Trial Information

Multicenter Clinical Trial to Evaluate Efficacy of High-dose Chemotherapy With Autologous Stem Cell Rescue for Children With High-risk Brain Tumors

Although significant progress has been made in the treatment of brain tumors, the prognosis
remains dismal in patients with relapsed tumor. The outlook for infants and young children
is also poor, primarily because of the limited use of radiotherapy, although a recent report
suggested that vigorous combination chemotherapy alone improved the survival of young
children without macroscopic metastases at diagnosis. The prognosis is also not satisfactory
when a large residual tumor remains after surgery or when leptomeningeal seeding is present
at diagnosis. Given the above situation, we plan to explore the possible efficacy of
high-dose chemotherapy and autologous stem cell rescue in patients with high-risk embryonal
tumors, relapsed brain tumors and in infants and young children with brain tumors.High-dose
chemotherapy and autologous stem cell rescue has improved the survival of children with
high-risk solid tumors. this treatment strategy is based on the hypothesis that a dose
escalation might improve the survival of children with high-risk solid tumors.Many
investigators demonstrated that further dose escalation using sequential high-dose
chemotherapy and autologous stem cell rescue might result in additional improvements in the
survival of patients with high-risk tumors. As embryonal brain tumors are a chemosensitive
tumors, a strategy using high-dose chemotherapy might be effective in the treatment of
high-risk embryonal brain tumors and relapsed brain tumors. In addition, it might defer or
eliminate irradiation in infants and young children with brain tumors