EPEN-39. CLINICAL STRATIFIED TREATMENT OF LOCALIZED PEDIATRIC INTRACRANIAL EPENDYMOMA WITH COMBINED LOCAL IRRADIATION AND CHEMOTHERAPY WITHIN THE PROSPECTIVE, MULTICENTER E-HIT TRIAL – THE MOLECULAR SUBGROUP MATTERS

BACKGROUND: Pediatric ependymoma is a heterogenous disease. Subgroup-specific clinical information on prospectively treated patients will help to improve treatment stratification. METHODS: Within the population based, prospective, multicenter E-HIT-trial (2001–2011) patients with localized ependymom...

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Detalles Bibliográficos
Autores principales: von Hoff, Katja, Obrecht, Denise, Wening, Janna, Mynarek, Martin, Gerber, Nicolas U, Benesch, Martin, Juhnke, B - Ole, Bison, Brigitte, Warmuth-Metz, Monika, Timmermann, Beate, Faldum, Andreas, Schüller, Ulrich, Pfister, Stefan M, Kool, Marcel, Pietsch, Torsten, Kortmann, Rolf-D, Kwiecien, Robert, Pajtler, Kristian W, Witt, Hendrik, Rutkowski, Stefan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
Materias:
Ependymoma
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7715574/
http://dx.doi.org/10.1093/neuonc/noaa222.174
Descripción
Sumario:BACKGROUND: Pediatric ependymoma is a heterogenous disease. Subgroup-specific clinical information on prospectively treated patients will help to improve treatment stratification. METHODS: Within the population based, prospective, multicenter E-HIT-trial (2001–2011) patients with localized ependymoma confirmed by neuropathological central-review, received hyperfractionated local radiotherapy (68Gy, 2x1Gy/day) followed by chemotherapy (stratum-A), or chemotherapy followed by local radiotherapy (54Gy, 1.8Gy/day) (children < 4years, stratum-B), or age-adapted radiotherapy with pre-/post-irradiation chemotherapy (residual tumor, diagnosis after 2005, stratum-C). Retrospective classification of DNA-methylation was available for n=164 E-HIT-trial participants, and n=80 patients with comparable treatment and prospective registration in the subsequent HIT-interim-registry (2012–2014). FINDINGS: For 291 E-HIT-trial patients, 5-year progression-free (PFS) and overall survival (OS) were 61±3%, and 81±2%. Five-year PFS/OS after complete resection were 71±4% and 87±3% in stratum-A (n=127), and 64±5% and 86±4% in stratum-B (n=86). Outcome was poor after incomplete resection, irrespective of treatment-stratum (n=78, 5-year PFS/OS: 43±6%, 68±5%). In the pooled trial- and registry-cohort, there were 152 patients with PF-EPN-A (5-year PFS/OS: 44±4%, 77±4%), 40 of them with 1q-gain (5-year PFS/OS: 28±7%, 66±8%), 21 with PF-EPN-B (5-year PFS/OS: 90±7%, 100%), 59 with ST-EPN-RELA (5-year PFS/OS: 63±7%, 87±5%), and 4 with ST-EPN-YAP1 (2 progression/relapse, no death). CONCLUSION: Outcome differed between molecular subgroups and insufficient survival rates were achieved for patients with PF-EPN-A with 1q-gain, despite combined radio- and chemotherapy treatment. Treatment reduction in the context of a clinical trial may be considered for PF-EPN-B.

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