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

Si et al. [44]

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]

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]

500 μg/d for 4 d, monthly

Intralesional

1 PR

Regression of melanoma cells

Hoeller et al. [46]

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]

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]

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]

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]

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]

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