Novel therapeutic approaches for pediatric diencephalic tumors: improving functional outcomes

Julia V Cockle; Elizabeth A Corley; Bassel Zebian; Samantha Hettige; Sucheta J Vaidya; Paola Angelini; Joanna Stone; R Jane Leitch; Assunta Albanese; Henry C Mandeville; Fernando Carceller; Lynley V Marshall

doi:10.3389/fonc.2023.1178553

Novel therapeutic approaches for pediatric diencephalic tumors: improving functional outcomes

Front Oncol. 2023 Oct 10:13:1178553. doi: 10.3389/fonc.2023.1178553. eCollection 2023.

Authors

Julia V Cockle^{1

2}, Elizabeth A Corley³, Bassel Zebian⁴, Samantha Hettige⁵, Sucheta J Vaidya¹, Paola Angelini^{1

2}, Joanna Stone¹, R Jane Leitch⁶, Assunta Albanese^{1

7}, Henry C Mandeville^{1

2

8}, Fernando Carceller^{1

2

3}, Lynley V Marshall^{1

2

3}

Affiliations

¹ Department of Neuro-oncology, Children and Young People's Unit, The Royal Marsden National Health Service (NHS) Foundation Trust, London, United Kingdom.
² Division of Clinical Studies, The Institute of Cancer Research, London, United Kingdom.
³ Pediatric and Adolescent Oncology Drug Development Team, Children and Young People's Unit, The Royal Marsden National Health Service (NHS) Foundation Trust, London, United Kingdom.
⁴ Department of Neurosurgery, Kings College Hospital National Health Service (NHS) Trust, London, United Kingdom.
⁵ Atkinson Morley Neurosurgery Centre, St George's University Hospital National Health Service (NHS) Foundation Trust, London, United Kingdom.
⁶ Department of Ophthalmology, Epsom and St Hellier University Hospitals Trust, Carshalton, United Kingdom.
⁷ Department of Pediatric Endocrinology, The Royal Marsden National Health Service (NHS) Foundation Trust, London, United Kingdom.
⁸ Department of Radiotherapy, The Royal Marsden National Health Service (NHS) Foundation Trust, London, United Kingdom.

Abstract

Pediatric diencephalic tumors represent a histopathologically and molecularly diverse group of neoplasms arising in the central part of the brain and involving eloquent structures, including the hypothalamic-pituitary axis (HPA), optic pathway, thalamus, and pineal gland. Presenting symptoms can include significant neurological, endocrine, or visual manifestations which may be exacerbated by injudicious intervention. Upfront multidisciplinary assessment and coordinated management is crucial from the outset to ensure best short- and long-term functional outcomes. In this review we discuss the clinical and pathological features of the neoplastic entities arising in this location, and their management. We emphasize a clear move towards 'function preserving' diagnostic and therapeutic approaches with novel toxicity-sparing strategies, including targeted therapies.

Keywords: Langerhans cell histiocytosis; craniopharyngioma; functional outcomes; germ cell tumor; glioma; molecularly targeted therapies; pediatric diencephalic tumors.

Publication types

Review

Grants and funding

LM is funded by The Oak Foundation via the Royal Marsden Cancer Charity (RMCC) (grant number: OCAY-17-832-OFI); EAC and FC are funded by The Giant Pledge via RMCC; we acknowledge funding to the Paediatric Drug Development and Neuro-Oncology Teams at The Royal Marsden from The Hall Hunter Foundation via RMCC and the UK’s Experimental Cancer Medicines Centre (ECMC) paediatric network grant (grant number: ECMCQQR-2022/100020), and funding support from the National Institute of Health Research (NIHR) Biomedical Research Centre at The Royal Marsden Hospital and The Institute of Cancer Research, London, United Kingdom.