Outcome and molecular analysis of young children with choroid plexus carcinoma treated with non-myeloablative therapy: Results from the SJYC07 trial

Anthony P.Y. Liu, Gang Wu, Brent A. Orr, Tong Lin, Jason M. Ashford, Johnnie K. Bass, Daniel C. Bowers, Tim Hassall, Paul G. Fisher, Daniel J. Indelicato, Paul Klimo, Frederick Boop, Heather Conklin, Arzu Onar-Thomas, Thomas E. Merchant, David W. Ellison, Amar Gajjar, Giles W. Robinson

Research output: Contribution to journalArticlepeer-review

9 Scopus citations


Background. Choroid plexus carcinoma (CPC) is a rare and aggressive tumor of infancy without a clear treatment strategy. This study describes the outcomes of children with CPC treated on the multi-institutional phase 2 SJYC07 trial and reports on the significance of clinical and molecular characteristics. Methods. Eligible children <3 years-old with CPC were postoperatively stratified to intermediate-risk (IR) stratum if disease was localized or high-risk (HR) stratum, if metastatic. All received high-dose methotrexate-containing induction chemotherapy. IR-stratum patients received focal irradiation as consolidation whereas HR-stratum patients received additional chemotherapy. Consolidation was followed by oral antiangiogenic maintenance regimen. Survival rates and potential prognostic factors were analyzed. Results. Thirteen patients (median age: 1.41 years, range: 0.21-2.93) were enrolled; 5 IR, 8 HR. Gross-total resection or near-total resection was achieved in ten patients and subtotal resection in 3. Seven patients had TP53-mutant tumors, including 4 who were germline carriers. Five patients experienced progression and died of disease; 8 (including 5 HR) are alive without progression. The 5-year progression-free survival (PFS) and overall survival rates were 61.5 ± 13.5% and 68.4 ± 13.1%. Patients with TP53-wild-type tumors had a 5-year PFS of 100% as compared to 28.6 ± 17.1% for TP53-mutant tumors (P = .012). Extent of resection, metastatic status, and use of radiation therapy were not significantly associated with survival. Conclusions. Non-myeloablative high-dose methotrexate-containing therapy with maximal surgical resection resulted in long-term PFS in more than half of patients with CPC. TP53-mutational status was the only significant prognostic variable and should form the basis of risk-stratification in future trials.

Original languageEnglish (US)
Article numbervdaa168
JournalNeuro-Oncology Advances
Issue number1
StatePublished - Jan 1 2021


  • TP53
  • choroid plexus carcinoma
  • clinical trial
  • high-dose methotrexate
  • infant

ASJC Scopus subject areas

  • Clinical Neurology
  • Oncology
  • Surgery


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