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Join our roundtable discussion led by Hannah Moir, featuring four experts in paediatric oncology: Kathy Pritchard-Jones, Nienke Streefkerk, Jacqueline Kraveka, and Giselle Sholler.
The panel share key takeaways and learnings from the 56th Annual Congress of the International Society of Pediatric Oncology (SIOP), which was held in Honolulu, Hawaii, USA, in October 2024.
Below you will find a written summary that encapsulates the key points of this discussion—click here to view.
During this discussion, our expert panel cover:
- the challenges in diagnostic testing;
- advances in the treatment of high-risk malignancies;
- developments in precision medicine and creating targeted therapies; and
- the future direction of research and the benefits of collaborative initiatives.
Expert Panel:
Kathy Pritchard-Jones1
Nienke Streefkerk2
Jacqueline Kraveka3
Giselle Sholler4
1. Professor of Paediatric Oncology, University College London (UCL) Great Ormond Street Institute of Child Health, London, UK. Immediate Past President of SIOP.
2. Medical Doctor, PhD candidate, and researcher, Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.
3. Professor of Paediatrics and Paediatric Haematologist-Oncologist, Medical University of South Carolina, Charleston, South Carolina, USA.
4. Paediatric Haematologist-Oncologist and Division Chief of Paediatric Haematology and Oncology, Penn State Health Children’s Hospital Division; and Professor of Paediatrics and Pharmacology, Penn State College of Medicine, Pennsylvania, USA.
Host:
Hannah Moir5
5. Head Medical Writer, Moderator, and Educator, EMJ, London, UK. Senior Fellow (Health & Exercise Prescription), Faculty of Health, Science, Social Care and Education, Kingston University, London, UK.
Insights from SIOP 2024 Video Summary
Acknowledgements: Medical writing assistance was provided by Brigitte Scott, MarYas Editorial Services, Cowlinge, UK.
In this interview on advances in paediatric oncology, four experts in the field, Kathy Pritchard-Jones, Giselle Sholler, Jackie Kraveka, and Nienke Streefkerk, discuss the highlights from the 56th Annual International Society of Pediatric Oncology (SIOP) Congress in October 2024 in Honolulu, Hawaii, USA. The experts explore precision medicine, advances in the treatment of high-risk malignancies, and platinum-related ototoxicity, in the context of global disparities in access to paediatric cancer care and differences in survival rates.
WHO Global Initiative for Childhood Cancer
Pritchard-Jones describes the key updates from the paediatric oncology campaigns presented by the WHO and SIOP, emphasising the importance of the WHO Global Initiative for Childhood Cancer,1 with 77 low- and middle-income countries now committed to achieving the target 60% survival rate. Pritchard-Jones also highlights the success of the CureAll framework,2 which divides the Global Initiative into 10 pillars of activity that are expected to lead to “real progress in childhood cancer services and outcomes”.
Renal Tumours in Children
Focussing on renal tumours, Pritchard-Jones discusses the challenges of treating children with rare subtypes of Wilms tumour and non-Wilms tumours, who have a poor outcome. Many countries are implementing molecular characterisation in paediatric renal oncology practice; however, Pritchard-Jones advocates for fine-tuning in the use of molecular biomarkers for the majority of children with Wilms tumour and identifying the rare subgroups that require an alternative approach or stronger therapy from the start. Pritchard-Jones explains that there is no ‘magic bullet’ for these patients, but presentations and discussions at SIOP 20243,4 have led to renewed enthusiasm for a worldwide trial in patients with Wilms tumour at high risk of relapse or refractory disease.
Molecular Profiling and Targeted Therapies in Medulloblastoma
The subject of molecular profiling is picked up by Sholler, who refers to a case study presented at SIOP 20245 of a child with medulloblastoma who had progressed following standard-of-care therapy. The use of genomic sequencing and molecular tumour boards to identify targeted therapies in this case led to the identification of four medications that resulted in the long-term survival of the patient. Sholler highlights the importance of understanding the biology and genomics of paediatric cancers, as this informs about different subgroups and guides the development of targeted therapies and immunotherapies.6 Furthermore, Sholler specifies the importance of international collaboration in the drive to access molecular tests and genomic data worldwide, as well as the need for funding and training sites to collect and analyse tissues for genomic sequencing.
Surgical Resection in Children with Neuroblastoma
Next, Kraveka discusses recent advancements in neuroblastoma research, including a post-hoc analysis showing that geographical location did not impact outcomes in patients using eflornithine (DFMO) as maintenance therapy to prevent relapse in neuroblastoma.7 Kraveka referred to presentations at SIOP 2024 that highlighted the importance of achieving complete surgical resection prior to consolidation in patients with neuroblastoma, as residual disease reduces event-free survival by 20%.8,9 Kraveka acknowledges the surgical expertise required to excise tumours that are often wrapped around vessels and nerves and identifies the potential benefits of increasing the dose of radiation therapy for residual disease after surgery.
Ototoxicity and Audiological Testing
Streefkerk explains that platinum treatment (mainly cisplatin) is a major risk factor for ototoxicity;10 therefore, audiological testing is essential in paediatric patients with cancer who are receiving platinum-based chemotherapy. According to Streefkerk, early detection of hearing loss is crucial to enable the early implementation of education and support for this impairment, thereby potentially mitigating long-term effects on quality of life. Standard audiological testing with a pure tone audiogram is challenging for young children, who require testing at higher frequencies. Otoacoustic emissions can be used as a screening method to identify children at high risk of hearing loss;11 however, a classification system is lacking for these tests. Streefkerk outlines that there are various initiatives in place that aim to make audiological testing more accessible in low- and middle-income countries.
