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J Pediatr Hematol Oncol. 2017 Nov;39(8):e473-e475. doi: 10.1097/MPH.0000000000000965.

Successful Treatment of Recurrent Li-Fraumeni Syndrome-related Choroid Plexus Carcinoma.

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*Department of Pediatrics, University of Colorado School of Medicine †Center for Cancer and Blood Disorders, Children's Hospital Colorado, Aurora, CO ‡Pediatric Neuro-Oncology, Children's Hospital Los Angeles, Los Angeles, CA §Department of Pediatric Oncology, Dana-Farber Cancer Institute ∥Department of Radiation Oncology, Massachusetts General Hospital, Boston, MA ¶Department of Pathology, University of Michigan School of Medicine, Ann Arbor, MI #Department of Pathology, UTHealth McGovern Medical School, Houston, TX **AbbVie, Chicago, IL.


The management of choroid plexus carcinoma (CPC) is challenging and multifaceted. Here, we discuss a 3-year-old girl with CPC and Li-Fraumeni syndrome who achieved full remission after surgery and chemotherapy, with radiation therapy spared. At recurrence, we used a novel, standard-dose cytotoxic chemotherapy regimen, focal proton radiation therapy, and targeted agents based on morphoproteomic analysis to achieve long-term survival. We highlight the rationale for our therapy at recurrence, as well as the risk-benefit analyses necessary in decision making for these patients. Our strategy may be effective in managing other patients with recurrent CPC and Li-Fraumeni syndrome.

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