Marrow-ablative Consolidation Chemotherapy and Molecular Targeted Therapy Delivered in a Risk-adapted Manner for Newly Diagnosed Children with Choroid Plexus Carcinoma: A Work in Progress

Choroid plexus carcinomas (CPC) are early childhood cancers characterized by loss of TP53 function and poor survival. We are analyzing data on TP53 status, survival and second cancers from the largest cohort of CPC receiving chemotherapy followed by consolidation with marrow-ablative chemotherapy (HDCx). Additionally, we discuss the rationale for targeted therapies for CPC patients. Currently, eight of 13 with Li-Fraumeni Syndrome (LFS)-associated CPC were treated and continued CPC-free, indicating that HDCx improves CPC-free survival in young children with TP53-mutated CPC. These data justify the inclusion of HDCx in the planned prospective international trial for children with TP53-mutated CPC.