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