Background and purpose: Gestational trophoblastic neoplasm (GTN) is a spectrum of disease arising from trophoblastic cells

and the majority of patients with GTN have favorable outcome because of the sensitivity to chemotherapy. While the cure rate for high-risk patients is still 70% to 80% as a result of drug resistance and disease recurrence. This study aimed to evaluate the clinical characteristics and outcome of patients with high-risk GTN. Methods: The clinical records of patients with high-risk GTN treated in Obstetrics and Gynecology Hospital of Fudan University from Jan. 2003 to Jan. 2013 were analyzed and reviewed retrospectively from the aspect of different treatment. Results: Fifty-one patients with high-risk GTN were admitted to this hospital. Among 51 high-risk GTN patients

46 patients were evaluated retrospectively and 5 patients were excluded for incomplete treatments. Of the 46 patients with high-risk GTN

27 patients were treated by chemotherapy alone

19 patients received chemotherapy and adjuvant surgical therapy. Forty-four patients received EMA-CO (VP-16+Act-D+MTX/VCR+CTX) as a first-line chemotherapy

81.82% (36/44) had complete remission and 8 patients developed resistance to EMA-CO. EMA-EP (VP- 16+Act-D+MTX/VP-16+cisplatin) was used as second-line chemotherapy for the 8 patients resistant to EMA-CO

6 patients (2 underwent adjuvant surgical therapy) achieved remission and 2 patients died as a result of drug-resistance and disease progression. For the remaining 2 patients

one was treated by 5-FU+KSM and pulmonary resection

and the other was treated by MTX for misdiagnosis as ectopic pregnancy and then converted to EMA-CO for the pathological diagnosis of choriocarcinoma after surgery. Both of them achieved complete remission. Ultimately

95.65% (44/46) patients achieved complete remission. Among the 19 patients who underwent adjuvant surgical therapy

94.70% (18/19) patients achieved complete remission after chemotherapy and adjuvant surgery

and the remaining one patient died of disease progression. Conclusion: Standard combination chemotherapy is crucial in the treatment of high-risk GTN. The role of adjuvant surgery in the management of high-risk GTN should not be underestimated.