Abstract Objective To analyze the clinical characteristics and prognosis of pediatric intracranial malignant germ cell tumors (iMGCTs) in a single institution. Methods A retrospective analysis was conducted on the clinical data of pediatric primary iMGCTs admitted to the Medical Oncology Department, Beijing Children’ s Hospital from May 2017 to December 2024. The clinical characteristics and prognosis were summarized. Results A total of 36 patients were enrolled. The time interval from symptom onset to diagnosis was 0.2 to 40 months. Based on histopathology and tumor markers, 9 cases (25.0%) were diagnosed as germinomas, 26 cases (72.2%) were non-germinomatous germ cell tumors (NGGCT), and one was unclassifiable. All patients received comprehensive treatment including chemotherapy, radiotherapy, and surgery under the multidisciplinary treatment (MDT) model. The 3-year and 5-year overall survival (OS) rates of germinomas were both 100%, and the 3-year and 5-year event-free survival (EFS) rates were 100% and 88.9%, respectively. For NGGCT cases, the 3-year and 5-year OS rates were 95.8% and 91.3%, respectively, and the 3-year and 5-year EFS rates were both 69.2%. Patients of NGGCTs who underwent surgical resection of the tumor during first-line treatment had significantly higher EFS than those who did not (88.2% vs 33.3%, P 0.05). Among the survivors, 14 cases (38.9%) had endocrine abnormalities. One patient of NGGCT developed a second malignance (rhabdomyosarcoma) 2.5 years after treatment. Conclusion Pediatric iMGCTs have diverse clinical manifestations, requiring early identification and timely diagnosis. Even with multidisciplinary treatment, the EFS of NGGCTs remains suboptimal. Pathological components containing choriocarcinoma and absence of surgical resection during first-line therapy are poor prognostic factors, highlighting the potential importance of surgery in NGGCTs management and enhancing chemotherapy intensity for high-risk patients.The treatment of iMGCTs should aim to minimize side effects while striving for long-term survival.
Yang et al. (Tue,) studied this question.