Treatment response as surrogate to predict risk for disease progression in pediatric medulloblastoma with persistent magnetic resonance imaging lesions after first-line treatment.

Autor:	Obrecht-Sturm D; Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Schömig L; Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Mynarek M; Mildred Scheel Cancer Career Center HaTriCS4, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.; Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Bison B; Department of Neuroradiology, University Hospital Augsburg, Augsburg, Germany., Schwarz R; Department for Radiotherapy, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Pietsch T; Institute of Neuropathology, Brain Tumor Reference Center of the German Society for Neuropathology and Neuroanatomy (DGNN), University of Bonn, DZNE German Center for Neurodegenerative Diseases, Bonn, Germany., Pfister SM; Department of Pediatric Hematology and Oncology, Heidelberg University Hospital, Heidelberg, Germany.; Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ) and German Consortium for Translational Cancer Research (DKTK), Heidelberg, Germany.; Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg University Hospital, Heidelberg, Germany., Sill M; Department of Pediatric Hematology and Oncology, Heidelberg University Hospital, Heidelberg, Germany.; Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ) and German Consortium for Translational Cancer Research (DKTK), Heidelberg, Germany.; Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg University Hospital, Heidelberg, Germany., Sturm D; Pediatric Glioma Research Group, German Cancer Research Center (DKFZ), Heidelberg, Germany.; Department of Pediatric Hematology and Oncology, Heidelberg University Hospital, Heidelberg, Germany.; Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg University Hospital, Heidelberg, Germany., Sahm F; CCU Neuropathology, German Cancer Research Center (DKFZ) and German Consortium for Translational Cancer Research (DKTK), Heidelberg University Hospital, Heidelberg, Germany.; Department of Neuropathology, University Heidelberg Heidelberg, Germany.; Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg University Hospital, Heidelberg, Germany., Kortmann RD; Department of Radiation Oncology, University Hospital Leipzig, Leipzig, Germany., Gerber NU; Department of Pediatric Oncology, University Children's Hospital Zürich, Zürich, Switzerland., von Bueren AO; Division of Pediatric Hematology and Oncology, Department of Pediatrics, Obstetrics and Gynecology, University Hospital of Geneva, Geneva, Switzerland., Fleischhack G; Pediatrics III, Pediatric Oncology and Hematology, University Hospital Essen, Essen, Germany., Schüller U; Research Institute Kinderkrebs-Zentrum Hamburg, Hamburg, Germany.; Department of Neuropathology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.; Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany., Nussbaumer G; Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria., Benesch M; Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical University of Graz, Graz, Austria., Rutkowski S; Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Jazyk:	angličtina
Zdroj:	Neuro-oncology [Neuro Oncol] 2024 Sep 05; Vol. 26 (9), pp. 1712-1722.
DOI:	10.1093/neuonc/noae071
Abstrakt:	Background: This study aims at clarifying the impact of persistent residual lesions following first-line treatment for pediatric medulloblastoma. Methods: Data on 84 pediatric patients with medulloblastoma and persistent residual lesions on centrally reviewed magnetic resonance imaging (MRI) at the end of first-line therapy were analyzed. Results: Twenty patients (23.8%) had residual lesions in the tumor bed (R+/M0), 51 (60.7%) had distant lesions (R0/M+) and 13 (15.5%) had both (R+/M+). Overall response to first-line therapy was minor or partial (≥ 25% reduction, minor response [MR]/PR) for 64 (76.2%) and stable disease (SD) for 20 patients (23.8%). Five-year post-primary-treatment progression-free (pptPFS) and overall survival (pptOS) were superior after MR/PR (pptPFS: 62.5 ± 7.0%[MR/PR] vs. 35.9 ± 12.8%[SD], P = .03; pptOS: 79.7 ± 5.9[MR/PR] vs. 55.5 ± 13.9[SD], P = .04). Furthermore, R+/M + was associated with a higher risk for progression (5-year pptPFS: 22.9 ± 17.9%[R+, M+] vs. 72.4 ± 12.0%[R+, M0]; P = .03). Watch-and-wait was pursued in 58 patients, while n = 26 received additional treatments (chemotherapy only, n = 19; surgery only, n = 2; combined, n = 3; valproic acid, n = 2), and their outcomes were not superior to watch-and-wait (5-year pptPFS: 58.5 ± 7.7% vs. 51.6 ± 10.7% P = .71; 5-year pptOS: 76.3 ± 6.9% vs. 69.8 ± 9.7%, P = .74). For the whole cohort, 5-year pptPFS by molecular subgroup (58 cases) were WNT: 100%, SHH: 50.0 ± 35.4%, group-4, 52.5 ± 10.5, group-3 54.2 ± 13.8%; (P = .08). Conclusions: Overall response and extent of lesions can function as surrogate parameters to predict outcomes in pediatric MB patients with persistent lesions after first-line therapy. Especially in the case of solitary persistent medulloblastoma MRI lesions, additional therapy was not beneficial. Therefore, treatment response, extent/kind of residual lesions and further diagnostic information need consideration for indication of additional treatments for persisting lesions. (© The Author(s) 2024. Published by Oxford University Press on behalf of the Society for Neuro-Oncology. All rights reserved. For commercial re-use, please contact reprints@oup.com for reprints and translation rights for reprints. All other permissions can be obtained through our RightsLink service via the Permissions link on the article page on our site—for further information please contact journals.permissions@oup.com.)
Databáze:	MEDLINE
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