Rosa Scarpitta1,#, Emiliano Cappello1,#, Alice Cangialosi1, Veronica Gori1, Giulia De Luca1,2, Giovanni Gori3, Guido Bocci1,*
Oncology Research, Vol.33, No.10, pp. 2603-2656, 2025, DOI:10.32604/or.2025.066260
- 26 September 2025
Abstract Central nervous system (CNS) tumors are the most common solid tumors in pediatric patients and the leading cause of childhood cancer-related mortality. Their rarity compared to adult cancers has made enrolling sufficient cases for clinical trials challenging. Consequently, pediatric CNS tumors were long treated with adult protocols despite distinct biological and clinical characteristics. This review examines key aspects of phase I pediatric oncology trials, including study design, primary outcomes, and pharmacological approaches, along with secondary considerations like clinical responses and ethical aspects. Firstly, we evaluated all phase I trial protocols focusing on pediatric CNS tumors… More >
Graphic Abstract