. Author manuscript; available in PMC: 2020 Jun 2.

Published in final edited form as: Contemp Clin Trials. 2020 Mar 4;91:105975. doi: 10.1016/j.cct.2020.105975

Table 1:

Summary of Clinical Trials evaluating the therapeutic role of GM-CSF in Peripheral Artery Disease

Study	Design	Sample Size	GM-CSF dose	Results
START¹³	Double-blinded, randomized, placebo-controlled trial	N=40 GM-CSF (N=20) Placebo (N=20)	Subcutaneous injections 10 μg/kg/day On alternate days for 14 days (7 doses)	Change in walking distance at 2 weeks: GM-CSF: 63±111 m vs. Placebo: 57±113 m (p=0.87)
START¹³	Double-blinded, randomized, placebo-controlled trial	N=40 GM-CSF (N=20) Placebo (N=20)		Change in pain-free walking distance at 2 weeks: GM-CSF: 28±49 m vs. Placebo: 30±43 m (p=0.89)
G-PAD-I¹⁴	Double-blinded, randomized, placebo-controlled trial	N=45 GM-CSF (N=29, three groups) Placebo (N=16)	Subcutaneous injections 3 μg/kg/day (N=10), 6 μg/kg/day (N=9), 10 μg/kg/day (N=10) Three times a week for two weeks (6 doses)	Change in brachial artery FMD at 12 weeks: GM-CSF: 2.9±06% to 4.6±0.6% (p=0.01) Placebo: 4.3±0.6% to 5.0±0.6% (p=0.50)
				Change in treadmill pain-free walking time at 12 weeks: GM-CSF: +38 s (p=0.008) Placebo: No significant change
				Change in treadmill total walking time at 12 weeks: GM-CSF: +55 s (p=0.016) Placebo: No significant change
GPAD-2¹⁵	Double-blinded, randomized, placebo-controlled trial	N=159 GM-CSF (N=80) Placebo (N=79)	Subcutaneous injections 500 μg/day Three times a week for four weeks (12 doses)	Change in treadmill peak walk time at 3 months: GM-CSF: 109 s (95% CI 67, 151) Placebo: 56 s (95% CI 14, 98) Difference: 53 s (95% CI −6, 112; p=0.08)
				Change in treadmill peak walk time at 6 months: GM-CSF: 112 s (95% CI 71, 153) Placebo: 77 s (95% CI 36, 117) Difference: 35 s (95% CI −26, 93; p=0.24)
				Change in treadmill claudication onset time at 3 months: GM-CSF: 81 s (95% CI 52, 111) Placebo: 61 s (95% CI 32, 91) Difference: 20 s (95% CI −22, 62; p=0.35)
				Change in treadmill claudication onset time at 6 months: GM-CSF: 93 s (95% CI 64, 122) Placebo: 61 s (95% CI 33, 89) Difference: 35 s (95% CI −8, 72; p=0.12)
PROPEL¹⁶	Double-blinded, randomized, placebo/attention-controlled trial with a 2 × 2 factorial design	N=210 GM-CSF + Supervised Exercise (N=53) Placebo + Supervised exercise (N=53) GM-CSF + Attention Control (N=53) Placebo + Attention Control (N=51)	Subcutaneous injections 250 μg/m²/day Three times a week for two weeks (6 doses)	Change in 6-minute walk distance at 12 weeks: GM-CSF + Exercise: 22.2 m (95% CI 5.4, 39.0) Placebo + Exercise: 28.5 m (95% CI 11.7, 45.4) Difference: −6.3 m (95% CI −30.2, 17.6; p=0.61)
				GM-CSF + Attention Control: −6.4 m (95% CI −23.0, 10.1) Placebo + Attention Control: −5.0 m (95% CI −22.3, 12.2) Difference: −1.4 m (95% CI −25.2, 22.4; p=0.91)
				Change in treadmill maximal walk time at 12 weeks: GM-CSF + Exercise: 3.5 min (95% CI 2.5, 4.5) Placebo + Exercise: 4.2 min (95% CI 3.2, 5.2) Difference: −0.7 min (95% CI −2.1, 0.8; p=0.35)
				GM-CSF + Attention Control: −0.1 min (95% CI −1.1, 0.9) Placebo + Attention Control: 0.5 min (95% CI −0.6, 1.6) Difference: −0.6 min (95% CI −2.1, 0.9; p=0.44)

Abbreviations: GM-CSF = granulocyte-macrophage colony stimulating factor, m = meter, FMD = flow mediated dilation, s = second, min = minute.