Development of randomized trials in adults with medulloblastoma—the example of eortc 1634-btg/noa-23

Hau P, Frappaz D, Hovey E, Mccabe MG, Pajtler KW, Wiestler B, Seidel C, Combs SE, Dirven L, Klein M, Anazodo A, Hattingen E, Hofer S, Pfister SM, Zimmer C, Kortmann RD, Sunyach MP, Tanguy R, Effeney R, Von Deimling A, Sahm F, Rutkowski S, Berghoff AS, Franceschi E, Pineda E, Beier D, Peeters E, Gorlia T, Vanlancker M, Bromberg JEC, Gautier J, Ziegler DS, Preusser M, Wick W, Weller M (2021)

Publication Type: Journal article

Publication year: 2021

Journal

Book Volume: 13

Article Number: 3451

Journal Issue: 14

DOI: 10.3390/cancers13143451

Abstract

Medulloblastoma is a rare brain malignancy. Patients after puberty are rare and bear an intermediate prognosis. Standard treatment consists of maximal resection plus radio-chemother-apy. 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 (Odomzo™, 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 trans-lated into treatment changes in post-pubertal patients with medulloblastoma and will help to design future clinical trials.

Involved external institutions

Universitätsklinikum Regensburg Germany (DE) Centre Léon-Bérard (UNICANCER) France (FR) University of Manchester United Kingdom (GB) Deutsches Krebsforschungszentrum (DKFZ) Germany (DE) Technische Universität München (TUM) Germany (DE) Universitätsklinikum Leipzig Germany (DE) Leiden University Netherlands (NL) Vrije Universiteit Amsterdam (VU) / University Amsterdam Netherlands (NL) Nelune Comprehensive Cancer Centre Australia (AU) Goethe-Universität Frankfurt am Main Germany (DE) Universitätsspital Zürich (USZ) Switzerland (CH) Royal Brisbane and Women's Hospital Australia (AU) Universitätsklinikum Heidelberg Germany (DE) Universitätsklinikum Hamburg-Eppendorf (UKE) Germany (DE) Medizinische Universität Wien Austria (AT) L’Istituto delle Scienze Neurologiche di Bologna (ISNB) Italy (IT) Hospital Clínic de Barcelona Spain (ES) Odense Universitetshospital (OUH) Denmark (DK) European Organisation for Research and Treatment of Cancer (EORTC) Belgium (BE) Erasmus University Medical Center (MC) Netherlands (NL) Sydney Children’s Hospitals Network Australia (AU)

How to cite

APA:

Hau, P., Frappaz, D., Hovey, E., Mccabe, M.G., Pajtler, K.W., Wiestler, B.,... Weller, M. (2021). Development of randomized trials in adults with medulloblastoma—the example of eortc 1634-btg/noa-23. Cancers, 13(14). https://doi.org/10.3390/cancers13143451

MLA:

Hau, Peter, et al. "Development of randomized trials in adults with medulloblastoma—the example of eortc 1634-btg/noa-23." Cancers 13.14 (2021).

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