Flores-Sanchez Jose D, Perez-Chadid Daniela A, Diaz-Coronado Rosdali Y, Hernandez-Broncano Eddy, Ugas-Charcape Carlos F, Ramirez Alberto, Racchumí-Vela Augusto E, Boop Frederick A, Preguntegui Ivethe
1Departments of Pediatric Neurosurgery and.
2Faculty of Medicine, Universidad CES, Medellín, Antioquia, Colombia.
J Neurosurg Pediatr. 2023 Jun 30;32(4):395-403. doi: 10.3171/2023.5.PEDS2365. Print 2023 Oct 1.
The objectives of this study were to determine the overall survival (OS) and event-free survival (EFS) rates of patients with medulloblastoma treated in a national pediatric hospital in Peru, as well as to identify demographic, clinical, imaging, postoperative, and histopathological characteristics and prognostic factors associated with OS and EFS.
The authors conducted a retrospective study analyzing information from the medical records of children with a diagnosis of medulloblastoma who underwent surgical treatment at the Instituto Nacional de Salud del Niño-San Borja, a public hospital in Lima, Peru, from 2015 to 2020. Clinical-epidemiological variables, degree of disease extension, risk stratification, extent of resection, postoperative complications, status of oncological treatment received, histological subtype, and neurological sequelae were taken into account. The Kaplan-Meier method and Cox regression analysis were used to estimate OS, EFS, and prognostic factors.
Of the 57 children evaluated with complete medical records, only 22 children (38.6%) underwent complete oncological treatment. OS was 37% (95% CI 0.25-0.55) at 48 months. EFS was 44% (95% CI 0.31-0.61) at 23 months. High-risk stratification-meaning patients with ≥ 1.5 cm2 of residual postoperative tumor, those younger than 3 years, those with disseminated disease (HR 9.69, 95% CI 1.40-67.0, p = 0.02), and those who underwent subtotal resection (HR 3.78, 95% CI 1.09-13.2, p = 0.04)-was negatively associated with OS. Failure to receive complete oncological treatment was negatively associated with OS (HR 20.0, 95% CI 4.84-82.6, p < 0.001) and EFS (HR 7.82, 95% CI 2.47-24.7, p < 0.001).
OS and EFS of patients with medulloblastoma in the author's milieu are below those reported in developed countries. Incomplete treatment and treatment abandonment in the authors' cohort were also high compared with high-income country statistics. Failure to complete oncological treatment was the most important factor associated with poor prognosis, both in terms of OS and EFS. High-risk patients and subtotal resection were negatively associated with OS. Interventions are needed to promote the completion of adjuvant oncological therapy for medulloblastoma in the disadvantaged Peruvian population.
本研究的目的是确定在秘鲁一家国立儿科医院接受治疗的髓母细胞瘤患者的总生存率（OS）和无事件生存率（EFS），并确定与OS和EFS相关的人口统计学、临床、影像学、术后及组织病理学特征和预后因素。
作者进行了一项回顾性研究，分析了2015年至2020年在秘鲁利马的一家公立医院——国立儿童健康研究所-圣博尔哈接受手术治疗的诊断为髓母细胞瘤的儿童的病历信息。考虑了临床流行病学变量、疾病扩展程度、风险分层、切除范围、术后并发症、接受的肿瘤治疗状态、组织学亚型和神经后遗症。采用Kaplan-Meier法和Cox回归分析来估计OS、EFS和预后因素。
在57名有完整病历评估的儿童中，只有22名儿童（38.6%）接受了完整的肿瘤治疗。48个月时的OS为37%（95%CI 0.25 - 0.55）。23个月时的EFS为44%（95%CI 0.31 - 0.61）。高危分层——即术后残留肿瘤≥1.5 cm²的患者、3岁以下的患者、有播散性疾病的患者（HR 9.69，95%CI 1.40 - 67.0，p = 0.02）以及接受次全切除的患者（HR 3.78，95%CI 1.09 - 13.2，p = 0.04）——与OS呈负相关。未接受完整的肿瘤治疗与OS（HR 20.0，95%CI 4.84 - 82.6，p < 0.00）和EFS（HR 7.82，95%CI 2.47 - 24.7，p < 0.001）呈负相关。
作者所在环境中髓母细胞瘤患者的OS和EFS低于发达国家报告的水平。与高收入国家的统计数据相比，作者队列中的治疗不完整和治疗放弃情况也很高。未完成肿瘤治疗是与OS和EFS预后不良相关的最重要因素。高危患者和次全切除与OS呈负相关。需要采取干预措施，以促进秘鲁弱势群体中髓母细胞瘤辅助肿瘤治疗的完成。
