Studies evaluating GM-CSF as a monotherapy in patients with advanced melanoma

CitationEvaluable patientsGM-CSF dose scheduleRoute of administrationClinical responseObservations
Si et al. [44]1315–50 μg/lesion at 2 sites per patientIntralesional1 PR, 8 SDResponding patients had increased T-cell and Langerhans cell infiltration of the tumor
Site 1: 5 times daily
Site 2: 5 times daily then once weekly for 6 mo
Nasi et al. [45]1610, 20, 40, or 80 μg/injection for 10 dIntralesional3 SDSignificant increase in DCs and T cells at injection sites
Vaquerano et al. [51]1500 μg/d for 4 d, monthlyIntralesional1 PRRegression of melanoma cells
Hoeller et al. [46]7400 μg/d for 5 d, 21-d cyclePerilesional6 with reduced lesion sizeIncreased infiltration of monocytes and lymphocytes was observed in injected and systemic sites
Ridolfi et al. [52]14150 μg/lesion plus IL-2 3 × 106 IU for 5 d, 21-day cycleIntralesional (GM-CSF) Perilesional (IL-2)2 PR, 2 MR, 7 SDSome evidence of systemic immune activation
Rao et al. [47]14250 μg twice daily for 7 d on alternating weeksAerosol delivery for lung metastases6 SDUpregulation of cytotoxic T lymphocytes was observed in peripheral blood
Markovic et al. [48]35500–2000 μg (250-μg/dose increments) twice daily on days 1–7 and 15–21, over 28 dAerosol delivery for lung metastases1 PR, 5 SDA trend toward increased immune response was observed with higher doses; MTD was not reached
Sato et al. [49]3125–2000 μg every 4 wkHepatic artery immunoembolization2 CR, 8 PR, 10 SDProlonged PFS correlated with higher GM-CSF doses
Eschelman et al. [50]522000 μg every 4 wkHepatic artery immunoembolization5 PR, 12 SDTrend toward increased OS with GM-CSF; prolonged OS with GM-CSF in patients with bulky metastases

CR = complete response; DC = dendritic cell; GM-CSF = granulocyte-macrophage colony-stimulating factor; IL-2 = interleukin-2; MTD = maximum tolerated dose; MR = mixed response; OS = overall survival; PD = progressive disease; PFS = progression free survival; PR = partial response; SD = stable disease.