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

Craig Erker, Martin Mynarek, Simon Bailey, Claire M. Mazewski, Lorena Baroni, Maura Massimino, Juliette Hukin, Dolly Aguilera, Andrea M. Cappellano, Vijay Ramaswamy, Alvaro Lassaletta, Sébastien Perreault, Cassie N. Kline, Revathi Rajagopal, George Michaiel, Michal Zapotocky, Vicente Santa-Maria Lopez, Andres Morales La Madrid, Chantel Cacciotti, Eric S. SandlerLindsey M. Hoffman, Darren Klawinski, Sara Khan, Ralph Salloum, Anna L. Hoppmann, Valérie Larouche, Kathleen Dorris, Helen Toledano, Stephen W. Gilheeney, Mohamed S. Abdelbaki, Beverly Wilson, Derek S. Tsang, Jeffrey Knipstein, Michal Yalon Oren, Shafqat Shah, Jeffrey C. Murray, Kevin F. Ginn, Zhihong J. Wang, Gudrun Fleischhack, Denise Obrecht, Svenja Tonn, Virginia L. Harrod, Kara Matheson, Bruce Crooks, Douglas R. Strother, Kenneth J. Cohen, Jordan R. Hansford, Sabine Mueller, Ashley Margol, Amar Gajjar, Girish Dhall, Jonathan L. Finlay, Paul A. Northcott, Stefan Rutkowski, Steven C. Clifford, Giles Robinson, Eric Bouffet, Lucie Lafay-Cousin

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6 Scopus citations

Abstract

PURPOSEInfant 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.METHODSWe 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.RESULTSThe 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 â ‰¥ 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).CONCLUSIONA 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.

Original languageEnglish
Pages (from-to)1921-1932
Number of pages12
JournalJournal of Clinical Oncology
Volume41
Issue number10
DOIs
StatePublished - Apr 1 2023

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