Decade-long disease, secondary malignancy, and brainstem injury outcomes in pediatric and young adult medulloblastoma patients treated with proton radiotherapy

Author:

Baliga Sujith1,Gallotto Sara2,Bajaj Benjamin2,Lewy Jacqueline2,Weyman Elizabeth2,Lawell Miranda P2,Yeap Beow Y2,Ebb David E3,Huang Mary3,Caruso Paul4,Perry Alisa2,Jones Robin M5,MacDonald Shannon M2,Tarbell Nancy J2,Yock Torunn I2

Affiliation:

1. Department of Radiation Oncology, The Ohio State University Wexner Medical Center, Columbus, Ohio, USA

2. Department of Radiation Oncology, Massachusetts General Hospital/Harvard Medical School, Boston, Massachusetts, USA

3. Department of Pediatric Hematology-Oncology, Massachusetts General Hospital, Boston, Massachusetts, USA

4. Department of Pediatric Neuroradiology, Massachusetts General Hospital, Boston, Massachusetts, USA

5. Department of Pediatric Neurology, Massachusetts General Hospital, Boston, Massachusetts, USA

Abstract

Abstract Background Survivors of pediatric medulloblastoma experience long-term morbidity associated with the toxic effects of postoperative radiotherapy (RT). Proton RT limits radiation dose to normal tissues thereby reducing side effects of treatment while maintaining high cure rates. However, long-term data on disease outcomes and long-term effects of proton RT remain limited. Methods One hundred seventy-eight pediatric medulloblastoma patients treated with proton RT between 2002 and 2016 at the Massachusetts General Hospital comprise the cohort of patients who were treated with surgery, radiation therapy, and chemotherapy. We evaluated event-free survival (EFS), overall survival (OS), and local control using the Kaplan-Meier method. The cumulative incidence of brainstem injury and secondary malignancies was assessed. Results Median follow-up was 9.3 years. One hundred fifty-nine patients (89.3%) underwent a gross total resection (GTR). The 10-year OS for the entire cohort, standard-risk (SR), and intermediate/high-risk (IR/HR) patients was 79.3%, 86.9%, and 68.9%, respectively. The 10-year EFS for the entire cohort, SR, and IR/HR cohorts was 73.8%, 79.5%, and 66.2%. The 10-year EFS and OS for patients with GTR/NTR were 75.3% and 81.0% vs 57.7% and 61.0% for subtotal resection (STR). On univariate analysis, IR/HR status was associated with inferior EFS, while both anaplastic histology and IR/HR status were associated with worse OS. The 10-year cumulative incidence of secondary tumors and brainstem injury was 5.6% and 2.1%, respectively. Conclusions In this cohort study of pediatric medulloblastoma, proton RT was effective, and disease outcomes were comparable to historically treated photon cohorts. The incidence of secondary malignancies and brainstem injury was low in this cohort with mature follow-up.

Publisher

Oxford University Press (OUP)

Subject

Cancer Research,Neurology (clinical),Oncology

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