A HIGH-DOSE BUSULFAN-THIOTEPA COMBINATION FOLLOWED BY AUTOLOGOUS BONE-MARROW TRANSPLANTATION IN CHILDHOOD RECURRENT EPENDYMOMA - A PHASE-IISTUDY

Sixteen children with refractory or relapsed ependymoma were entered i n a phase-II study of high-dose chemotherapy followed by autologous bo ne marrow transplantation (ABMT). The conditioning regimen consisted o f busulfan 150 mg/m(2)/day for 4 days and thiotepa 300 mg/m(2)/day for the 3 following days. All patients had previously been treated by sur gery and conventional chemotherapy. Eight of them had also received ir radiation at doses ranging from 45 to 55 Gy at the tumor site. At the time of transplantation, 9 patients were in first relapse, 5 in second relapse and 2 in third relapse or more; all had measurable disease; 1 5 patients were evaluable for response. No radiologic response >50% wa s observed. Stable disease and progressive disease were documented in 10 and 5 cases, respectively. The duration of response to this treatme nt, which lasted for a median time of 7 months (range: 5-8 months), wa s only evaluable in 5 patients who did not receive further treatment a fter ABMT. To date, there are 3 disease-free survivors at 15, 25 and 2 7 months all of whom were treated with second complete surgical resect ion and local radiotherapy (55 Gy). Toxicity was severe, mainly digest ive and cutaneous, and 1 toxicity-related death occurred. Unlike medul loblastomas, ependymomas do not appear to be sensitive to this combina tion therapy. New therapeutic approaches are warranted.