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Gestational Trophoblastic Disease Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Low-Risk Gestational Trophoblastic Neoplasia (FIGO Score 0–6) Treatment

There is no consensus on the best chemotherapy regimen for initial management of low-risk gestational trophoblastic neoplasia (GTN), and first-line regimens vary by geography and institutional preference. Most regimens have not been compared head-to-head, and the level of evidence for efficacy is often limited to 3iiDii except as noted below. Even if there are differences in initial remission rate among the regimens, salvage with alternate regimens is very effective, and the ultimate cure rates are generally 99% or more. The initial regimen is generally given until a normal beta human chorionic gonadotropin (beta-hCG) (for the institution) is achieved and sustained for 3 consecutive weeks (or at least for one treatment cycle beyond normalization of the beta-hCG). A salvage regimen is instituted if any of the following occur:

  • A plateau of the beta-hCG for 3 weeks (defined as a beta-hCG decrease of 10% or less for 3 consecutive weeks).
  • A rise in beta-hCG of greater than 20% for 2 consecutive weeks.
  • Metastases appear.

The use of chemotherapy in the first-line management of low-risk GTN has been assessed in a Cochrane Collaboration systematic review.[1] In that systematic review, four randomized controlled trials were identified.[2,3,4,5]

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Three of the randomized trials [3,4,5] compared the same two commonly used regimens:

  • Biweekly (pulsed) dactinomycin (1.25 mg/m2 intravenously [IV]) .
  • Weekly intramuscular methotrexate (30 mg/m2).

These three trials included a total of 392 patients. All three trials showed better primary complete response (CR) rates without the need for additional salvage therapy associated with pulsed dactinomycin (relative risk [RR] of cure, 3.00; 95% confidence interval [CI], 1.10–8.17), even though the magnitude of benefit showed substantial heterogeneity (I2 statistic = 79%).[3,4,5][Level of evidence: 1iiDii] Fewer courses of therapy were needed to achieve CR and cure with dactinomycin treatment. As expected, salvage chemotherapy was nearly uniformly successful, because almost all low-risk GTN patients are ultimately cured, irrespective of the initial chemotherapeutic regimen. There were no statistically significant differences in most toxicities, including the following:

  • Nausea and vomiting.
  • Diarrhea.
  • Hematologic toxicity.
  • Hepatic toxicity.
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