Abstract
Few studies have investigated the factors associated with the prognosis of children with medulloblastoma. This retrospective observational study evaluated the association of molecular subtype, clinical characteristics, and pathological types with the outcomes of children with medulloblastoma. This study included 40 patients with medulloblastoma who underwent surgical resection at the Affiliated Children's Hospital of Fudan University between January 2004 and June 2014. The primary outcome was overall survival (OS). Risk factors associated with survival, disease progression, and recurrence were analyzed by univariate Cox regression analysis; the identified significant risk factors were further analyzed by Kaplan-Meier survival curves. Immunohistochemistry analysis of Yes-associated protein 1 (YAP1) and GRB2-associated protein 1 (GAB1) was used for medulloblastoma subtype identification; 20% of tumors were the WNT subtype, and 32.5% were the SHH subtype, with the remainder being non-SHH/WNT. Factors associated with OS included M stage, calcification, cerebrospinal fluid fistula, postoperative treatment (radiotherapy, chemotherapy, or both), postoperative Karnofsky Performance Scale score, and molecular subtype (P < 0.044). Patients with the WNT subtype had better survival outcomes (hazard ratio [HR] = 0.16, 95% confidence interval [CI]: 0.05–0.58). Number of symptoms, M stage, and postoperative radiotherapy were associated with disease progression (P ≤ 0.033). The risk of recurrence increased with advanced M stage (HR = 30.71; 95% CI: 3.92—240.44, P = 0.001). Patients receiving both chemotherapy and radiotherapy were less likely to have a recurrence (P = 0.040). Molecular subtyping of medulloblastoma was more predictive of survival than histopathology in patients undergoing adjuvant therapy.