Department of Pediatrics, Institut d'Hemato-oncologie Pediatrique, Lyon, France.
Pediatr Blood Cancer. 2014 Feb;61(2):253-9. doi: 10.1002/pbc.24730. Epub 2013 Aug 12.
Some children with extracranial germ cell tumors (GCT) relapse after or do not respond to first-line treatment combining chemotherapy and surgery, of whom very few experience long-term survival despite multimodal salvage treatment.
This prospective study, part of the French TGM95 Protocol for non-seminomatous GCT (NSGCT), included 19 (7%) children with malignant refractory or recurrent extracranial NSGCT who were studied to identify prognostic factors and determine the best salvage treatment.
At the end of the first-line treatment, 10 and 9 children were in complete and incomplete remission, respectively. Events occurred within 2 years (5-23 months) after initial diagnosis. A progression was observed in 13 patients at least in one site initially involved. Two patients had a purely biological relapse (increase in isolated markers), and four patients had a purely metastatic relapse (brain location in three cases). After salvage treatment combining surgery and various types of chemotherapy (including high-dose chemotherapy (HDCT) in 10 cases), the 5-year event-free survival and overall survival rates were of 26% (95%CI: 9.6-46.8%) and 32% (95%CI: 12.9-52.2%), respectively. Patients who underwent complete surgery (or without any detectable tumor) had higher survival rate than patients who underwent partial surgery or for whom surgery was not feasible (P = 0.0003) at first relapse while this rate was similar between patients treated or not with HDCT.
In pediatric recurrent or refractory NSGCT, complete excision of the tumor appears essential. The role of HDCT remains debated.
一些患有颅外生殖细胞瘤(GCT)的儿童在接受化疗和手术联合一线治疗后复发或无反应,尽管采用多模式挽救治疗,其中极少数患者能长期存活。
本前瞻性研究是法国 TGM95 非精原细胞瘤生殖细胞肿瘤(NSGCT)方案的一部分,纳入了 19 名(7%)患有恶性难治性或复发性颅外 NSGCT 的儿童,旨在确定预后因素并确定最佳挽救治疗方案。
在一线治疗结束时,分别有 10 名和 9 名儿童处于完全缓解和不完全缓解。事件发生在初始诊断后 2 年内(5-23 个月)。在至少一个最初受累部位观察到 13 名患者的进展。两名患者仅有单纯的生物学复发(孤立标志物增加),四名患者仅有单纯的转移性复发(三种情况下为脑部)。在挽救治疗中,联合手术和各种类型的化疗(包括 10 例大剂量化疗(HDCT))后,5 年无事件生存率和总生存率分别为 26%(95%CI:9.6-46.8%)和 32%(95%CI:12.9-52.2%)。首次复发时行完全手术(或无任何可检测到的肿瘤)的患者比行部分手术或手术不可行的患者生存率更高(P = 0.0003),而在接受或未接受 HDCT 治疗的患者中,生存率相似。
在儿科复发性或难治性 NSGCT 中,肿瘤的完全切除似乎至关重要。HDCT 的作用仍存在争议。