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| 001 | 289304 | ||
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| 100 | 1 | _ | |a Obrecht-Sturm, Denise |0 0000-0002-3216-8452 |b 0 |
| 245 | _ | _ | |a Treatment response as surrogate to predict risk for disease progression in pediatric medulloblastoma with persistent MRI lesions after first-line treatment. |
| 260 | _ | _ | |a Oxford |c 2024 |b Oxford Univ. Press |
| 336 | 7 | _ | |a article |2 DRIVER |
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| 500 | _ | _ | |a 2024 Sep 5;26(9):1712-1722 |
| 520 | _ | _ | |a 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 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, 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=0.03; pptOS: 79.7±5.9[MR/PR] vs. 55.5±13.9[SD], p=0.04). Further, 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=0.03). 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=0.71; 5-year pptOS: 76.3±6.9% vs. 69.8±9.7%, p=0.74). For the whole cohort, five-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=0.08).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 case of solitary persistent medulloblastoma MRI lesion, additional therapy was not beneficial. Therefore, treatment response, extent/kind of residual lesions and further diagnostic information needs consideration for indication of additional treatments for persisting lesions. |
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| 650 | _ | 7 | |a MRI |2 Other |
| 650 | _ | 7 | |a children |2 Other |
| 650 | _ | 7 | |a medulloblastoma |2 Other |
| 650 | _ | 7 | |a persistent residual disease |2 Other |
| 700 | 1 | _ | |a Schömig, Lena |b 1 |
| 700 | 1 | _ | |a Mynarek, Martin |0 0000-0003-3302-2719 |b 2 |
| 700 | 1 | _ | |a Bison, Brigitte |b 3 |
| 700 | 1 | _ | |a Schwarz, Rudolf |b 4 |
| 700 | 1 | _ | |a Pietsch, Torsten |b 5 |
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| 700 | 1 | _ | |a Sill, Martin |0 P:(DE-He78)45440b44791309bd4b7dbb4f73333f9b |b 7 |u dkfz |
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| 700 | 1 | _ | |a Kortmann, Rolf-Dieter |b 10 |
| 700 | 1 | _ | |a Gerber, Nicolas U |b 11 |
| 700 | 1 | _ | |a von Bueren, André O |b 12 |
| 700 | 1 | _ | |a Fleischhack, Gudrun |b 13 |
| 700 | 1 | _ | |a Schüller, Ulrich |0 0000-0002-8731-1121 |b 14 |
| 700 | 1 | _ | |a Nussbaumer, Gunther |b 15 |
| 700 | 1 | _ | |a Benesch, Martin |b 16 |
| 700 | 1 | _ | |a Rutkowski, Stefan |b 17 |
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