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Controlled Clinical Trial
. 2008 Feb;86(3):337-47.
doi: 10.1007/s11060-007-9478-0. Epub 2007 Sep 29.

Autologous hematopoietic stem cell transplantation for high-risk brain tumors in children

Daniel Ka Leung Cheuk  1 , Tsz Leung Lee, Alan Kwok Shing Chiang, Shau Yin Ha, Godfrey Chi Fung Chan
Affiliations
Controlled Clinical Trial

Autologous hematopoietic stem cell transplantation for high-risk brain tumors in children

Daniel Ka Leung Cheuk et al. J Neurooncol. 2008 Feb.

Abstract

Autologous hematopoietic stem cell transplant (AHSCT) has been advocated as a form of salvage therapy for children with high-risk or relapsed brain tumors but only limited data are available currently. We report the outcomes of pediatric brain tumors treated with AHSCT in a quaternary referral center in Hong Kong over 10 years (June 1996-May 2006). Thirteen patients with medulloblastoma (n = 9), cerebral primitive neuroectodermal tumor (n = 1), ependymoma (n = 1), germ cell tumor (n = 1) and cerebellar rhabdoid (n = 1) were transplanted because of tumor residual (n = 1) or recurrence (n = 12). Uniform upfront treatment protocols were adopted according to specific tumor types. Prior to AHSCT, 8 patients (61.5%) achieved complete remission and 5 (38.5%) were in partial remission. Conditioning employed thiotepa 300 mg/m2, etoposide 250 mg/m2)and carboplatin 500 mg/m2 daily for 3 days. Toxicity included mucositis and neutropenic fever in all patients, grade 4 hepatic toxicity in 4 patients (including hepatic veno-occlusive disease in 2 patients) and grade 4 renal toxicity in 1 patient. The 5-year event-free survival was 53.9%. Five patients died of disease recurrence or progression 8-21 months after transplant with a median disease-free period of 8 months post-transplant. One died of transplant-related complications in the early post-transplant period. Seven survived for a median of 5.4 years (maximum follow-up of 9.8 years), with six having Lansky-Karnofsky performance score above 80. All survivors had complete remission before transplant though 2 had leptomeningeal spread. We conclude that AHSCT can achieve long-term survival in children with recurrent brain tumor. However, those with macroscopic residual tumor before transplant cannot be salvaged.

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Figures

Fig. 1
Fig. 1
Survival of all patients after AHSCT
Fig. 2
Fig. 2
(a) Survival of patients with or without complete remission, (b) Survival of patients with or without metastatic relapse, (c) Survival of patients with or without RT at initial diagnosis
Fig. 3
Fig. 3
Survival of medulloblastoma patients after AHSCT
See this image and copyright information in PMC

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