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Introduction <p>Gestational trophoblastic neoplasia (GTN) is a highly curable malignancy arising from placental trophoblasts, yet a small subset of patients develops multidrug resistance with limited therapeutic options. The discovery of high programmed death-ligand&#xa0;1 (PD-L1) expression across trophoblastic tumors has provided a compelling biological rationale for immunotherapy, particularly immune checkpoint blockade targeting the PD-1/PD-L1 axis.</p> Objective <p>To summarize current evidence on immunotherapy in GTN, integrating biological foundations, clinical experiences, and ongoing clinical trials, and to discuss future perspectives toward individualized, fertility-preserving management.</p> Methods <p>A narrative review was conducted according to structured PRISMA-based principles. Literature was retrieved from PubMed, Scopus, and Web of Science from 2000 to 2025 using predefined descriptors related to GTN and immunotherapy. Eligible studies included clinical trials, case series, case reports, and translational research addressing immune checkpoint inhibitors in GTN.</p> Results <p>GTN exhibits high PD-L1 expression, mirroring the immune-privileged nature of the placenta. Checkpoint inhibitors alone, such as pembrolizumab, avelumab, or the combination of camrelizumab plus apatinib (that potently suppresses the kinase activity of vascular endothelial growth factor&#xa0;2), have demonstrated complete and durable responses in approximately 70–80% of patients with multidrug-resistant GTN, with acceptable safety and preserved fertility.</p> Conclusions <p>Immunotherapy has expanded therapeutic GTN, transforming refractory disease as a result of its immune responsiveness. Checkpoint inhibition not only achieves high remission rates but also offers fertility preservation and long-term survivorship. The future challenge lies in optimizing combination strategies, refining biomarkers, and ensuring equitable global access to these emerging treatments.</p>

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Checkpoint Inhibition in Gestational Trophoblastic Neoplasia: A Narrative Review on the Reawakening of Antitumor Immunity

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年2月2日

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  • Marcio Bezerra Barcellos,
  • Antônio Braga,
  • Raphael Alevato,
  • Sully Turon,
  • Gustavo Yano Callado,
  • Solange Artimos,
  • Sue Yazaki Sun,
  • Jorge Rezende-Filho,
  • Edward Araujo Júnior,
  • Andreia Cristina de Melo,
  • Ross S. Berkowitz,
  • Neil S. Horowitz,
  • Roberta Granese

摘要

Introduction

Gestational trophoblastic neoplasia (GTN) is a highly curable malignancy arising from placental trophoblasts, yet a small subset of patients develops multidrug resistance with limited therapeutic options. The discovery of high programmed death-ligand 1 (PD-L1) expression across trophoblastic tumors has provided a compelling biological rationale for immunotherapy, particularly immune checkpoint blockade targeting the PD-1/PD-L1 axis.

Objective

To summarize current evidence on immunotherapy in GTN, integrating biological foundations, clinical experiences, and ongoing clinical trials, and to discuss future perspectives toward individualized, fertility-preserving management.

Methods

A narrative review was conducted according to structured PRISMA-based principles. Literature was retrieved from PubMed, Scopus, and Web of Science from 2000 to 2025 using predefined descriptors related to GTN and immunotherapy. Eligible studies included clinical trials, case series, case reports, and translational research addressing immune checkpoint inhibitors in GTN.

Results

GTN exhibits high PD-L1 expression, mirroring the immune-privileged nature of the placenta. Checkpoint inhibitors alone, such as pembrolizumab, avelumab, or the combination of camrelizumab plus apatinib (that potently suppresses the kinase activity of vascular endothelial growth factor 2), have demonstrated complete and durable responses in approximately 70–80% of patients with multidrug-resistant GTN, with acceptable safety and preserved fertility.

Conclusions

Immunotherapy has expanded therapeutic GTN, transforming refractory disease as a result of its immune responsiveness. Checkpoint inhibition not only achieves high remission rates but also offers fertility preservation and long-term survivorship. The future challenge lies in optimizing combination strategies, refining biomarkers, and ensuring equitable global access to these emerging treatments.