Comparison of the efficacy and safety of five-day methotrexate versus pulse actinomycin D for low-risk gestational trophoblastic neoplasia: a single-center historical cohort study☆.

OBJECTIVE

In 2019, our institution changed the first-line regimen for low-risk gestational trophoblastic neoplasia from 5-day methotrexate (MTX) (20 mg × 5 days, intramuscular injection) to pulse actinomycin D (1.25 mg/m2 intravenous) every 14 days. We compared the efficacy and safety of these regimens.

METHODS

Between 2007 and 2022, 105 low-risk gestational trophoblastic neoplasia cases were identified. The patients' background, outcomes, and adverse effects were analyzed retrospectively using the Mann-Whitney U test, Fisher's exact test, and propensity score-matching analyses.

RESULTS

Among the patients, 83 were treated before 2019 (MTX group), and 22 were treated after 2019 (actinomycin D group). Age and pre-treatment human chorionic gonadotropin levels were comparable between the groups. However, the International Federation of Gynecology and Obstetrics score was significantly greater in the MTX group (p < .05). The primary remission rate was significantly greater in the actinomycin D group (81.8%, 18/22) than in the MTX group (38.6%, 32/83, p < .01). Drug resistance was observed in 24.1% (20/83) and 18.2% (4/22) of patients in the MTX and actinomycin D groups, respectively (p = .78). The superior effects of actinomycin D were also observed in the analysis of matched patient backgrounds, including age, human chorionic gonadotropin levels, and International Federation of Gynecology and Obstetrics scores (p < .05). Severe adverse effects requiring a change in chemotherapy were reported in 37.3% (31/83) of the patients in the MTX group but none in the actinomycin D group.

CONCLUSIONS

Pulse actinomycin D was more effective and safer than 5-day MTX. Considering the convenience of fewer hospital visits, the pulse actinomycin D regimen is a better option for low-risk gestational trophoblastic neoplasia in the Japanese population.