Despite the fact that >100,000 patients worldwide have been treated with PT, the level of published evidence to support superiority over conventional treatment remains low. It is debated that randomised control trials in this area would be inconsistent with the principle of clinical equipoise. In contrast, there is a call for level 1 evidence to justify drastic changes in patient care, particularly in light of recent reports of unexpected toxicities. In time, careful evaluation, follow-up and clinical trials will likely support the preferential use of PT in children.
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