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Outcomes of Infants and Young Children With Relapsed Medulloblastoma After Initial Craniospinal Irradiation-Sparing Approaches: An International Cohort Study

Lookup NU author(s): Professor Simon BaileyORCiD, Professor Steven CliffordORCiD


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PURPOSE: Infant and young childhood medulloblastoma (iMB) is usually treated without craniospinal irradiation (CSI) to avoid neurocognitive late effects. Unfortunately, many children relapse. The purpose of this study was to assess salvage strategies and prognostic features of patients with iMB who relapse after CSI-sparing therapy. METHODS: We assembled a large international cohort of 380 patients with relapsed iMB, age younger than 6 years, and initially treated without CSI. Univariable and multivariable Cox models of postrelapse survival (PRS) were conducted for those treated with curative intent using propensity score analyses to account for confounding factors. RESULTS: The 3-year PRS, for 294 patients treated with curative intent, was 52.4% (95% CI, 46.4 to 58.3) with a median time to relapse from diagnosis of 11 months. Molecular subgrouping was available for 150 patients treated with curative intent, and 3-year PRS for sonic hedgehog (SHH), group 4, and group 3 were 60%, 84%, and 18% (P = .0187), respectively. In multivariable analysis, localized relapse (P = .0073), SHH molecular subgroup (P = .0103), CSI use after relapse (P = .0161), and age ≥ 36 months at initial diagnosis (P = .0494) were associated with improved survival. Most patients (73%) received salvage CSI, and although salvage chemotherapy was not significant in multivariable analysis, its use might be beneficial for a subset of children receiving salvage CSI < 35 Gy (P = .007). CONCLUSION: A substantial proportion of patients with relapsed iMB are salvaged after initial CSI-sparing approaches. Patients with SHH subgroup, localized relapse, older age at initial diagnosis, and those receiving salvage CSI show improved PRS. Future prospective studies should investigate optimal CSI doses and the role of salvage chemotherapy in this population.

Publication metadata

Author(s): Erker C, Mynarek M, Bailey S, Mazewski CM, Baroni L, Massimino M, Hukin J, Aguilera D, Cappellano AM, Ramaswamy V, Lassaletta A, Perreault S, Kline CN, Rajagopal R, Michaiel G, Zapotocky M, Santa-Maria Lopez V, La Madrid AM, Cacciotti C, Sandler ES, Hoffman LM, Klawinski D, Khan S, Salloum R, Hoppmann AL, Larouche V, Dorris K, Toledano H, Gilheeney SW, Abdelbaki MS, Wilson B, Tsang DS, Knipstein J, Oren MY, Shah S, Murray JC, Ginn KF, Wang ZJ, Fleischhack G, Obrecht D, Tonn S, Harrod VL, Matheson K, Crooks B, Strother DR, Cohen KJ, Hansford JR, Mueller S, Margol A, Gajjar A, Dhall G, Finlay JL, Northcott PA, Rutkowski S, Clifford SC, Robinson G, Bouffet E, Lafay-Cousin L

Publication type: Article

Publication status: Published

Journal: Journal of Clinical Oncology

Year: 2023

Volume: 41

Issue: 10

Pages: 1921-1932

Print publication date: 01/04/2023

Online publication date: 22/12/2022

Acceptance date: 28/10/2022

ISSN (print): 0732-183X

ISSN (electronic): 1527-7755

Publisher: American Society of Clinical Oncology


DOI: 10.1200/JCO.21.02968

PubMed id: 36548930


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