Epidemiology, treatment patterns, and outcomes in gestational trophoblastic neoplasia: a systematic review

Current global data on epidemiology, treatment patterns, outcomes, and care pathways for patients with gestational trophoblastic neoplasia (GTN) are needed.

A systematic review included publications from Embase and Medline (interventional: 2005-2025; observational: 2021-2025) and conference abstracts (2022-2024). Benign gestational trophoblastic disease (GTD) studies, case reports, and case series were excluded. Treatment response rates and 95% CIs were calculated from aggregated data.

Of 117 included studies, 66.7% (n=78) were cohort studies; 54.7% (n=64) were conducted in Asia. GTN incidence from GTD varied from 6.2% in the UK to 16.8% across Asian studies. Methotrexate and dactinomycin were the most common real-world first-line treatments in low-risk GTN. Second-line and high-risk patients' treatment included diverse multiagent regimens and immunotherapy. Across trials, first-line treatment response with dactinomycin (82.9%) was higher than methotrexate (70.5%); in observational studies, response was similar and inversely associated with GTN risk score. Nausea and other gastrointestinal events were more common with dactinomycin than methotrexate.

Incidence of GTN varied by region and setting. Consensus supports methotrexate or dactinomycin as first-line therapy in low-risk GTN. Treatment options were more diverse in high-risk GTN and later-line regimens. Unmet needs to improve response rates and minimize toxicity persist.