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

Maxim Yankelevich; Wafik Zaky; Lucie Lafay-Cousin; Diana Osorio; Jenny Adamski; Uwe Kordes; Jonathan L Finlay; Michael Prados; Sabine Mueller

doi:10.1093/noajnl/vdae109

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

Neurooncol Adv. 2024 Jul 5;6(1):vdae109. doi: 10.1093/noajnl/vdae109. eCollection 2024 Jan-Dec.

Authors

Maxim Yankelevich¹, Wafik Zaky², Lucie Lafay-Cousin³, Diana Osorio², Jenny Adamski⁴, Uwe Kordes⁵, Jonathan L Finlay⁶, Michael Prados⁷, Sabine Mueller⁷

Affiliations

¹ Division of Oncology, St. Cristopher's Hospital for Children, Philadelphia, Pennsylvania, USA.
² Department of Pediatrics, MD Anderson Cancer Center, Houston, Texas, USA.
³ Department of Pediatrics and Oncology, Alberta Children's Hospital, Calgary, Alberta, Canada.
⁴ Birmingham Women's and Children's Hospital NHS Foundation Trust, Birmingham, UK.
⁵ Department of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
⁶ Ohio State University College of Medicine, The Ohio State University, Columbus, Ohio, USA.
⁷ Departments of Pediatrics and Neurosurgery, University of California-San Francisco, San Francisco, California, USA.

Abstract

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, 8 of the 13 with Li-Fraumeni Syndrome-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.

Keywords: Li-Fraumeni syndrome; choroid plexus carcinoma; marrow-ablative chemotherapy.