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Angela Mastronuzzi Highlights Link Between Early Radiologic Response and Prognosis in Children with NF1
Aug 15, 2025, 11:04

Angela Mastronuzzi Highlights Link Between Early Radiologic Response and Prognosis in Children with NF1

Angela Mastronuzzi, Head of Neuro-Oncology Unit at Bambino Gesù Children’s Hospital and President of AIEOP, shared a post on LinkedIn about a paper by Claudia Santoro et al. published on Journal of Neuro-Oncology:

” ‘Real-world experience with selumetinib in children with neurofibromatosis type 1: a multicentric retrospective study’

We present novel data of a correlation between early radiological response (at C6) and the response at the end of observation, a clinical benefit in terms of pain control in stable lesions, CBR at C24 confirming the favorable course of treated patients and the evaluation of major responses.

Further studies are warranted to refine our understanding of tumor growth dynamics, optimal treatment strategies and clinical benefit assessment beside and beyond the radiological tumor response. If confirmed, these results could have significant clinical implications for the management of NF1-associated PNs.

Our findings suggest that the response after 6 and 12 selumetinib cycles may predict long-term outcomes, providing clinicians with an early indicator for therapeutic decision-making.”

Title: Real-world experience with selumetinib in children with neurofibromatosis type 1: a multicentric retrospective study

Authors: Claudia Santoro, Mariachiara Servedio, Maria Cristina Diana, Irene Russo, Elena Arkhangelskaya, Gianluca Piccolo, Andrea Santangelo, Angela Mastronuzzi, Antonella Cacchione, May El Hachem, Carmela Russo, Mario Cirillo, Ilaria Cecconi, Antonio Grasso, Mariateresa Loiotine, Nicola Santoro, Mariachiara Resta, Carmela De Meco, Consolata Soddu, Eugenia Spreafico, Bartolomeo Rossi, Chiara Fossati, Chiara Leoni, Silverio Perrotta, Teresa Perillo

Read the full article on Journal of Neuro-Oncology.

Angela Mastronuzzi

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