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Roger Packer, Yimei Li, Kristina Hardy, Anna Janss, Catherine Billups, Leanne Embry, Yuanyuan Han, Patricia Cullen, Thomas Merchant, Nancy Tarbell, Ian Pollack, Amar Gajjar, Sarah Leary, Maryam Fouladi, Jeff Michalski, MBCL-07. 5-YEAR EVENT-FREE SURVIVAL (EFS) AND NEUROCOGNITIVE OUTCOME IN CHILDREN WITH MEDULLOBLASTOMA (MB) BETWEEN 3 AND 5 YEARS OF AGE: RESULTS OF CHILDREN’S ONCOLOGY GROUP STUDY ACNS0331, Neuro-Oncology, Volume 20, Issue suppl_2, June 2018, Page i118, https://doi.org/10.1093/neuonc/noy059.405
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Abstract
Primarily due to concerns about severe neurocognitive sequelae, immediate post-surgical craniospinal radiotherapy (CSRT) is often avoided in young children (<5 years of age) with MB; the price paid for such an approach in relative loss of disease control or the cognitive benefits are poorly characterized. In ACNS0331, 226 participants, aged 3-7, were randomized to either 18 or 23.4 Gy CSRT, along with local-boost RT and chemotherapy. We report outcomes for the subset between 3-5 years at diagnosis (n=83). 5-year EFS was 72 ± 5% and was similar in the 32 less than 4 years (67 ± 9%) and the 51 between 4 and 5 years of age (76 ± 7%). Although not adequately powered, there was no statistical difference in EFS based on CSRT dose (p=0.88). 81 neurocognitive assessments were obtained on 56 children. Initial median IQ was 97 (range 50-127) and dropped a median of 3 points at the second timepoint (27-48 months from diagnosis). Those <4 had a 9-point median drop compared to 2.5 in older children; there was extensive individual variability in the degree of IQ change (range = -23 to 13). There was no statistical difference in IQ as a function of CSRT, although sample size was small (p=0.52). Omissions in CSRT in this age cohort should be done cautiously, especially in those between 4 and 5, given the 5 year EFS and the concerning, but not devastating, neurocognitive declines. Pending molecular subgrouping may help in deciding which patients have the most to lose or gain by CSRT avoiding regimens.