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Individualized therapeutic approaches for relapsed and refractory pediatric ependymomas: a single institution experience

P. Tinka, P. Pokorná, M. Kýr, Z. Pavelka, K. Vejmělková, H. Pálová, J. Neradil, M. Ježová, O. Slabý, J. Štěrba

. 2025 ; 173 (2) : 479-488. [pub] 20250416

Jazyk angličtina Země Spojené státy americké

Typ dokumentu časopisecké články

Perzistentní odkaz   https://www.medvik.cz/link/bmc25015480

Grantová podpora
EU4Health Programme 2021-2027 European Reference Network on Paediatric Cancer
MUNI/A/1395/2022 Specific University Research provided by MEYS
101059788 CREATIC funded by European Union's Horizon Europe Coordination and Support Action
LX22NPO5102 National Institute for Cancer Research - programme EXCELES
NV19-03-00562 Ministerstvo Zdravotnictví Ceské Republiky

PURPOSE: This retrospective study aims to show a real-life single-center experience with clinical management of relapsed pediatric ependymomas using results from comprehensive molecular profiling. METHODS: Eight relapsed ependymomas were tested by whole exome sequencing, RNA sequencing, phosphoproteomic arrays, array comparative genome hybridization, and immunohistochemistry staining for PD-L1 expression and treated with an individualized approach implementing targeted inhibitors, immunotherapy, antiangiogenic metronomic treatment, or other agents. Treatment efficacy was evaluated using progression-free survival (PFS), overall survival (OS), survival after relapse (SAR), and PFS ratios. RESULTS: Genomic analyses did not reveal any therapeutically actionable alterations. Surgery remained the cornerstone of patient treatment, supplemented by adjuvant radiotherapy. Empiric agents were chosen quite frequently, often involving drug repurposing. In six patients, prolonged PFS after relapse was seen because of immunotherapy, MEMMAT, or empiric agents and is reflected in the PFS ratio ≥ 1. The 5-year OS was 88%, the 10-year OS was 73%, the 2-year SAR was 88%, and the 5-year SAR was 66%. CONCLUSION: We demonstrated the feasibility and good safety profile. Promising was the effect of immunotherapy on ZFTA-positive ependymomas. However, further research is required to establish the most effective approach for achieving sustained remission in these patients.

Citace poskytuje Crossref.org

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