Background. 5-fluorouracil-based multiagent chemotherapy has been used as the primary treatment for high-risk gestational trophoblastic neoplasia (GTN) in China for a few decades. This study aims to assess the efficacy and toxicity of floxuridine, actinomycin D, etoposide, and vincristine (FAEV) as primary treatment for patients with GTN who had International Federation of Gynecology and Obstetrics (FIGO) scores ≥5. Methods. A total of 207 patients with GTN who had FIGO scores ≥5 were treated with FAEV as first-line chemotherapy at Peking Union Medical College Hospital between January 2002 and December 2017. Complete remission (CR), resistance, survival, toxicity, and reproductive outcomes were analyzed. Results. Of the 207 patients treated with FAEV, 9 (4.3%) required a change of chemotherapy owing to toxicity and 1 (0.5%) died of cerebral hernia 5 weeks after commencing treatment. The remaining 197 patients were assessable to determine the response to FAEV; among them, 168 (85.3%) achieved CR with FAEV and 29 (14.7%) developed resistance to FAEV. The 5-year overall survival rate of the entire cohort was 97.4%. Grade 3-4 neutropenia, thrombocytopenia, and anemia occurred in 28.4%, 6.8%, and 6.2% of cycles, respectively. No acute toxicity-related deaths occurred. Five patients developed acute myeloid leukemia 10-50 months after exposure to chemotherapy; another patient developed duodenal cancer 2 years after completing therapy. Sixty-one patients who preserved fertility wanted to become pregnant; 56 of them conceived.
Conclusion.The FAEV regimen is an effective primary treatment for patients with GTN who have FIGO scores ≥5, and has predictable and manageable toxicity. The Oncologist 2021;9999:• • Implications for Practice: The most commonly used multiagent chemotherapy for high-risk gestational trophoblastic neoplasia (GTN) is EMA/CO worldwide. However, 5-fluorouracil-based multiagent chemotherapy has been used as the primary treatment for high-risk GTN in China for a few decades. This study evaluated the efficacy and toxicity of FAEV as a primary treatment for patients with GTN who have FIGO scores ≥5. Our data demonstrated that FAEV as primary treatment achieved favorable outcomes for patients with FIGO scores ≥5. Toxicities that result from the FAEV regimen are predictable and manageable. The FAEV regimen may provide another option for the treatment of GTN.