Epidemiology Treatment Patterns and Outcomes in Gestational Trophoblastic Neoplasia: A Systematic Literature Review and Gap Analysis
Author(s)
Mairead M. Kearney, MB, BCh, MPH, MBA, MSc Econ1, Jason Hoffman, PharmD2, Laura Cilek, PhD3, Jason Simeone, PhD, MS4.
1Global Value Demonstration, Market Access and Pricing, Biopharma, Global Operations, Merck Healthcare KGaA, Darmstadt, Germany, 2EMD Serono Research & Development Institute, Inc., an affiliate of Merck KGaA, Billerica, MA, USA, 3Real-World Evidence, GIPAM GmbH, Wismar, Germany, 4Real-World Evidence, GIPAM Inc., Winchester, MA, USA.
1Global Value Demonstration, Market Access and Pricing, Biopharma, Global Operations, Merck Healthcare KGaA, Darmstadt, Germany, 2EMD Serono Research & Development Institute, Inc., an affiliate of Merck KGaA, Billerica, MA, USA, 3Real-World Evidence, GIPAM GmbH, Wismar, Germany, 4Real-World Evidence, GIPAM Inc., Winchester, MA, USA.
OBJECTIVES: To understand data on epidemiology, treatment patterns, and outcomes in patients with gestational trophoblastic neoplasia (GTN).
METHODS: This systematic literature review examined English-language publications on GTN found in Embase and MEDLINE via Ovid (previous 20 years for interventional and 5 years for observational studies), plus relevant abstracts from target conferences from 2022-2024. Studies on benign gestational trophoblastic disease (GTD), case reports, and case series were excluded. Screening and extraction were performed by 2 independent reviewers; study quality was assessed using relevant instruments. Pooled response rates and 95% CIs were calculated using random-effects models.
RESULTS: Of 117 studies included, two-thirds were cohort studies (n=78 [67.8%]) and approximately half (n=64 [53.3%]) were conducted in Asia. GTN incidence varied widely by region and setting; the incidence of GTN among all registered patients with GTD in the UK was 6.2%, whereas the incidence in 3 hospital-based studies in Asia was 19.2%. In observational studies, the most common first-line treatments for low-risk GTN were methotrexate and dactinomycin monotherapy. Treatments used in second- line and for high-risk patients included diverse multi-agent regimens and immunotherapy. Across trials, the response rate to first-line treatment was higher with dactinomycin (87.8%; 95% CI, 80.3%-92.7%) vs methotrexate (76.4%; 95% CI, 60.3%-87.4%]). In observational studies, response rates were similar and were inversely associated with GTN risk score. In interventional and observational studies, the incidence of nausea and other gastrointestinal adverse events was higher with dactinomycin vs methotrexate. Study quality varied widely. Ten observational studies reported that patients often had successful pregnancies following fertility-sparing treatment.
CONCLUSIONS: GTN incidence in patients with GTD ranged from 6.2% (UK) to 19.2% (Asia). General consensus was observed for methotrexate or dactinomycin as first-line treatment in low-risk GTN. Treatment regimens were more diverse in high-risk GTN and later lines. However, first-line regimens are not interchangeable and unmet needs exist.
METHODS: This systematic literature review examined English-language publications on GTN found in Embase and MEDLINE via Ovid (previous 20 years for interventional and 5 years for observational studies), plus relevant abstracts from target conferences from 2022-2024. Studies on benign gestational trophoblastic disease (GTD), case reports, and case series were excluded. Screening and extraction were performed by 2 independent reviewers; study quality was assessed using relevant instruments. Pooled response rates and 95% CIs were calculated using random-effects models.
RESULTS: Of 117 studies included, two-thirds were cohort studies (n=78 [67.8%]) and approximately half (n=64 [53.3%]) were conducted in Asia. GTN incidence varied widely by region and setting; the incidence of GTN among all registered patients with GTD in the UK was 6.2%, whereas the incidence in 3 hospital-based studies in Asia was 19.2%. In observational studies, the most common first-line treatments for low-risk GTN were methotrexate and dactinomycin monotherapy. Treatments used in second- line and for high-risk patients included diverse multi-agent regimens and immunotherapy. Across trials, the response rate to first-line treatment was higher with dactinomycin (87.8%; 95% CI, 80.3%-92.7%) vs methotrexate (76.4%; 95% CI, 60.3%-87.4%]). In observational studies, response rates were similar and were inversely associated with GTN risk score. In interventional and observational studies, the incidence of nausea and other gastrointestinal adverse events was higher with dactinomycin vs methotrexate. Study quality varied widely. Ten observational studies reported that patients often had successful pregnancies following fertility-sparing treatment.
CONCLUSIONS: GTN incidence in patients with GTD ranged from 6.2% (UK) to 19.2% (Asia). General consensus was observed for methotrexate or dactinomycin as first-line treatment in low-risk GTN. Treatment regimens were more diverse in high-risk GTN and later lines. However, first-line regimens are not interchangeable and unmet needs exist.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO99
Topic
Clinical Outcomes, Study Approaches
Topic Subcategory
Clinical Outcomes Assessment
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology