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

Obrecht-Sturm, Denise; Bison, Brigitte; Kortmann, Rolf-Dieter; Mynarek, Martin; Rutkowski, Stefan; Fleischhack, Gudrun; Schömig, Lena; Pfister, Stefan M; Sahm, Felix; Schüller, Ulrich; Gerber, Nicolas U; Schwarz, Rudolf; von Bueren, André O; Nussbaumer, Gunther; Sturm, Dominik; Sill, Martin; Benesch, Martin; Pietsch, Torsten

doi:10.1093/neuonc/noae071

Journal Article

DZNE-2024-01108

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

Obrecht-Sturm, D. ; Schömig, L. ; Mynarek, M. ; Bison, B. ; Schwarz, R. ; Pietsch, T.DZNE* ; Pfister, S. M. ; Sill, M. ; Sturm, D. ; Sahm, F. ; Kortmann, R.-D. ; Gerber, N. U. ; von Bueren, A. O. ; Fleischhack, G. ; Schüller, U. ; Nussbaumer, G. ; Benesch, M. ; Rutkowski, S.

2024
Oxford Univ. Press Oxford

Neuro-Oncology 26(9), 1712 - 1722 (2024) [10.1093/neuonc/noae071]

This record in other databases:

Please use a persistent id in citations: doi:10.1093/neuonc/noae071

Abstract: This study aims at clarifying the impact of persistent residual lesions following first-line treatment for pediatric medulloblastoma.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.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).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.

Keyword(s): Humans (MeSH) ; Medulloblastoma: diagnostic imaging (MeSH) ; Medulloblastoma: pathology (MeSH) ; Male (MeSH) ; Child (MeSH) ; Female (MeSH) ; Magnetic Resonance Imaging: methods (MeSH) ; Cerebellar Neoplasms: diagnostic imaging (MeSH) ; Cerebellar Neoplasms: pathology (MeSH) ; Disease Progression (MeSH) ; Child, Preschool (MeSH) ; Adolescent (MeSH) ; Follow-Up Studies (MeSH) ; Prognosis (MeSH) ; Retrospective Studies (MeSH) ; Survival Rate (MeSH) ; Infant (MeSH) ; Neoplasm, Residual: diagnostic imaging (MeSH) ; Neoplasm, Residual: pathology (MeSH) ; MRI ; children ; medulloblastoma ; persistent residual disease