High-dose Chemotherapy With Autologous Stem Cell Rescue in Pediatric High-risk Brain Tumors

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

• Study No.: KSPNO-S-081 Reduced-dose Craniospinal Radiotherapy Followed by High-dose Chemotherapy and Autologous Stem Cell Rescue in Children with Newly Diagnosed High-risk Brain Tumor
• Study No.: KSPNO-S-082 High-dose Chemotherapy and Autologous Stem Cell Rescue in Infants and Young Children with Newly Diagnosed High-risk Brain Tumor To Avoid or Reduce Craniospinal Radiation
• Study No.: KSPNO-S-083 High-dose Chemotherapy and Autologous Stem Cell Rescue in Children with Recurrent Brain Tumor or Non-germinomatous Germ Cell Tumor with Inadequate Response to Conventional Treatment

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

Inclusion Criteria:

• KSPNO-S-081: newly diagnosed embryonal brain tumor, age >= 3 years
• KSPNO-S-082: all high-grade or malignant brain tumor, age <3 years
• KSPNO-S-083: recurrent embryonal brain tumors, recurrent CNS germ cell tumor

Exclusion Criteria:

• Patients with severe comorbid organ dysfunction (NCI grade > 2 organ toxicity) prior to high-dose chemotherapy
• Patients with progressed tumor prior to high-dose chemotherapy
• Patients whose parents do not want to undergo high-dose chemotherapy and autologous stem cell rescue