A multi-institutional retrospective study of intracranial ependymoma in children: Identification of risk factors

Purpose: The goal of this multi-institutional retrospective study of children with intracranial ependymoma was to identify risk factors associated with unfavorable overall survival (OS) and event-free survival (EFS). Patients and Methods: Clinical data, including demographics, tumor location, spread, histology, details of surgery, radiation treatment, and chemotherapy were collected. Clinical characteristics and univariate and multivariate analyses of risk factors for OS and EFS are presented. Results: Eleven U.S. institutions contributed 83 patients treated from 1987 to 1991. The OS at 5 and 7 years was 57% and 46%, and EFS at 5 and 7 years was 42% and 33%. Patients 3 years of age or younger differed from the older group by more common infratentorial location, less common gross total resection (GTR), and postoperative use of chemotherapy rather than radiation. This younger group of patients had worse survival (P < 0.01) than the older age group. Other than young age, less than GTR and World Health Organization (WHO) II grade 3 histology were significant adverse risk factors for EFS in univariate and multivariate analyses. OS shared the same adverse risk factors except for histology in multivariate analysis, which was only of borderline significance (P = 0.05). Progression at the original tumor location, present in 89% of patients, was the major pattern of tumor recurrence. Adjuvant chemotherapy in the group older than 3 years or craniospinal radiation in M₀ patients did not significantly change EFS. Conclusions: Adverse outcome in childhood intracranial ependymoma is related to age (3 years or younger), histology (grade 3), and degree of surgical resection (less than GTR). New approaches, particularly for local tumor control in younger patients, are needed to improve survival.