Retrospective analysis of patients with relapsed/refractory medulloblastoma after autologous stem cell transplantation

Abstract

Aims: Medulloblastoma is very rare and accounts for only 1% of intracranial tumors in adults. There are limited treatment options for adult medulloblastoma patients who have relapsed or refractory disease. We aimed to show real-life data on health outcomes in adult patients with recurrent or refractory medulloblastoma patients who received autologous hematopoietic stem cell transplantation (AHSCT). Methods: We analyzed the data of 15 patients who underwent AHSCT after ifosfamide, carboplatin, and etoposide (ICE) as high-dose chemotherapy (HDCT) regimen for relapsed or refractory medulloblastoma from 2010 to 2020. Overall response rate (ORR), overall survival (OS) and progression-free survival (PFS) of the patients were evaluated. Results: Fifteen patients were observed in this study. The mean age of the study group was 27.9±8.1 years and 53.3% of the patients were female (n=8). ORR was 100%. The median OS and PFS were 24 months [95% confidence interval (CI): 11.4-36.5] and 13 months (95% CI: 10.2-15.8), respectively. One-year OS and PFS rates were 77% and 55.2%, respectively. Five-year OS rate was 82.5%. Conclusions: AHSCT with ICE as HDCT regimen is a safe and effective treatment option for relapsed or refractory adult medulloblastoma patients with an acceptable ORR, OS and PFS time.