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Table 2 Studies evaluating GM-CSF as a monotherapy in patients with advanced melanoma

From: Current status of granulocyte–macrophage colony-stimulating factor in the immunotherapy of melanoma

Citation Evaluable patients GM-CSF dose schedule Route of administration Clinical response Observations
Si et al. [44] 13 15–50 μg/lesion at 2 sites per patient Intralesional 1 PR, 8 SD Responding 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] 16 10, 20, 40, or 80 μg/injection for 10 d Intralesional 3 SD Significant increase in DCs and T cells at injection sites
Vaquerano et al. [51] 1 500 μg/d for 4 d, monthly Intralesional 1 PR Regression of melanoma cells
Hoeller et al. [46] 7 400 μg/d for 5 d, 21-d cycle Perilesional 6 with reduced lesion size Increased infiltration of monocytes and lymphocytes was observed in injected and systemic sites
Ridolfi et al. [52] 14 150 μg/lesion plus IL-2 3 × 106 IU for 5 d, 21-day cycle Intralesional (GM-CSF) Perilesional (IL-2) 2 PR, 2 MR, 7 SD Some evidence of systemic immune activation
Rao et al. [47] 14 250 μg twice daily for 7 d on alternating weeks Aerosol delivery for lung metastases 6 SD Upregulation of cytotoxic T lymphocytes was observed in peripheral blood
Markovic et al. [48] 35 500–2000 μg (250-μg/dose increments) twice daily on days 1–7 and 15–21, over 28 d Aerosol delivery for lung metastases 1 PR, 5 SD A trend toward increased immune response was observed with higher doses; MTD was not reached
Sato et al. [49] 31 25–2000 μg every 4 wk Hepatic artery immunoembolization 2 CR, 8 PR, 10 SD Prolonged PFS correlated with higher GM-CSF doses
Eschelman et al. [50] 52 2000 μg every 4 wk Hepatic artery immunoembolization 5 PR, 12 SD Trend toward increased OS with GM-CSF; prolonged OS with GM-CSF in patients with bulky metastases
  1. 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.