Treatment of high-risk gestational trophoblastic neoplasia with weekly high-dose methotrexate–etoposide

ObjectiveTo assess toxicity and efficacy of weekly high-dose methotrexate–etoposide (HD MTX–ETO) in high-risk gestational trophoblastic neoplasia (GTN).MethodsRetrospective chart review of high-risk GTN patients treated with HD MTX–ETO (methotrexate 1000 mg/m² day 1, etoposide 100 mg/m² days 1 and 2, q 1 wk).Results134 cycles of HD MTX–ETO were administered to twelve patients; median number of cycles was 8 (range 2–39 cycles). Median follow up was 25.5 months (range 11–69). 7 of these patients switched due to ototoxicity from EP–EMA (etoposide 150 mg/m², cisplatin 75 mg/m² i.v. day 1; etoposide 100 mg/m², methotrexate 300 mg/m², dactinomycin 0.5 mg i.v. day 8, q 14 d) to HD MTX–ETO, after an average of 7 cycles of EP-EMA. Six achieved complete remission without disease recurrence. One patient with a placental site trophoblastic tumour died due to progressive disease. Five patients received HD MTX–ETO primarily; 1 patient with choriocarcinoma presenting with metastases to the brain and liver (WHO score 19) was switched to EP–EMA and died due to complications under EP–EMA. The other 4 achieved complete remission without disease recurrence.HD MTX–ETO was well tolerated; non-haematological toxicity was low except for alopecia and fatigue. Nine patients had grade 2–4 anaemia and received packed cells. Eight patients had grade 3–4 neutropenia and received G-CSF. Two patients developed febrile neutropenia without sepsis.ConclusionsThese preliminary results show a better toxicity profile with HD MTX–ETO than EP–EMA and encouraging efficacy. HD MTX–ETO might be a treatment option for some patients with high-risk GTN and needs further investigation.

► High-dose methotrexate–etoposide is described for the treatment of high-risk GTN.
► Toxicity profile was low except for myelosuppression.
► The remission rate was 83% (10/12 patients) after a median of 8 weekly cycles.