 hematotoxicity. Minimal activity observed cohort wide spectrum Given relatively low administered, does not exclude possibility higher doses. Secondly, consistent that expected adults. For further studies population, we recommend use combination other agents.