TY  - JOUR
A1  - Hau, Peter
A1  - Frappaz, Didier
A1  - Hovey, Elizabeth
A1  - McCabe, Martin G.
A1  - Pajtler, Kristian Wilfried
A1  - Wiestler, Benedikt
A1  - Seidel, Clemens
A1  - Combs, Stephanie
A1  - Dirven, Linda
A1  - Klein, Martin
A1  - Anazodo, Antoinette
A1  - Hattingen, Elke
A1  - Hofer, Silvia
A1  - Pfister, Stefan
A1  - Zimmer, Claus
A1  - Kortmann, Rolf-Dieter
A1  - Sunyach, Marie-Pierre
A1  - Tanguy, Ronan
A1  - Effeney, Rachel
A1  - Deimling, Andreas von
A1  - Sahm, Felix
A1  - Rutkowski, Stefan
A1  - Berghoff, Anna
A1  - Franceschi, Enrico
A1  - Pineda, Estela
A1  - Beier, Dagmar
A1  - Peeters, Ellen
A1  - Gorlia, Thierry
A1  - Vanlancker, Maureen
A1  - Bromberg, Jacoline E. C.
A1  - Gautier, Julien
A1  - Ziegler, David S.
A1  - Preusser, Matthias
A1  - Wick, Wolfgang
A1  - Weller, Michael
T1  - Development of randomized trials in adults with medulloblastoma - the example of EORTC 1634-BTG/NOA-23
T2  - Cancers
N2  - Medulloblastoma is a rare brain malignancy. Patients after puberty are rare and bear an intermediate prognosis. Standard treatment consists of maximal resection plus radio-chemotherapy. Treatment toxicity is high and produces disabling long-term side effects. The sonic hedgehog (SHH) subgroup is highly overrepresented in the post-pubertal and adult population and can be targeted by smoothened (SMO) inhibitors. No practice-changing prospective randomized data have been generated in adults. The EORTC 1634-BTG/NOA-23 trial will randomize patients between standard-dose vs. reduced-dosed craniospinal radiotherapy and SHH-subgroup patients between the SMO inhibitor sonidegib (OdomzoTM, Sun Pharmaceuticals Industries, Inc., New York, USA) in addition to standard radio-chemotherapy vs. standard radio-chemotherapy alone to improve outcomes in view of decreased radiotherapy-related toxicity and increased efficacy. We will further investigate tumor tissue, blood, and cerebrospinal fluid as well as magnetic resonance imaging and radiotherapy plans to generate information that helps to further improve treatment outcomes. Given that treatment side effects typically occur late, long-term follow-up will monitor classic side effects of therapy, but also health-related quality of life, cognition, social and professional outcome, and reproduction and fertility. In summary, we will generate unprecedented data that will be translated into treatment changes in post-pubertal patients with medulloblastoma and will help to design future clinical trials.
KW  - medulloblastoma
KW  - adult
KW  - clinical trial
KW  - randomized
KW  - subgrouping
KW  - cerebrospinal fluid
KW  - magnetic resonance imaging
KW  - radiotherapy
KW  - chemotherapy
KW  - targeted therapy
Y1  - 2021
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/62929
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-629297
SN  - 2072-6694
N1  - This research is funded by Deutsche Krebshilfe, grant number 70113453; by Canteen Australia; by Cancer Australia, grant number 1165910; by the Dutch Cancer Society, grant number 2021-1/13555; by the French Ministry of Health, grant number PHRC-K20-179; by the Swiss Brain Tumor Foundation; by an internal grant of the EORTC Brain Tumor Group, and by an educational grant of Sun Pharmaceuticals Industries Ltd.
VL  - 13
IS  - 14, art. 3451
SP  - 1
EP  - 20
PB  - MDPI
CY  - Basel
ER  -