Autologous hematopoetic stem cell transplantation for children with high risk brain tumors
Autologous hematopoetic stem cell transplantation for children with high risk brain tumors
Abstract
Purpose: Although the outcome of brain tumor in children has improved significantly over decades, the high-risk brain tumors such as recurrent, residual or infant disease have a poor prognosis with conventional chemotherapy and radiotherapy after surgical resection. Over the past decade, autologous hematopoetic stem cell transplantation (AHSCT) has been applied to improve the outcome for children with high-risk brain tumors. We report on the outcome for high-risk brain tumors receiving AHSCT at a single institution. Method: Between October 1999 and August 2008, 20 patients received AHSCT using peripheral blood stem cells for high-risk brain tumor at Asan Medical Center. We reviewed their medical records including transplant data and analyzed the outcome as of August 2009. Results: Of a total of 20 patients, 8 had medulloblastoma (2 infant, 2 metastatic, 3 recurrent), 6 had supratentorial PNET (1 infant, 1 residual, 4 recurrent), 2 had pineoblastoma (1 infant, 1 recurrent), 1 had ATRT, and 2 had recurrent GCT. At the time of transplantation, 13 patients were in CR and 7 in PR. Prior to APBSCT, 9 patients (CR 5 & PR 4) received conditioning with cyclophosphamide/melphalan (CM) and 11 (CR 8 & PR 3) with carboplatin/thiotepa/etoposide (CTE). With a median follow-up of 42.0 months, 3-y EFS and OS were 50.8% and 58.3%, respectively. CR at the time of AHCT tended to have a better EFS compared to PR (64.5% vs. 28.6%, P=0.103). The 3-y EFS for 11 patients conditioned with CTE was better than that of 9 with CM (79.6% vs. 22.2%, P=0.014). Conclusion: AHSCT with CTE is highly successful treatment modality for high-risk brain tumors in children. Given the limitation of this study including small number of patients as well as heterogeneous population, further study is warranted to clarify the role of AHSCT in pediatric brain tumors.