Outcome and molecular analysis of young children with choroid plexus carcinoma treated with non-myeloablative therapy: results from the SJYC07 trial. Issue 1 (15th December 2020)

• Record Type: Journal Article
• Authors: Liu, Anthony P Y; Wu, Gang; Orr, Brent A; Lin, Tong; Ashford, Jason M; Bass, Johnnie K; Bowers, Daniel C; Hassall, Tim; Fisher, Paul G; Indelicato, Daniel J; Klimo, Paul; Boop, Frederick; Conklin, Heather; Onar-Thomas, Arzu; Merchant, Thomas E; Ellison, David W; Gajjar, Amar; Robinson, Giles W
• Is Part Of: Neuro-oncology advances. Volume 3:Issue 1(2021)
• Publication Date: 2020-12-15
• Subjects: choroid plexus carcinoma -- clinical trial -- high-dose methotrexate -- infant -- TP53; 616.99481
• DOI: 10.1093/noajnl/vdaa168
• Languages: English
• ISSNs: 2632-2498

Abstract

Abstract: 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.