摘要 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.
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.
Jianzhong Yu, We Shi, Hao Li. Factors affecting the prognosis of children with medulloblastoma: A single institution retrospective analysis of 40 cases[J]. 临床转化神经科学, 2017, 3(1): 16-27.
Jianzhong Yu, We Shi, Hao Li. Factors affecting the prognosis of children with medulloblastoma: A single institution retrospective analysis of 40 cases. Translational Neuroscience and Clinics, 2017, 3(1): 16-27.
20170512093758 Table 1 Characteristics of the patients with medulloblastoma (n = 40).
20170512093838 Figure 1 Immunohistochemical analysis of YAP and GAB1 to identify medulloblastoma subtypes. (a, b) The SHH subtype was positive for both (a) YAP1 and (b) GAB1. (c, d) The WNT subtype was positive for YAP1 and negative for GAB1. (e, f) The non-SHH/WNT subtype was negative for both YAP1 and GAB1. Magnification, 400×; scale bar, 50 μm.
20170512093852 Table 2 Univariate Cox proportional hazard analysis of factors associated with poor survival.
20170512093933 Figure 2 Kaplan-Meier curves of overall survival according to (a) M stage, (b) calcification, (c) cerebral fluid fistula, (d) postoperative radiotherapy, (e) postoperative chemotherapy, (f) postoperative adjuvant therapy, (g) postoperative KPS score, (h) molecular subtype. The log-rank test was used to test the survival status between groups.
20170512093942 Table 3 Univariate Cox proportional hazard analysis of factors associated with disease progression.
20170512093956 Table 4 Univariate Cox proportional hazard analysis of factors associated with medulloblastoma recurrence.
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2017, Vol. 3 
