Chris O Brien Lifehouse, Sydney, Australia.
University of Sydney, Sydney, Australia.
BJU Int. 2022 Jun;130 Suppl 1:5-16. doi: 10.1111/bju.15648. Epub 2022 Mar 31.
To report treatment patterns and survival outcomes of patients with relapsed and refractory metastatic germ cell tumours (GCTs) treated with high-dose chemotherapy (HDCT) and autologous stem-cell transplantation in low-volume specialized centres within the widely dispersed populations of Australia and New Zealand between 1999 and 2019.
We conducted a retrospective analysis of 111 patients across 13 institutions. Patients were identified from the Australasian Bone Marrow Transplant Recipient Registry. We reviewed treatment regimens, survival outcomes, deliverability and toxicities. Primary endpoints included overall (OS) and progression-free survival (PFS). Cox proportional hazards models were used to test the association of survival outcomes with patient and treatment factors.
The median (range) age was 30 (14-68) years and GCT histology was non-seminomatous in 84% of patients. International Prognostic Factors Study Group (IPFSG) prognostic risk category was very low/low, intermediate, high and very high in 18%, 36%, 25% and 21% of patients, respectively. Salvage conventional-dose chemotherapy (CDCT) was administered prior to HDCT in 59% of patients. Regimens included paclitaxel, ifosfamide, carboplatin and etoposide (50%), carboplatin and etoposide (CE; 28%), carboplatin, etoposide and ifosfamide (CEI; 6%), carboplatin, etoposide and cyclophosphamide (CEC; 5%), CEC-paclitaxel (6%) and other (5%). With a median follow-up of 4.4 years, the 1-, 2- and 5-year PFS rates were 62%, 57% and 52%, respectively, and OS rates were 73%, 65% and 61%, respectively. There were five treatment-related deaths. Progression on treatment occurred in 17%. In a univariable analysis, worse International Germ Cell Cancer Collaborative Group (IGCCCG) and IPFSG prognostic groups were associated with inferior survival outcomes. An association of inferior survival was not found with the number of high-dose cycles received nor when HDCT was delivered after salvage CDCT.
This large dual-national registry-based study reinforces the efficacy and deliverability of HDCT for relapsed and refractory metastatic GCT in low-volume specialized centres in Australia and New Zealand, with survival outcomes comparable to those found in international practice.
报告 1999 年至 2019 年间，在澳大利亚和新西兰分布广泛的低容量专业中心，采用大剂量化疗（HDCT）和自体干细胞移植治疗复发性和难治性转移性生殖细胞瘤（GCT）患者的治疗模式和生存结果。
我们对 13 家机构的 111 名患者进行了回顾性分析。患者从澳大拉西亚骨髓移植受者登记处确定。我们回顾了治疗方案、生存结果、可交付性和毒性。主要终点包括总生存期（OS）和无进展生存期（PFS）。Cox 比例风险模型用于检验生存结果与患者和治疗因素的关系。
中位（范围）年龄为 30（14-68）岁，84%的患者 GCT 组织学为非精原细胞瘤。国际预后因素研究组（IPFSG）预后风险类别分别为极低/低、中、高和极高，分别占 18%、36%、25%和 21%。59%的患者在 HDCT 前接受了挽救性常规剂量化疗（CDCT）。方案包括紫杉醇、异环磷酰胺、卡铂和依托泊苷（50%）、卡铂和依托泊苷（CE；28%）、卡铂、依托泊苷和异环磷酰胺（CEI；6%）、卡铂、依托泊苷和环磷酰胺（CEC；5%）、CE-紫杉醇（6%）和其他（5%）。中位随访 4.4 年后，1、2 和 5 年 PFS 率分别为 62%、57%和 52%，OS 率分别为 73%、65%和 61%。有 5 例与治疗相关的死亡。17%的患者在治疗过程中出现进展。单变量分析显示，国际生殖细胞癌协作组（IGCCCG）和 IPFSG 预后较差的患者生存结果较差。接受的大剂量周期数和挽救性 CDCT 后行 HDCT 与较差的生存结果无关。
这项大型、基于两国注册的研究证实了在澳大利亚和新西兰的低容量专业中心采用 HDCT 治疗复发性和难治性转移性 GCT 的疗效和可交付性，生存结果与国际实践相当。
