Trophoblastic Disease Centre, Weston Park Cancer Centre, Sheffield, S10 2SJ, United Kingdom.
Best Pract Res Clin Obstet Gynaecol. 2021 Jul;74:67-80. doi: 10.1016/j.bpobgyn.2021.01.006. Epub 2021 Feb 2.
Low-risk gestational trophoblastic neoplasia (GTN), defined as FIGO/WHO score 0-6, is highly curable with an overall survival rate, which is approximately 100%. For most low-risk GTN patients, first-line single-agent chemotherapy with either methotrexate or actinomycin-D is recommended with overall complete human chorionic gonadotrophin (hCG) response rates of 60%-90% in mostly retrospective, non-randomised studies. The few randomised trials that exist are not appropriately powered or designed to define the optimal first-line treatment. Approximately 25%-30% of low-risk patients will develop resistance to initial single-agent chemotherapy with an increase in the FIGO score, a diagnosis of choriocarcinoma, higher pre-treatment hCG and the presence of metastatic disease being associated with an increase in the risk of resistance. The optimal treatment of patients scoring WHO 5 and 6 remains poorly defined given that approximately 70%-80% of these patients develop resistance to first-line single-agent chemotherapy, and there is an urgent need to refine the FIGO/WHO scoring system so that these patients can be identified for more intensive therapy from the outset. Despite this, almost all low-risk patients who experience treatment failure with first-line monotherapy will be cured with either sequential single-agent chemotherapy or multiagent chemotherapy with or without surgery. Given the associated increased short and longer-term toxicities associated with multi-agent chemotherapy, promising strategies to reduce the exposure of women to combination chemotherapy in low-risk disease have been investigated, including the use of carboplatin and immune check-point inhibitors. Further evaluation is required to define optimal patient selection, particularly with the use of immunotherapeutic agents given their significant increased costs and lack of longer-term safety data. Although there is a clear need to revise the FIGO/WHO (2000) scoring system, consistent international use of this is recommended to facilitate the comparison of data along with future focus in the development of international collaborative translational and clinical research, including randomised controlled trials.
低危型妊娠滋养细胞肿瘤（GTN）定义为 FIGO/WHO 评分 0-6，整体存活率约为 100%，具有高度可治愈性。对于大多数低危型 GTN 患者，推荐使用甲氨蝶呤或放线菌素-D 进行一线单药化疗，大多数回顾性、非随机研究中，总完全人绒毛膜促性腺激素（hCG）反应率为 60%-90%。目前存在的少数随机试验没有足够的效力或设计来确定最佳的一线治疗方法。大约 25%-30%的低危患者会对初始单药化疗产生耐药性，FIGO 评分升高、绒毛膜癌的诊断、治疗前 hCG 升高以及转移性疾病的存在与耐药风险的增加相关。鉴于大约 70%-80%的这些患者对一线单药化疗产生耐药性，对于 WHO 5 和 6 评分的患者，最佳治疗方法仍未得到明确界定，因此迫切需要改进 FIGO/WHO 评分系统，以便从一开始就能为这些患者提供更强化的治疗。尽管如此，几乎所有在一线单药治疗中失败的低危患者，如果使用序贯单药化疗或联合化疗（有或没有手术）治疗，都将被治愈。鉴于联合化疗相关的短期和长期毒性增加，已经研究了一些有前途的策略来降低女性在低危疾病中接触联合化疗的风险，包括使用卡铂和免疫检查点抑制剂。需要进一步评估来确定最佳患者选择，特别是在使用免疫治疗药物时，因为这些药物的成本显著增加，且缺乏长期安全性数据。虽然确实需要修订 FIGO/WHO（2000）评分系统，但建议一致采用国际标准，以促进数据的比较，并为未来国际协作转化和临床研究（包括随机对照试验）的发展提供重点。
