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Lookup NU author(s): Dr Jack GoddardORCiD, Dr Claire Keeling, Professor Steven CliffordORCiD, Professor Simon BaileyORCiD
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© 2025 The Authors. Several treatment protocols are used for the treatment of high-risk medulloblastoma (HR-MB). In 2015, the UK Children's Cancer and Leukaemia Group issued guidance recommending treatment as per the SJMB03 protocol, whilst also recognising that the COG-99701 protocol may be used. Patients were defined as high-risk if metastatic at presentation, large-cell/anaplastic histology, MYC amplification, significant residual disease or MYCN amplification. Recently, the latter two only define high risk if other adverse features are present. Methods: Retrospective multi-centre service evaluation of treatment of HR-MB at five UK centres. Patients were included if treated as per SJMB03 or COG-99701. Patients were excluded if initially treated for standard-risk medulloblastoma and subsequently treated with these protocols due to upstaging or disease progression. Results: 58 patients were identified: 26 treated as per SJMB03, 32 as per COG-99701. 5-year OS was 83 % (95 %CI 73–94 %) and 5-year PFS was 65 % (53–80 %). For patients treated as per SJMB03, 5-year OS and PFS were 80 % (65–97 %) and 75 % (60–95 %) respectively; for patients treated as per COG-99701, 5-year OS and PFS were 85 % (73–100 %) and 60 % (43–83 %). There was no significant difference in outcomes between protocols. There was a higher incidence of grade 3/4 ototoxicity (44 % vs 6 %, p = 0.001) and admission to paediatric intensive care (19 % vs 0 %, p = 0.014) in patients treated as per SJMB03 compared to COG-99701. Conclusion: These real-world outcomes are consistent with the published literature on HR-MB patients treated with these protocols within clinical trials, and provide important evidence to inform their use in routine practice.
Author(s): Apps JR, Goddard J, Sahmoud S, Green K, Hedge K, Keeling C, Cherungonath A, Srikumar B, Lumb B, Whitby J, Hayden J, Gains J, Mandeville H, Joanne L, Pizer B, Kilday JP, Clifford SC, Bailey S, Jorgensen M, Carceller F, Adamski J
Publication type: Article
Publication status: Published
Journal: EJC Paediatric Oncology
Year: 2025
Volume: 6
Print publication date: 01/12/2025
Online publication date: 10/07/2025
Acceptance date: 09/07/2025
Date deposited: 20/08/2025
ISSN (electronic): 2772-610X
Publisher: Elsevier BV
URL: https://doi.org/10.1016/j.ejcped.2025.100311
DOI: 10.1016/j.ejcped.2025.100311
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