High-dose carboplatin, thiotepa, and etoposide with autologous stem- cell rescue for patients with recurrent medulloblastoma

Purpose: Medulloblastoma is a highly lethal disease when it recurs. Very few patients survive with conventional treatment. This study evaluated the use of high-dose carboplatin, thiotepa, and etoposide with autologous stem- cell rescue (ASCR) in patients with recurrent medulloblastoma. Methods: Chemotherapy consisted of carboplatin 500 mg/m² (or area under the curve = 7 mg/mL · min via Calvert formula) on days -8, -7, and -6; and thiotepa 300 mg/m² and etoposide 250 mg/m² on days -5, -4, and -3; followed by ASCR on day 0. In addition to the study-prescribed therapy, 21 patients received other treatment: neurosurgical resection in seven, conventional chemotherapy in 17, and external-beam irradiation in 11 cases. Results: Twenty-three patients with recurrent medulloblastoma, aged two to 44 years (median, 13 years) at ASCR, were treated. Three patients died of treatment-related toxicities within 21 days of ASCR; multiorgan system failure in two, and Aspergillus infection with venoocclusive disease in one. Seven of 23 patients (30%) are event-free survivors at a median of 54 months post-ASCR (range, 24 to 73 months). Kaplan-Meier estimates of event-free (EFS) and overall survival are 34% ± 10% and 46% ±11%, respectively, at 36 months post-ASCR. Conclusion: This strategy may provide long-term survival for some patients with recurrent medulloblastoma.