METHODS: We analyzed the data of six boys with pathologically confirmed PEGCC between 2009 and 2021. Their clinicodemographic and histopathological characteristics as well as treatments and clinical outcomes were retrieved from their medical charts.
RESULTS: The patients\' median age was 15 (range: 12-17) years. The most common primary tumor site was the mediastinum (67%, 4/6), with one case each in the retroperitoneum (16.7%) and brain (16.7%). Except for the patient with brain PEGCC, all presented with metastasis at the time of diagnosis. The following metastatic sites were observed: the lungs (100%, 5/5), brain (3/5, 60%), liver (3/5, 60%), kidneys (2/5, 40%), and spleen (1/5, 20%). Most patients had dry cough, dyspnea, and hemoptysis at initial presentation, likely due to lung metastasis. Serum human chorionic gonadotropin (HCG) levels were highly elevated in all patients. All patients received platinum-based cytotoxic chemotherapy. The patient with brain choriocarcinoma underwent surgical tumor resection; all others underwent only surgical biopsy. Strong positive PD-L1 immunohistochemical staining was noted for two patients. One patient received the PD-L1 inhibitor pembrolizumab and achieved a good response. Our cohort\'s 1-year survival rate was 33.3%, with a median survival of 4.34 months. Serum HCG levels remained normal in the two survivors during follow-up visits.
CONCLUSIONS: The poor response to current platinum-based chemotherapy remains a major challenge in the management of pediatric PEGCC. Adding pembrolizumab to a conventional chemotherapy regimen may improve the outcomes in boys with PEGCC.
方法:我们分析了2009年至2021年间经病理证实为PEGCC的六个男孩的数据。他们的临床人口统计学和组织病理学特征以及治疗和临床结果从他们的医学图表中检索。
结果:患者的中位年龄为15岁(范围:12-17岁)。最常见的原发肿瘤部位是纵隔(67%,4/6),腹膜后(16.7%)和脑(16.7%)各1例。除了脑部PEGCC患者,均在诊断时出现转移。观察到以下转移部位:肺(100%,5/5),大脑(3/5,60%),肝脏(3/5,60%),肾脏(2/5,40%),和脾脏(1/5,20%)。大多数病人有干咳,呼吸困难,初次报告时咯血,可能是由于肺转移。所有患者的血清人绒毛膜促性腺激素(HCG)水平均高度升高。所有患者均接受基于铂的细胞毒性化疗。患有脑绒毛膜癌的患者接受了手术肿瘤切除术;所有其他人仅接受了手术活检。两名患者的PD-L1免疫组织化学染色呈强阳性。一名患者接受了PD-L1抑制剂派姆单抗,并获得了良好的反应。我们队列的1年生存率为33.3%,中位生存期为4.34个月。在随访期间,两名幸存者的血清HCG水平保持正常。
结论:目前以铂类为基础的化疗反应不佳仍然是儿科PEGCC治疗的主要挑战。在常规化疗方案中加入派姆单抗可能会改善PEGCC男孩的预后。