Open Access

ARTICLE

Outcomes and Toxicity of Adult Medulloblastoma Treated with Pediatric Multimodal Protocols: A Single-Institution Experience

Antonio Ruggiero^1,2,*, Dario Talloa¹, Alberto Romano¹, Giorgio Attinà¹, Stefano Mastrangelo^1,2, Palma Maurizi^1,2, Tommaso Verdolotti³, Gianpiero Tamburrini^4,5, Silvia Chiesa⁶, Rina di Bonaventura⁷, Pier Paolo Mattogno⁷, Alessandro Olivi^7,8, Alessio Albanese^7,8

1 Pediatric Oncology Unit, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, 00168, Italy
2 Department of Women and Child Health and Public Health, Università Cattolica del Sacro Cuore, Rome, 00168, Italy
3 ARC Advanced Radiology Center (ARC), Department of Oncological Radiotherapy and Hematology, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, 00168, Italy
4 Pediatric Neurosurgery, Fondazione Policlinico Universitario A. Gemelli, IRCCS, Rome, 00168, Italy
5 Department of Neuroscience, Section of Neurosurgery, Università Cattolica del Sacro Cuore, Rome, 00168, Italy
6 Gemelli Advanced Radiotherapy, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, 00168, Italy
7 Neurosurgery Unit, Department of Neurosciences, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, 00168, Italy
8 Neurosurgery Unit, Department of Neurosciences, Università Cattolica del Sacro Cuore, Rome, 00168, Italy

* Corresponding Author: Antonio Ruggiero. Email:

Oncology Research 2025, 33(12), 3855-3867. https://doi.org/10.32604/or.2025.067948

Received 16 May 2025; Accepted 20 August 2025; Issue published 27 November 2025

Abstract

Background: Adult medulloblastoma (MB) represents less than 1% of central nervous system malignancies, lacking standardized therapeutic approaches due to its rarity. This retrospective single-center analysis aimed to assess survival outcomes and treatment-associated toxicities in adult MB patients managed with pediatric-derived protocols. Methods: Eighteen patients (≥18 years) with MB treated at Fondazione Policlinico Universitario Agostino Gemelli Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) (January 1997–January 2024) were analyzed. All received craniospinal radiotherapy with posterior fossa boost, followed by adjuvant chemotherapy utilizing pediatric regimens (PNET3, PNET4, PNET5, or high-risk protocols incorporating high-dose chemotherapy with autologous stem cell rescue). Primary outcomes included overall survival (OS) and progression-free survival (PFS). Secondary analyses focused on comprehensive toxicity assessment. Results: The cohort included 11 males and 7 females (median age: 23 years). Metastatic disease was present in 6 patients (33%) at diagnosis. Histopathological distribution showed classic MB (55.5%), desmoplastic/nodular (39%), and large cell/anaplastic variants (5.5%). Molecular subgrouping (available in 6 patients) identified SHH subgroup in four cases and WNT subgroup in two. Three-year and five-year overall survival rates reached 94.5% and 88.8%, respectively. Treatment-related adverse events included grade 3–4 hematologic toxicities, clinically significant weight loss, and grade ≥3 neurological and ototoxic complications. These toxicities necessitated treatment modifications including dose adjustments, cycle delays, and occasional early discontinuation. Conclusions: Adult MB patients treated with pediatric-adapted protocols demonstrated excellent long-term survival outcomes, comparable to or surpassing historical data. Despite frequent toxicity requiring treatment modifications, these regimens proved feasible with acceptable risk-benefit profiles. These results support implementing modified pediatric protocols for adult MB management. Future multicenter investigations with larger cohorts are essential for refining risk stratification, optimizing treatment intensity, and evaluating long-term outcomes in this rare malignancy.

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