Management and prognosis of ultra high-risk gestational trophoblastic neoplasia patients: a long-term retrospective...

The prognosis for patients with ultra high-risk gestational trophoblastic neoplasia (GTN) is significantly worse, and there is currently no consensus regarding the optimal treatment strategies for this specific patient population.

This study aims to investigate the clinical characteristics, treatment responses, outcomes, and prognostic risk factors in ultra high-risk GTN patients with an International Federation of Gynecology and Obstetrics (FIGO) score of 13 or higher.

Retrospective study.

Medical records of 36 GTN patients with FIGO score ⩾ 13, treated at the first affiliated hospital of Zhengzhou University, China, from January 2015 to June 2024, were retrospectively reviewed. Chi-square tests, univariate analysis, and Kaplan-Meier survival analysis were employed for data analysis.

Etoposide, methotrexate, actinomycin D, cyclophosphamide, and vincristine (EMA-CO) was the most commonly used chemotherapy regimen (17/36, 47.2%). Twenty-one patients responded well to the initial chemotherapy regimen and achieved complete remission (CR). One patient was switched to salvage chemotherapy due to resistance to the initial regimen and subsequently achieved CR. Four patients experienced relapse; of these, three attained CR. The median follow-up time was 46 months (range 12-85 months). The CR rate was 69.4% (25/36). A total of 20 patients (55.6%) received adjuvant treatments, including surgery and radiotherapy. Stage IV disease, liver metastases, uncommon distant metastatic sites, and ⩾3 metastatic sites were significant predictors of mortality.

In our study, the overall CR rate for ultra high-risk GTN patients with FIGO score ⩾ 13 was 69.4%. Five patients (45.5%) experienced early mortality. All patients with brain metastases who received chemotherapy in conjunction with whole-brain or stereotactic radiotherapy achieved CR. Immune checkpoint inhibitors demonstrated potential efficacy in treating chemotherapy-resistant GTN.