Key Points
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Radiotherapy is integral to the management of many paediatric CNS tumours, but exposes healthy tissue to unwanted radiation that leads to acute and long-term toxicities
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Continued improvement in long-term survivorship of certain paediatric CNS tumours means that strategies to avoid radiotherapy-related toxicities can improve quality of life and overall functional status of survivors
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Proton therapy reduces the unwanted, toxicity-associated low-to-intermediate radiation dose that healthy tissue receives in modern X-ray therapy
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The improvement in dose distribution achieved with proton therapy can meaningfully affect the risk of long-term radiotherapy effects, such as secondary malignancy, cognitive toxicity, endocrinopathy, hearing loss and vasculopathic effects
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Ongoing research aims to characterize the differential relative biological effectiveness of proton therapy, which has been suggested to increase the risk of imaging changes in susceptible structures, such as the brainstem
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Despite its higher up-front costs, proton therapy has been shown to be more cost effective than X-ray therapy owing to the dramatic reduction in the excess costs of managing long-term toxicities
Abstract
Radiotherapy is an integral and highly effective aspect of the management of many paediatric CNS tumours, including embryonal tumours, astrocytic tumours and ependymal tumours. Nevertheless, continued improvements in long-term survivorship of such tumours means that radiotherapy-related toxicities that affect quality of life and overall functional status for survivors are increasingly problematic, and strategies that mitigate these adverse effects are needed. One such strategy is proton therapy, which has distinct advantages over conventional photon therapy and enables greater precision in the delivery of tumoricidal radiation doses with reduced irradiation of healthy tissues. These dose distribution advantages can translate into clinical benefits by reducing the risk of long-term adverse effects of radiotherapy, such as secondary malignancy, cognitive toxicity, endocrinopathy, hearing loss and vasculopathic effects. As the availability of proton therapy increases with the development of new proton centres, this treatment modality is increasingly being used in the management of paediatric CNS tumours. In this Review, we provide an introduction to the types of paediatric CNS tumours for which proton therapy can be considered, and discuss the available evidence that proton therapy limits toxicities and improves quality of life for patients. We will also consider uncertainties surrounding the use of proton therapy, evidence for its cost-effectiveness, and its future role in the management of paediatric CNS tumours.
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V.G. has received speaking honoraria from prIME Oncology and US Oncology. None of these activities are related to this paper. M.P.M. has served as a consultant for BMS, Cavion, Celldex, Elekta, Novartis, Novocure and Roche; has previously held stock options in Pharmacyclics where he served on the Board of Directors; and has received research funding from Cellectar, NIH and Novocure. None of these activities are related to this paper. T.I.Y. declares no competing interests.
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Gondi, V., Yock, T. & Mehta, M. Proton therapy for paediatric CNS tumours — improving treatment-related outcomes. Nat Rev Neurol 12, 334–345 (2016). https://doi.org/10.1038/nrneurol.2016.70
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DOI: https://doi.org/10.1038/nrneurol.2016.70
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