Pembrolizumab in gestational trophoblastic neoplasia: Systematic review and meta-analysis with sub-group analysis of potential prognostic factors

M Barcellos and A Braga and MM Rech and SA de Oliveira and JM Madi and SY Sun and J de Rezende and KM Elias and NS Horowitz and RS Berkowitz, CLINICS, 80, 100583 (2025).

DOI: 10.1016/j.clinsp.2025.100583

Objective To assess the performance of pembrolizumab for the treatment of Gestational Trophoblastic Neoplasia (GTN). Methods The Medical Subject Headings related to immunotherapy/pembrolizumab and GTN were used alone or in combination to retrieve relevant articles. The authors searched in EMBASE, MEDLINE/PubMed, Elsevier's Scopus, and Web of Science until November/2024. The authors included any randomized controlled trials, cohort studies, case series, and case reports focusing on pembrolizumab treatment in GTN. Meta-analysis of proportions was carried out employing a random-effects model. The meta-analysis employed the inverse variance method, with the arcsine link function for the analysis of proportional data. All analyses were performed using Stata 18. For all analyses, a p-value < 0.05 indicated statistical significance. This study was registered on PROSPERO (CRD42023493329). Results A total of 550 studies were identified after a literature search among which 15 original studies were included in the systematic review and in the meta-analysis. Pembrolizumab induced complete sustained remission in 71.59% (95% CI 53.27-84.78%; I-2 = 0.00%, H-2 = 1.00, p = 0.90) of cases. The subgroups meta-analysis showed pembrolizumab had similar performance, regardless of age (< 40 vs. >= 40-years-old, p = 0.38), GTN histopathology (Placental Site Trophoblastic Tumor PSTT, Epithelioid Trophoblastic Tumor ETT/noninvasive mole/others versus invasive mole/choriocarcinoma, p = 0.48), time from diagnosis to the beginning of immunotherapy (< 4 vs. >= 4-years, p = 0.84), pembrolizumab combined with chemotherapy (yes vs. no, p = 0.66). Conclusions Pembrolizumab seems an effective treatment for patients with high-risk GTN with chemoresistant or relapsed disease, including cases of PSTT/ETT, notwithstanding patient age, time to initiate immunotherapy and whether or not it was associated with chemotherapy.

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