Streefkerk describes studies on the otoprotective effects of systemic sodium thiosulfate in paediatric patients with cancer, which showed similar survival rates and an approximately 50% reduction in hearing loss with cisplatin–sodium thiosulphate versus cisplatin alone.12,13 Streefkerk outlines that an interval of 6 hours between the end of cisplatin therapy and administration of systemic sodium thiosulphate has been shown to protect the ear from damage without impacting cisplatin efficacy. Streefkerk notes the challenges of administering sodium thiosulfate locally, and the necessity for further research in this area in adults, before advancing into paediatric care.
Data Sharing in Paediatric Oncology Practice and Research
Data sharing is a challenging but essential component of paediatric oncology practice and research, according to Pritchard-Jones, who refers to the success of the collaborative International Benchmarking of Childhood Cancer Survival by Stage (BENCHISTA) Project,14 which compares childhood cancer survival rates across 27 countries. Pritchard-Jones emphasises the need for successful use cases to demonstrate the safe sharing of patient-level data; to enable future collaborations, data comparisons, and monitoring of trends over time; and to enhance understanding of the variations in overall survival. Supporting low- and middle-income countries in these data sharing activities is essential to optimise information exchange.
Final Thoughts from the Experts
The experts conclude that international collaboration, data sharing, further research, and efforts to expand access to clinical information and genomic data are key to advancing paediatric oncology care and improving patient outcomes. Highlights of SIOP 2024 for the experts include the focus on rare and more challenging cancers, the importance of implementation science in ensuring approval of a pharmaceutical product translates to patient benefit, and the well-attended session on female leaders in paediatric oncology, which showed that challenges faced by female leaders in the field are universal. The experts recognise the value of SIOP in bringing together multidisciplinary professionals from around the world in a supportive environment that informs and inspires audiences with the latest scientific advancements in the field, and “nurtures the great minds of the future”.
References
1. World Health Organization (WHO). The Global Initiative for Childhood Cancer. Available at: https://www.who.int/initiatives/the-global-initiative-for-childhood-cancer. Last accessed: 12 December 2024.
2. World Health Organization (WHO). CureAll Framework: WHO Global Initiative for Childhood Cancer. 2021. Available at: https://iris.who.int/bitstream/handle/10665/347370/9789240025271-eng.pdf. Last accessed: 12 December 2024.
3. Groenendijk A et al. Characteristics and outcome of patients with second and subsequent relapse of Wilms tumor – a report from the SIOP Renal Tumor Study Group. Abstract 1941. SIOP 2024, 17-20 October, 2024.
4. Al-Saadi R et al. Multimodality detection of tumour rupture in children with Wilms tumour. Abstract 950. SIOP 2024, 17-20 October, 2024.
5. Moore A et al. Precision medicine for patient with medulloblastima identifies targeted therapy. Abstract 327. SIOP 2024, 17-20 October, 2024.
6. Lewis EC et al. A subset analysis of a phase II trial evaluating the use of DFMO as maintenance therapy for high-risk neuroblastoma. Int J Cancer. 2020;147(11):3152-9.
7. Kraveka J et al. Survival outcomes in patients with high-risk neuroblastoma (HRNB) receiving eflornithine (DFMO) maintenance treatment with matched external controls: subgroup analysis on geographic region. Abstract 2046. SIOP 2024, 17-20 October, 2024.
8. Ladenstein R et al. Influence of surgical excision on the survival of patients with MYCN amplified localized neuroblastoma: a report from the HR-NBL1/SIOPEN study. Abstract O003. SIOP 2024, 17-20 October, 2024.
9. Irtan S et al. Only MIBG positive post surgery residues impact on outcomes in patients with stage 4 neuroblastoma: results from the HR-NBL1/SIOPEN trial. Abstract O032. SIOP 2024, 17-20 October, 2024.
10. Romano A et al. Early and long-term ototoxicity noted in children due to platinum compounds: prevalence and risk factors. Biomedicines. 2023;11(2):261.
11. Streefkerk N et al. Optimal conditions for and challenges in adequate interpretation of audiological testing of cisplatin-exposed children younger than 5 years with oto-acoustic emissions, based on international consensus. Abstract 737. SIOP 2024, 17-20 October, 2024.
12. Freyer DR et al. Effects of sodium thiosulfate versus observation on development of cisplatin-induced hearing loss in children with cancer (ACCL0431): a multicentre, randomised, controlled, open-label, phase 3 trial. Lancet Oncol. 2017;18(1):63-74. Erratum in: Lancet Oncol. 2017;18(6):e
13. Brock PR et al. Sodium thiosulfate for protection from cisplatin-induced hearing loss. N Engl J Med. 2018;378(25):2376-85.
14. Lopez-Cortes A et al; BENCHISTA Project Working Group. Cancer data quality and harmonization in Europe: the experience of the BENCHISTA Project – international benchmarking of childhood cancer survival by stage. Front Oncol. 2023;13:1232451. Erratum in: Front Oncol. 2024;14:1397101.
