5-Fluorouracil as first-line treatment for low-risk gestational trophoblastic neoplasia

Purpose: To investigate the efficacy and prognostic factors in response to 5-fluorouracil (5-FU) in low-risk gestational trophoblastic neoplasia (GTN). Methods: This single-center retrospective study analyzed the hospital records of 204 LRGTN patients admitted to Department of Gynecology, Liaoning Cancer Hospital & Institute of China from 2002 to 2016 for retrieval of their clinical data, chemotherapy regimens, related side-effects, and evaluation of treatment efficacy and prognostic factors. Results: The median progression-free survival (PFS) was 55 months (3 - 190 months). The overall cure rate was 100 %, with no tumor-related deaths. When a single-agent regimen i.e. 5-FU, was selected for initiation of treatment for 132 patients while only 49 of them were treated with chemotherapy, the effective cure rate was 62.88 % (83/132); while the overall drug resistance r was 27.27 % (36/132). For patients with FIGO scores >= 4 points, the incidence of drug resistance was 71.43 % (5/7), while the incidence of III/IV myelosuppression was 10.61 % (14/132). A total of 38 patients (18.63 %) received surgical treatment in addition to chemotherapy. A comparison was made between two groups of patients with non-drug resistance, i.e., patients with unexpected GTN diagnosed postoperatively and those who received chemotherapy preoperatively. It was found that the number of courses of GTN chemotherapy for those who were unexpectedly diagnosed postoperatively was more than that for those who received chemotherapy preoperatively (p = 0.004). Conclusion: The single drug (5-FU) was effective in the management of low-risk (LR)-GTN. Treatment failure was related to drug resistance, high tumor score, and severe toxicity. Multi-agent regiments in combination with surgery, were an effective treatment method for GTN. For patients without metastasis and fertility requirements, surgery after chemotherapy significantly shortened the treatment cycle without increasing complications.