Trial results for proton radiotherapy in pediatric brain tumors were posted on ClinicalTrials.gov on 2025-06-03. The study reported a 3-year local disease control probability of 89.9% for participants receiving the treatment.
Background
Patients with brain tumors often receive radiation to prevent tumor growth. Standard radiation, while effective, can damage normal surrounding tissue, leading to side effects. Proton radiotherapy is an alternative form of radiation designed to spare normal tissues while precisely targeting the tumor or tumor bed. Proton techniques are reported to irradiate 2-3 times less normal tissue compared to standard radiation. This therapy has been utilized in the treatment of other cancers, with research suggesting potential benefits in tissue sparing.
Trial design
This completed Phase 2 study enrolled 100 participants. The trial investigated proton radiotherapy for various pediatric brain tumor conditions, including Brain Tumor, Low Grade Glioma, Astrocytoma, Ependymoma, and Ganglioglioma. The intervention studied was proton radiotherapy, focusing on its efficacy and safety in pediatric patients requiring partial brain irradiation.
Key results
Key measurements and analyses from the trial include:
- Endocrine Dysfunction:
- The 3-year cumulative incidence of endocrine dysfunction was 14.5% (95% Confidence Interval: 8.3% to 22.3%).
- The 5-year cumulative incidence was 20.2% (95% Confidence Interval: 12.7% to 28.9%).
- Neurocognitive Sequelae:
- Mean scores on a scale for neurocognitive sequelae were 99.2 (Standard Deviation 14.8) and 100.3 (Standard Deviation 16.2).
- The mean difference in neurocognitive sequelae was 1.1 (95% Confidence Interval: -2.6 to 4.9), with a p-value of 0.543.
- Disease Control:
- The 3-year local disease control probability was 89.9% (95% Confidence Interval: 83.1% to 94.9%).
- The 5-year local disease control probability was 85.9% (95% Confidence Interval: 78.2% to 91.9%).
- The 3-year distant disease control probability was 97.0% (95% Confidence Interval: 92.1% to 99.2%).
- Cumulative Incidence of Grade 3+ Toxicities:
- The cumulative incidence of Grade 3+ toxicities was 12.2% and 16.3% of participants.
- Cumulative Incidence of Ototoxicity:
- The cumulative incidence of ototoxicity was 12.5% of participants.
What this means
The results suggest that proton radiotherapy offers favorable disease control rates for pediatric brain tumors, with a 3-year local disease control probability of nearly 90% and a 3-year distant disease control probability over 95%. While endocrine dysfunction and ototoxicity were observed, the rates provide a benchmark for expected side effects. The neurocognitive sequelae analysis showed a mean difference of 1.1 with a p-value of 0.543, indicating no statistically significant difference in neurocognitive outcomes within the study. These findings support proton radiotherapy as a viable treatment option, potentially mitigating long-term side effects due to its tissue-sparing properties.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT01288235, titled "Proton Radiotherapy for Pediatric Brain Tumors Requiring Partial Brain Irradiation," were posted on 2025-06-03 on clinicaltrials.gov.