Efficacy and safety of biweekly single-dose actinomycin D versus multiday methotrexate in low-risk gestational trophoblastic neoplasia: a prospective multicenter randomized trial

作者：Fang Jiang, C Guan, Lanzhou Jiao, Ting Xu, Xirun Wan, S. S. Shi, Beibei Wu, Xiaojun Zhang, Linlin Zhen, Jidong Miao, Ming Tian, Ming Du, Chen Li, Fred Feng, Junjun Yang, Tong Ren, Jun Zhao, Yuan Li, Xia Zhang, Xin Lu, Yang Xiang · 发表于：Annals of Oncology · 年份：2025 · DOI：10.1016/j.annonc.2025.06.006 · 被引用次数：11 · 研究领域：Gestational Trophoblastic Disease Studies、Maternal and fetal healthcare、Pregnancy and preeclampsia studies

BACKGROUND: Cure rates for low-risk gestational trophoblastic neoplasia (GTN) are high, but there is no consensus on optimal first-line chemotherapy. Here we evaluated the efficacy and safety of biweekly single-dose actinomycin D (Act-D) versus an 8-day methotrexate (MTX)-folinic acid regimen as first-line single-agent chemotherapy for low-risk GTN. PATIENTS AND METHODS: , maximum 2 mg, every 14 days) or MTX-folinic acid (50 mg i.m. days 1, 3, 5, and 7; leucovorin rescue, days 2, 4, 6, and 8). Treatment continued until β-human chorionic gonadotropin normalization, followed by 2-3 consolidation cycles. Primary outcomes were complete remission (CR) rates for single-agent chemotherapy and overall CR rates. Secondary outcomes were time to CR, chemotherapy cycles, toxicity, and anti-Müllerian hormone changes. RESULTS: Between 27 September 2020, and 18 June 2024, 228 patients were randomized to MTX or Act-D. Act-D achieved significantly higher single-agent CR rates than MTX (72.8% versus 54.4%, P = 0.0038) with shorter median remission time (7.86 versus 9.43 weeks, P = 0.0296). Overall CR rates were 100% in both groups following combination chemotherapy for resistant cases. Most adverse events were grade 1-2, but grade ≥2 nausea and vomiting and hair loss were more frequent with Act-D, and alanine aminotransferase was more frequently elevated in the MTX group. Anti-Müllerian hormone reductions were transient in both groups. After a 28.5-month median follow-up, recurrence rates rema...