Journal Article

DKFZ-2021-00673

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Relevance of Molecular Groups in Children with Newly Diagnosed Atypical Teratoid Rhabdoid Tumor: Results from Prospective St. Jude Multi-Institutional Trials.

Upadhyaya, S. A. ; Robinson, G. ; Onar-Thomas, A. ; Orr, B. A. ; Johann, P. ; Wu, G. ; Billups, C. A. ; Tatevossian, R. G. ; Dhanda, S. K. ; Srinivasan, A. ; Broniscer, A. ; Qaddoumi, I. ; Vinitsky, A. ; Armstrong, G. T. ; Bendel, A. ; Hassall, T. E. ; Partap, S. ; Fisher, P. G. ; Crawford, J. R. ; Chintagumpala, M. M. ; Bouffet, E. ; Gururangan, S. ; Mostafavi, R. ; Sanders, R. P. ; Klimo, P. ; Patay, Z. ; Indelicato, D. J. ; Nichols, K. E. ; Boop, F. A. ; Merchant, T. E. ; Kool, M.DKFZ* ; Ellison, D. W. ; Gajjar, A.

2021
AACR Philadelphia, Pa. [u.a.]

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Please use a persistent id in citations: doi:10.1158/1078-0432.CCR-20-4731

Abstract: Report relevance of molecular groups to clinico-pathologic features, germline SMARCB1/SMARCA4 alterations (GLA), and survival of children with atypical teratoid rhabdoid tumor (ATRT) treated in two multi-institutional clinical trials.Seventy-four participants with newly diagnosed ATRT were treated in two trials: infants (SJYC07: age<3 years; n=52) and children (SJMB03: age 3-21 years; n=22), using surgery, conventional chemotherapy (infants), or dose-dense chemotherapy with autologous stem cell rescue (children), and age- and risk-adapted radiation therapy [focal (infants) and craniospinal (CSI) (children)]. Molecular groups ATRT-MYC (MYC), ATRT-SHH (SHH), and ATRT-TYR (TYR) were determined from tumor DNA methylation profiles.Twenty-four participants (32%) were alive at time of analysis at a median follow-up of 8.4 years (range, 3.1-14.1 years). Methylation profiling classified 64 ATRTs as TYR (n=21), SHH (n=30), and MYC (n=13), SHH group being associated with metastatic disease. Among infants, TYR group had the best overall survival (OS) (P=0.02). However, outcomes did not differ by molecular groups among infants with non-metastatic (M0) disease. Children with M0 disease and <1.5 cm2 residual tumor had a 5-year progression-free survival (PFS) of 72.7{plus minus}12.7% and OS of 81.8{plus minus}11%. Infants with M0 disease had a 5-year PFS of 39.1{plus minus}11.5% and OS of 51.8{plus minus}12%. Those with metastases fared poorly [5-year OS 25{plus minus}12.5% (children) and 0% (infants)]. SMARCB1 GLAs were not associated with PFS.Among infants, those with ATRT-TYR had the best OS. ATRT-SHH was associated with metastases and consequently with inferior outcomes. Children with non-metastatic ATRT benefit from post-operative CSI and adjuvant chemotherapy.

Note: 27(10):2879-2889

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Contributing Institute(s):

1. B062 Pädiatrische Neuroonkologie (B062)

Research Program(s):

1. 312 - Funktionelle und strukturelle Genomforschung (POF4-312) (POF4-312)

Appears in the scientific report 2021

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Database coverage:
; BIOSIS Previews ; Biological Abstracts ; Clarivate Analytics Master Journal List ; Current Contents - Clinical Medicine ; Essential Science Indicators ; IF >= 10 ; JCR ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

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