Background: Chromatin remodeling complexes including SWI/SNF are implicated in a wide variety of cellular processes including nuclear organization, chromosomal stability and gene expression, and mutations in SWI/SNF components play an important role in many cancer types. In neuroblastoma (NB), recent whole-genome/whole-exome sequencing efforts results have detected genetic alterations in chromatin remodeling genes such as ARID1A and ARID1B.
Methods: To explore the potential recurrence of genetic alterations in chromatin remodeling genes in a clinically representative cohort of NB patients (255 diagnostic samples), we designed a TruSeq Custom Amplicon panel (TSCA, Illumina) targeting 33 SWI/SNF genes (261.686bp). Libraries prepared from 50ng of genomic DNA were subjected to 150bp paired-end sequencing, with a high coverage (mean 2000X). After sequence alignment, two analyses were initiated. Clonal/sub-clonal mutations were detected by ACGT-base calling approach and statistical comparison between samples and controls. Structural variations will be searched for by gene dosage normalization within and between samples/controls.
Furthermore, a series of 31 NB cell lines and 6 germline controls were included in this study.
Results: A total of 96 clonal mutations (mutated allele fraction >20%) were detected. Overall, 35% of NB patients showed a mutation in at least one chromatin-remodeling gene; the most frequently mutated genes were ARID1A (10/255), ARID1B (3/255), BRD7 (3/255), MLL3 (10/255) and SMARCC2 (6/255) genes. Furthermore, 11 NB cell lines showed a clonal mutation in at least one of the studied genes. Mutations detected in NB cells lines were validated by RNA sequencing. Analyses to detect sub-clonal mutations and structural variations are ongoing.
No statistically significant differences in survival of patients with chromatin-remodeling genes wild-type versus chromatin-remodeling genes mutated at clonal level were observed.
Conclusions: The high frequency of clonal mutations highlights the dysregulation of chromatin remodeling in pediatric tumorigenesis and suggests potential new approaches for the management of patients with neuroblastoma.