Real-world management and long-term outcomes in adolescent, young adult, and adult medulloblastoma: Experience from a monocentric series with multimodal and targeted approaches Alberto Bosio, Marta Maccari, Marta Padovan, Mario Caccese, Maital Bolshinsky, et al. Neuro Oncology Practice, 2026 Abstract Background Medulloblastoma (MB) is an exceedingly rare malignancy in adolescents and young adults (AYA) and adults. This study aims to describe clinical characteristics, treatments, including the use of targeted therapy, outcomes, and long-term sequelae in a single-center cohort of AYA/adult MB patients. Methods We retrospectively analyzed MB patients aged ≥16 years treated at Veneto Institute of Oncology, Padua, Italy, between 2011 and 2025. Clinical, molecular, and treatment data were collected. Treatment response was assessed using RAPNO criteria, and treatment-related toxicity according to CTCAE v5.0. Survival outcomes were estimated using the Kaplan-Meier method. Results We included 29 patients. At diagnosis, 86% were symptomatic, 55% had an ECOG performance status (PS) of 0, and desmoplastic/nodular was the most frequent histology (48%). Molecular subgrouping was available in 45%, with SHH activation as the most common subgroup (31%, 9/29 patients). All patients underwent craniospinal radiotherapy; adjuvant chemotherapy regimens included cisplatin-etoposide±cyclophosphamide (62%), cisplatin-lomustine±vincristine (21%), and intensive pediatric regimens (10%), while 2 patients (7%) did not receive chemotherapy due to comorbidities. First-line complete response was achieved in 65%. Overall, 38% experienced progression, and 24% died. Three patients received vismodegib at recurrence. Three-year PFS and OS rates were 78% and 92%. Stratified analyses indicated worse outcomes in patients with high-risk classification, subtotal resection, and lower baseline ECOG PS. Grade 3-4 toxicity occurred in 30%. Long-term complications included neurocognitive impairment (21%) and radiotherapy-induced secondary malignancies (10%). Conclusions Long-term survivorship is often achievable in AYA/adult MB, though late toxicities remain a concern. Molecular profiling supports risk stratification and therapy personalization. Long-term toxicities highlight the need for de-intensified strategies.
Targeted therapies in optic pathway gliomas Edoardo Agosti, Pier Paolo Panciani, Giuseppe Lombardi, Matthias Preusser, Giorgia De Rosa, et al. Cancer Treatment Reviews, 2026
Targeted and systemic therapies for recurrent adult ependymomas: real-world outcomes from a single institution and concise literature review Marta Maccari, Alberto Bosio, Mario Caccese, Marta Padovan, Angela Guerriero, et al. Frontiers in Oncology, 2026 Background Recurrent adult ependymomas lack standard systemic therapies and evidence on chemotherapy and targeted agents is limited. This study aimed to retrospectively evaluated outcomes of systemic therapies, including targeted combinations, in a real-world cohort. Methods Adult patients with intracranial or spinal ependymoma treated at our institution between 2013 and 2025 who received at least one systemic line at recurrence were included. Tumor response was assessed according to RANO criteria. Primary endpoints were disease control rate (DCR) and progression-free survival (PFS). Secondary endpoints were overall survival from the start of the first line treatment (OSt) and overall survival from diagnosis (OS). Results Among 47 patients, 12 received systemic therapy at recurrence. The median follow up duration for the entire cohort was 28.3 months. Temozolomide (TMZ) was the most commonly used agent (n=12). TMZ monotherapy achieved a DCR of 57% with 6- and 12-month PFS rates of 85.7% and 57.1%, respectively. Targeted therapy was administered to 7 patients: the TMZ-Lapatinib combination provided limited benefit (DCR 33%, median PFS 2.9 months), bevacizumab-based regimens showed variable efficacy; bevacizumab alone achieved a DCR of 33% with one case of prolonged stabilization (>58 months), while bevacizumab plus fotemustine yielded a PFS of 14.1 months. Treatments were generally well tolerated, with limited grade 3 toxicities. Sequential systemic therapy, up to five lines, was feasible in selected cases. Median OSt was not reached; 12-month OS was 66.7%. Conclusions This real-world analysis indicates that temozolomide is feasible and associated with disease stabilization in selected patients, while bevacizumab-based combinations showed signals of clinical activity in recurrent adult ependymomas. Prospective, biomarker-driven multicenter trials are warranted to optimize systemic strategies in this rare disease.
Tumor-related epilepsy in glioma: A multidisciplinary overview Roberto Michelucci, Giada Pauletto, Antonio Silvani, Elena Pasini, Tamara Ius, et al. Epilepsia, 2025 Seizures are a common and challenging symptom in brain tumors, affecting approximately 60% of patients. Tumor‐related epilepsy (TRE) in glioma patients requires personalized and dynamic management in a multidisciplinary environment, especially for its intricate pathophysiology and unpredictable disease evolution. This investigation provides an updated overview about the pathophysiological mechanisms and treatment options of TRE associated with gliomas, based on expert contributions belonging to different areas. By combining the most recent discoveries and expert opinions, this study seeks to provide useful advice for TRE management in glioma patients. To improve patient outcomes and quality of life, prospective, standardized, multicentric studies should be promoted to optimize TRE patient care and refine therapeutic approaches.
Bevacizumab in recurrent glioblastoma: does dose matter? Our monocentric and comparative experience Giulia Cerretti, Alberto Bosio, Giovanni Librizzi, Giovanna Pintacuda, Mario Caccese, et al. Journal of Neuro Oncology, 2025 Purpose Bevacizumab is an anti-angiogenetic treatment that can be used in patients with recurrent glioblastoma, but there are limited and controversial data on the optimal dose and schedule, associated toxicities and survival benefits of different doses. Methods A retrospective analysis of patients with recurrent IDHwt glioblastoma treated with bevacizumab at the Veneto Institute of Oncology was performed. Patients received bevacizumab in 2 different schedules (5 mg/kg or 10 mg/kg q2w), as monotherapy or in combination with chemotherapy. Results 81 patients were analyzed, 33 received bevacizumab 5 mg/Kg, 48 received bevacizumab 10 mg/Kg. Median PFS was 4 months in both patients treated with 5 mg/kg and those treated with 10 mg/kg (p-value=0.08), median OS was 5 months in patients treated with 5 mg/kg and 7 months in those treated with 10 mg/kg (p-value=0.10). There was no difference in the use of steroid therapy between the two groups. The incidence of adverse events was not statistically different. Conclusions There was no statistically significant difference in survival, PFS, response, toxicity and steroid reduction between the two different doses. These results may support the use of lower doses of the drug with comparable benefit for patients and with additional advantage in terms of health care costs.
