High-dose carboplatin, thiotepa, and etoposide with autologous stem-cell rescue for patients with recurrent medulloblastoma. Children's Cancer Group.

TLDR

Recurrent medulloblastoma is highly lethal, with few patients surviving conventional therapy. This study assessed whether high‑dose carboplatin, thiotepa, and etoposide followed by autologous stem‑cell rescue could improve outcomes in recurrent medulloblastoma. Twenty‑three patients (age 2–44, median 13) received carboplatin 500 mg/m² on days –8 to –6, thiotepa 300 mg/m² and etoposide 250 mg/m² on days –5 to –3, then ASCR on day 0, with some also undergoing surgery, conventional chemotherapy, or radiation. Three patients died within 21 days of ASCR, but 7 of 23 (30%) remained event‑free at a median of 54 months, with 36‑month event‑free and overall survival estimates of 34 % ± 10 % and 46 % ± 11 %, indicating potential long‑term benefit.

Abstract

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.Chemotherapy consisted of carboplatin 500 mg/m2 (or area under the curve = 7 mg/mL x 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.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 78 months). Kaplan-Meier estimates of event-free (EFS) and overall survival are 34% +/- 10% and 46% +/- 11%, respectively, at 36 months post-ASCR.This strategy may provide long-term survival for some patients with recurrent medulloblastoma.