Stratified Medicine Paediatrics: Cell free DNA and serial tumour sequencing identifies subtype specific cancer evolution and epigenetic states
We profiled a large heterogenous cohort of matched diagnostic-relapse tumour tissue and paired plasma-derived cell free DNA (cfDNA) from patients with relapsed and progressive solid tumours of childhood. Tissue and cfDNA sequencing results were concordant, with a wider spectrum of mutant alleles and higher degree of intra-tumour heterogeneity captured by the latter, if sufficient circulating […]
PARC: a phase I/II study evaluating the safety and activity of pegylated recombinant human arginase BCT-100 in relapsed/refractory cancers of children and young adults
CONCLUSION: Arginine depletion is a clinically safe and achievable strategy in children with cancer. The RP2D of BCT-100 in children with relapsed/refractory cancers is established at 1600U/kg intravenously weekly and can lead to sustained disease stability in this hard to treat population. Front Oncol. 2024 Jan 31;14:1296576. doi: 10.3389/fonc.2024.1296576. eCollection 2024. ABSTRACT BACKGROUND: The survival […]
Randomized Trial of Two Induction Therapy Regimens for High-Risk Neuroblastoma: HR-NBL1.5 International Society of Pediatric Oncology European Neuroblastoma Group Study
CONCLUSION: No difference in outcome was observed between rCOJEC and MSKCC-N5; however, acute toxicity was less with rCOJEC, and therefore rCOJEC is the preferred induction regimen for International Society of Pediatric Oncology European Neuroblastoma Group. J Clin Oncol. 2021 Aug 10;39(23):2552-2563. doi: 10.1200/JCO.20.03144. Epub 2021 Jun 21. ABSTRACT PURPOSE: Induction therapy is a critical component […]
Frequency and Prognostic Impact of ALK Amplifications and Mutations in the European Neuroblastoma Study Group (SIOPEN) High-Risk Neuroblastoma Trial (HR-NBL1)
CONCLUSION: Genetic alterations of ALK (clonal mutations and amplifications) in HR-NB are independent predictors of poorer survival. These data provide a rationale for integration of ALK inhibitors in upfront treatment of HR-NB with ALK alterations. J Clin Oncol. 2021 Oct 20;39(30):3377-3390. doi: 10.1200/JCO.21.00086. Epub 2021 Jun 11. ABSTRACT PURPOSE: In neuroblastoma (NB), the ALK receptor […]
Influence of Surgical Excision on the Survival of Patients With Stage 4 High-Risk Neuroblastoma: A Report From the HR-NBL1/SIOPEN Study
CONCLUSION: In patients with stage 4 high-risk neuroblastoma who have responded to induction therapy, CME of the primary tumor is associated with improved survival and local control after HDT, local radiotherapy (21 Gy), and immunotherapy. J Clin Oncol. 2020 Sep 1;38(25):2902-2915. doi: 10.1200/JCO.19.03117. Epub 2020 Jul 8. ABSTRACT PURPOSE: To evaluate the impact of surgeon-assessed […]
Outcome and prognostic features in paediatric pineoblastomas: analysis of cases from the Surveillance, Epidemiology, and End Results registry (1990-2007)
CONCLUSION: This study represents the largest analysis of paediatric pineoblastomas to date. The only clinically relevant prognostic factor was older age at diagnosis. The role of surgery and adjuvant radiotherapy on overall survival remains to be defined. Acta Neurochir (Wien). 2019 Sep;161(9):1799-1807. doi: 10.1007/s00701-019-03909-1. Epub 2019 May 18. ABSTRACT BACKGROUND: Paediatric pineoblastomas are rare central […]
Pathways to Diagnosis for Teenagers and Young Adults with Cancer in European Nations: A Pilot Study
CONCLUSION: Focused research upon the mechanisms underpinning complex cancer pathways, and focusing that research upon specific cancer types within TYA may be the next key areas of study. J Adolesc Young Adult Oncol. 2018 Oct;7(5):604-611. doi: 10.1089/jayao.2018.0045. Epub 2018 Jul 23. ABSTRACT PURPOSE: The diagnosis of cancer is often prolonged in teenagers and young adults […]
A phase I trial of AT9283 (a selective inhibitor of aurora kinases) in children and adolescents with solid tumors: a Cancer Research UK study
CONCLUSION: AT9283 was well tolerated in children and adolescents with solid tumors with manageable hematologic toxicity. Target inhibition was demonstrated. Disease stabilization was documented in intracranial and extracranial pediatric solid tumors and a phase II dose determined. Clin Cancer Res. 2015 Jan 15;21(2):267-73. doi: 10.1158/1078-0432.CCR-14-1592. Epub 2014 Nov 4. ABSTRACT PURPOSE: A phase I trial […]
Urine catecholamines in paediatrics
No abstract Arch Dis Child Educ Pract Ed. 2011 Jun;96(3):107-11. doi: 10.1136/adc.2010.207126. NO ABSTRACT PMID:21566154 | DOI:10.1136/adc.2010.207126