Gestational Trophoblastic Disease Treatment (PDQ®): Treatment - Health Professional Information [NCI]-High-Risk Gestational Trophoblastic Neoplasia (FIGO Score ≥7) Treatment Multiagent chemotherapy is standard for the initial management of high-risk gestational trophoblastic neoplasia (GTN). A systematic literature review revealed only one randomized controlled trial (and no high-quality trials)-conducted in the 1980s-comparing multiagent chemotherapy regimens for high-risk GTN.[1] In the trial, only 42 women were randomly assigned to either a CHAMOMA regimen (i.e., methotrexate, folinic acid, hydroxyurea, dactinomycin, vincristine, melphalan, and doxorubicin) or MAC (i.e., methotrexate, dactinomycin, and chlorambucil).[2] There was substantially more life-threatening toxicity in the CHAMOMA arm and no evidence of higher efficacy. However, there were serious methodologic problems with this trial. It was reportedly designed as an equivalency trial, but owing to the small sample size, the trial was inadequately powered to assess equivalence. In addition, the characteristics of the patients randomly assigned to the two study arms were not reported (although the authors stated that there were no major differences in the patient populations assigned to each arm), nor was the method of randomization or allocation concealment described.

There are no randomized trials comparing regimens in common use to establish the superiority of one over another. Therefore, the literature does not permit firm conclusions about the best chemotherapeutic regimen.[1][Level of evidence 3iiiDii] However, since EMA/CO (i.e., etoposide, methotrexate, and dactinomycin/cyclophosphamide and vincristine) is the most commonly used regimen, the specifics are provided in Table 2 below.[3,4,5]

Table 2. Specifics of the EMA/CO Regimena,b,c

Day	Drug	Dose
IV = intravenously; PO = orally.
a Adapted from Bower et al.[3]
b Adapted from Escobar et al.[4]
c Adapted from Lurain et al.[5]
1	Etoposide	100 mg/m2 IV for 30 min
	Dactinomycin	0.5 mg IV push
	Methotrexate	300 mg/m2 IV for 12 h
2	Etoposide	100 mg/m2 IV for 30 min
	Dactinomycin	0.5 mg IV push
	Folinic Acid	15 mg or PO every 12 h × 4 doses, beginning 24 h after the start of methotrexate
8	Cyclophosphamide	600 mg/m2 IV infusion
	Vincristine	0.8-1.0 mg/m2 IV push (maximum dose 2 mg)

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