Rhabdomyosarcoma

SX Skapek, A Ferrari, AA Gupta, PJ Lupo… - Nature reviews disease …, 2019 - nature.com
Rhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children and
represents a high-grade neoplasm of skeletal myoblast-like cells. Decades of clinical and …

Molecular characteristics and therapeutic vulnerabilities across paediatric solid tumours

DTW Jones, A Banito, TGP Grünewald, M Haber… - Nature Reviews …, 2019 - nature.com
The spectrum of tumours arising in childhood is fundamentally different from that seen in
adults, and they are known to be divergent from adult malignancies in terms of cellular …

Whole genome, transcriptome and methylome profiling enhances actionable target discovery in high-risk pediatric cancer

M Wong, C Mayoh, LMS Lau, DA Khuong-Quang… - Nature medicine, 2020 - nature.com
Abstract The Zero Childhood Cancer Program is a precision medicine program to benefit
children with poor-outcome, rare, relapsed or refractory cancer. Using tumor and germline …

Genomes for kids: the scope of pathogenic mutations in pediatric cancer revealed by comprehensive DNA and RNA sequencing

S Newman, J Nakitandwe, CA Kesserwan, EM Azzato… - Cancer discovery, 2021 - AACR
Genomic studies of pediatric cancer have primarily focused on specific tumor types or high-
risk disease. Here, we used a three-platform sequencing approach, including whole …

Actionable tumor alterations and treatment protocol enrollment of pediatric and young adult patients with refractory cancers in the National Cancer Institute–Children's …

DW Parsons, KA Janeway, DR Patton… - Journal of Clinical …, 2022 - ascopubs.org
PURPOSE The National Cancer Institute–Children's Oncology Group Pediatric MATCH trial
aimed to facilitate evaluation of molecular-targeted therapies in biomarker-selected cohorts …

The pediatric precision oncology INFORM registry: clinical outcome and benefit for patients with very high-evidence targets

CM Van Tilburg, E Pfaff, KW Pajtler, KPS Langenberg… - Cancer discovery, 2021 - AACR
INFORM is a prospective, multinational registry gathering clinical and molecular data of
relapsed, progressive, or high-risk pediatric patients with cancer. This report describes long …

Recent progress in the treatment of cancer in children

E Butler, K Ludwig, HL Pacenta… - CA: a cancer journal …, 2021 - Wiley Online Library
Although significant improvements have been made in the outcomes of children with cancer,
the pace of improvement has slowed in recent years as the limits of therapy intensification …

ALK in neuroblastoma: biological and therapeutic implications

RM Trigg, SD Turner - Cancers, 2018 - mdpi.com
Neuroblastoma (NB) is the most common and deadly solid tumour in children. Despite the
development of new treatment options for high-risk NB, over half of patients relapse and five …

[HTML][HTML] The evolution of master protocol clinical trial designs: a systematic literature review

EL Meyer, P Mesenbrink, C Dunger-Baldauf… - Clinical Therapeutics, 2020 - Elsevier
Purpose Recent years have seen a change in the way that clinical trials are being
conducted. There has been a rise of designs more flexible than traditional adaptive and …

[HTML][HTML] ACCELERATE–Five years accelerating cancer drug development for children and adolescents

ADJ Pearson, SL Weiner, PC Adamson… - European Journal of …, 2022 - Elsevier
Rapid evaluation and subsequent regulatory approval of new drugs are critical to improving
survival and reducing long-term side-effects for children and adolescents with cancer. The …