Pilot Study of Intrathecal Topotecan and Maintenance Chemotherapy for High-risk Embryonal Central Nervous System Tumors in Children Under 6 Years

What this Trial Is About

Researchers are evaluating the feasibility of adding intrathecal chemotherapy and maintenance therapy after high-dose chemotherapy for treating newly diagnosed high-risk embryonal brain tumors (HR-EBTs) in children under 6 years old. This pilot study focuses on patients with specific high-risk central nervous system embryonal tumors confirmed by histologic or molecular diagnosis, aiming to improve treatment approaches for this vulnerable population. Participants will receive a comprehensive treatment plan starting with three cycles of induction chemotherapy including intrathecal cytarabine and hydrocortisone, alongside vincristine, cyclophosphamide, cisplatin, and etoposide. This is followed by three cycles of consolidation chemotherapy with carboplatin, thiotepa, and autologous stem cell rescue. After consolidation, patients enter a maintenance phase lasting 48 to 54 weeks, with therapy tailored by risk group: low-risk patients receive monthly intrathecal topotecan plus tamoxifen and retinoic acid, while high-risk patients receive monthly intrathecal topotecan combined with isotretinoin, celecoxib, etoposide, temozolomide, and cyclophosphamide. During the study, patients will be monitored through regular follow-up visits every three months for two years after treatment to assess progression-free and overall survival. Response to treatment will be evaluated using modified Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria. The study also includes genetic and genomic analyses of tumor and cerebrospinal fluid samples to explore the biological characteristics of these tumors. Safety and toxicity of the added therapies will be closely observed throughout the study duration.

Pilot Study of IT Topotecan and Maintenance Chemotherapy for HR-EBTs in Children < 6 Years, Post Consolidation