Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2024;89(3):230-238.
doi: 10.1159/000533972. Epub 2023 Sep 13.

Immunotherapy for Gestational Trophoblastic Neoplasia: A New Paradigm

Inge O Baas  1 , Anneke M Westermann  2 , Benoit You  3 , Pierre-Adrien Bolze  3 , Michael Seckl  4 , Ehsan Ghorani  4
Affiliations
Review

Immunotherapy for Gestational Trophoblastic Neoplasia: A New Paradigm

Inge O Baas et al. Gynecol Obstet Invest. 2024.

Abstract

Background: Immune checkpoint immunotherapy (CPI) targeting programmed cell death 1 (PD-1)/ligand (PD-L1) has been shown to be an effective treatment for gestational trophoblastic neoplasia (GTN). This includes those with multidrug resistance, ultra-high-risk disease, and epithelioid trophoblastic tumour/placental site trophoblastic tumour subtypes that are inherently chemotherapy resistant, but there is also emerging evidence in low-risk disease.

Objectives: We set out to generate an overview of the current data supporting the use of CPI for GTN in both high-risk and low-risk disease and to consider future research goals and directions in order to implement CPI in current treatment guidelines.

Methods: We identified and reviewed the published data on the use of CPI agents in GTN.

Outcome: 133 patients were identified who had been treated with CPI for GTN with pembrolizumab (23), avelumab (22), camrelizumab (57), toripalimab (15), or other anti-PD-1 agents (16), of whom 118 had high-risk diseases, relapse or multi-drug resistant disease, and 15 low-risk diseases. Overall 85 patients achieved complete remission, 77 (of 118) with high-risk disease, and 8 (of 15) with low-risk disease. 1 patient with complete remission in the high-risk group developed a relapse 22 months after anti-PD-1 treatment had been stopped. Treatment was generally well tolerated across studies.

Conclusions and outlook: The majority of high-risk patients (77/118) treated with CPI are cured and this is particularly relevant amongst those with chemotherapy resistant disease who otherwise have very limited treatment options. Priorities for future research include determining whether these agents have a role earlier in the disease course, the utility of combination with chemotherapy, and effects on future fertility. Treatment availability remains a concern due to the high price of these agents.

Keywords: (Inter)national treatment guidelines; Gestational trophoblastic neoplasia; Immune checkpoint immunotherapy; Multidrug resistance.

PubMed Disclaimer

Conflict of interest statement

The authors declare no conflict of interest.

References

    1. Bolze PA, Riedl C, Massardier J, Lotz JP, You B, Schott AM, et al. Mortality rate of gestational trophoblastic neoplasia with a FIGO score of ≥13. Am J Obstet Gynecol. 2016 Mar;214(3):390.e1–8. - PubMed
    1. Savage P, Winter M, Parker V, Harding V, Sita-Lumsden A, Fisher RA, et al. Demographics, natural history and treatment outcomes of non-molar gestational choriocarcinoma: a UK population study. BJOG. 2020 Aug;127(9):1102–7. - PubMed
    1. Schmid P, Nagai Y, Agarwal R, Hancock B, Savage PM, Sebire NJ, et al. Prognostic markers and long-term outcome of placental-site trophoblastic tumours: a retrospective observational study. Lancet. 2009;374(9683):48–55. - PubMed
    1. Froeling FEM, Ramaswami R, Papanastasopoulos P, Kaur B, Sebire NJ, Short D, et al. Intensified therapies improve survival and identification of novel prognostic factors for placental-site and epithelioid trophoblastic tumours. Br J Cancer. 2019 Mar;120(6):587–94. - PMC - PubMed
    1. Frijstein MM, Lok CAR, van Trommel NE, Ten Kate-Booij MJ, Massuger LFAG, van Werkhoven E, et al. Management and prognostic factors of epithelioid trophoblastic tumors: results from the international society for the study of trophoblastic diseases database. Gynecol Oncol. 2019 Feb;152(2):361–7. - PubMed

Substances