The objective of this study was to analyze the toxicity and the effica
cy of single-agent 5-day methotrexate for women with metastatic gestat
ional trophoblastic disease. The study is a retrospective analysis of
52 patients who received repetitive 5-day cycles of intramuscular meth
otrexate as primary therapy for metastatic trophoblastic disease betwe
en 1975 and 1990. The majority of patients were low-risk by both clini
cal and World Health Organization prognostic index score criteria. Six
ty percent achieved primary remission with a median of 3 cycles of sin
gle-agent methotrexate. Therapy was changed because of toxicity and dr
ug-resistance by hCG criteria in 11 (21%) and 10 (19%) patients, respe
ctively. Pretherapy hCG >10,000 mIU/ml was associated with the develop
ment of drug-resistance. Remission was achieved in all patients, with
only 2 (4%) requiring multiagent therapy. The use of repetitive 5-day
cycles of methotrexate is efficacious therapy of low-risk metastatic t
rophoblastic disease. Future studies are needed to define a cost-effec
tive and minimally toxic therapy that retains a high primary remission
rate in these patients. (C) 1994 Academic Press, Inc.