Osorio DS, et al. - The feasibility and tolerability of tandem courses of high-dose thiotepa with autologous hematopoietic cell rescue (AHCR) were examined in patients with recurrent, refractory, or poor prognosis solid tumor malignancies. Results approved that two cycles of high-dose thiotepa with AHCR were well tolerated even in these heavily pretreated patients. Furthermore, this therapy might offer prolonged survival in patients with recurrent malignant brain tumors, particularly medulloblastoma and central nervous system germ cell tumors (CNS GCT).

Methods

• Thiotepa was administered intravenously at a dose of 200 mg/m²/day (6.67 mg/kg/day) daily for 3 days followed by AHCR.
• A second course of thiotepa was given 4 weeks later provided blood counts recovered sufficiently without evidence of tumor progression.

Results

•  58 patients received 96 courses.
• 38 (65%) patients received two courses of therapy. 
• In the outpatient setting, 27 courses (28%) were administered completely.
• 3.4% toxic mortality rate was observed.
• Five of 26 patients with medulloblastoma were alive at a median of 35 months, whereas 21 patients died at a median of 11.7 months.
• Four of five patients with central nervous system germ cell tumors (CNS GCT) were alive 68–103 months following AHCR.