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

TitleOutcome and molecular analysis of young children with choroid plexus carcinoma treated with non-myeloablative therapy: results from the SJYC07 trial
Authors
Keywordschoroid plexus carcinoma
clinical trial
high-dose methotrexate
infant
TP53
Issue Date2020
PublisherOxford University Press: Open Access Journals. The Journal's web site is located at https://academic.oup.com/noa
Citation
Neuro-Oncology Advances, 2020, v. 3 n. 1, p. article no. vdaa168 How to Cite?
AbstractBackground: 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.
Persistent Identifierhttp://hdl.handle.net/10722/308352
ISSN
2021 Impact Factor: 4.036
2020 SCImago Journal Rankings: 0.746
PubMed Central ID

 

DC FieldValueLanguage
dc.contributor.authorLiu, APY-
dc.contributor.authorWu, G-
dc.contributor.authorOrr, BA-
dc.contributor.authorLin, T-
dc.contributor.authorAshford, JM-
dc.contributor.authorBass, JK-
dc.contributor.authorBowers, DC-
dc.contributor.authorHassall, Tim-
dc.contributor.authorFisher, PG-
dc.contributor.authorIndelicato, DJ-
dc.contributor.authorKlimo, P-
dc.contributor.authorBoop, F-
dc.contributor.authorConklin, H-
dc.contributor.authorOnar-Thomas, A-
dc.contributor.authorMerchant, TE-
dc.contributor.authorEllison, DW-
dc.contributor.authorGajjar, A-
dc.contributor.authorRobinson, GW-
dc.date.accessioned2021-12-01T07:52:13Z-
dc.date.available2021-12-01T07:52:13Z-
dc.date.issued2020-
dc.identifier.citationNeuro-Oncology Advances, 2020, v. 3 n. 1, p. article no. vdaa168-
dc.identifier.issn2046-2069-
dc.identifier.urihttp://hdl.handle.net/10722/308352-
dc.description.abstractBackground: 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.-
dc.languageeng-
dc.publisherOxford University Press: Open Access Journals. The Journal's web site is located at https://academic.oup.com/noa-
dc.relation.ispartofNeuro-Oncology Advances-
dc.rightsThis work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.-
dc.subjectchoroid plexus carcinoma-
dc.subjectclinical trial-
dc.subjecthigh-dose methotrexate-
dc.subjectinfant-
dc.subjectTP53-
dc.titleOutcome and molecular analysis of young children with choroid plexus carcinoma treated with non-myeloablative therapy: results from the SJYC07 trial-
dc.typeArticle-
dc.identifier.emailLiu, APY: apyliu@hku.hk-
dc.identifier.authorityLiu, APY=rp01357-
dc.description.naturepublished_or_final_version-
dc.identifier.doi10.1093/noajnl/vdaa168-
dc.identifier.pmid33506206-
dc.identifier.pmcidPMC7813199-
dc.identifier.hkuros330647-
dc.identifier.volume3-
dc.identifier.issue1-
dc.identifier.spagearticle no. vdaa168-
dc.identifier.epagearticle no. vdaa168-
dc.publisher.placeUnited Kingdom-

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