TY  - JOUR
AU  - Peyrl, Andreas
AU  - Chocholous, Monika
AU  - Sabel, Magnus
AU  - Lassaletta, Alvaro
AU  - Sterba, Jaroslav
AU  - Leblond, Pierre
AU  - Nysom, Karsten
AU  - Torsvik, Ingrid
AU  - Chi, Susan N
AU  - Perwein, Thomas
AU  - Jones, Neil
AU  - Holm, Stefan
AU  - Nyman, Per
AU  - Mörse, Helena
AU  - Öberg, Anders
AU  - Weiler-Wichtl, Liesa
AU  - Leiss, Ulrike
AU  - Haberler, Christine
AU  - Schmook, Maresa T
AU  - Mayr, Lisa
AU  - Dieckmann, Karin
AU  - Kool, Marcel
AU  - Gojo, Johannes
AU  - Azizi, Amedeo A
AU  - André, Nicolas
AU  - Kieran, Mark
AU  - Slavc, Irene
TI  - Sustained Survival Benefit in Recurrent Medulloblastoma by a Metronomic Antiangiogenic Regimen: A Nonrandomized Controlled Trial.
JO  - JAMA oncology
VL  - 9
IS  - 12
SN  - 2374-2437
CY  - Chicago, Ill.
PB  - American Medical Association
M1  - DKFZ-2023-02179
SP  - 1688-1695
PY  - 2023
N1  - 2023 Dec 1;9(12):1688-1695
AB  - Medulloblastoma recurrence in patients who have previously received irradiation has a dismal prognosis and lacks a standard salvage regimen.To evaluate the response rate of pediatric patients with medulloblastoma recurrence using an antiangiogenic metronomic combinatorial approach (Medulloblastoma European Multitarget Metronomic Anti-Angiogenic Trial [MEMMAT]).This phase 2, investigator-initiated, multicenter nonrandomized controlled trial assessed 40 patients with relapsed or refractory medulloblastoma without a ventriculoperitoneal shunt who were younger than 20 years at original diagnosis. Patients were enrolled between April 1, 2014, and March 31, 2021.Treatment consisted of daily oral thalidomide, fenofibrate, celecoxib, and alternating 21-day cycles of low-dose (metronomic) oral etoposide and cyclophosphamide, supplemented by intravenous bevacizumab and intraventricular therapy consisting of alternating etoposide and cytarabine.The primary end point was response after 6 months of antiangiogenic metronomic therapy. Secondary end points included progression-free survival (PFS), overall survival (OS), and quality of life. Adverse events were monitored to assess safety.Of the 40 patients (median [range] age at treatment start, 10 [4-17] years; 25 [62.5
LB  - PUB:(DE-HGF)16
C6  - pmid:37883081
DO  - DOI:10.1001/jamaoncol.2023.4437
UR  - https://inrepo02.dkfz.de/record/285006
ER  -