Successful treatment of refractory gestational trophoblastic neoplasm with high-dose etoposide and cyclophosphamide.

A patient with gestational trophoblastic neoplasm failed treatment with several standard chemotherapy regimens and had progressive disease with development of lung and brain metastases and a rising HCG level. Following resection of the metastases and whole-brain radiotherapy she was treated with high-dose etoposide and cyclophosphamide. She promptly attained a complete remission and remains free of disease 15 months after completion of therapy. This regimen, although initially developed for leukemia and lymphoma treatment, has potential as a therapy for refractory gestational trophoblastic neoplasm because it delivers high doses of agents very active in this disease.