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Peer-reviewedCC BY 4.0Frontiers in Oncology

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

  1. 1Merck Healthcare KGaA
  2. 2EMD Serono Research & Development Institute, Inc. , , , , an affiliate of Merck KGaA
  3. 3GIPAM GmbH

Abstract

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

Methods: 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.

Results: 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.

Discussion: 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.

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BibTeX
@article{kearney2026epidemiology,
  title = {{Epidemiology, treatment patterns, and outcomes in gestational trophoblastic neoplasia: a systematic review}},
  author = {Mairead Kearney and Jason Hoffman and Laura Cilek and Jason Simeone},
  journal = {Frontiers in Oncology},
  year = {2026},
  month = oct,
  doi = {10.3389/fonc.2026.1923113},
  url = {https://www.frontiersin.org/articles/10.3389/fonc.2026.1923113/full},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - Epidemiology, treatment patterns, and outcomes in gestational trophoblastic neoplasia: a systematic review
AU  - Mairead Kearney
AU  - Jason Hoffman
AU  - Laura Cilek
AU  - Jason Simeone
PY  - 2026
DA  - 2026/10/14
JO  - Frontiers in Oncology
DO  - 10.3389/fonc.2026.1923113
UR  - https://www.frontiersin.org/articles/10.3389/fonc.2026.1923113/full
AB  - Introduction: Current global data on epidemiology, treatment patterns, outcomes, and care pathways for patients with gestational trophoblastic neoplasia (GTN) are needed.  Methods: 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.  Results: 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.  Discussion: 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.
SN  - 2234-943X
ER  -