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Bone marrow mobilization with granulocyte macrophage colony-stimulating factor improves endothelial dysfunction and
Veerappan Subramaniyam1, Edmund K Waller, Jonathan R Murrow
1Division of Cardiology, Emory University School of Medicine, 1364 Clifton Rd., Atlanta, GA 30322, USA.
Insights
Granulocyte-macrophage colony-stimulating factor (GM-CSF) therapy safely improved endothelial function and exercise capacity in peripheral arterial disease (PAD) patients by mobilizing progenitor cells.
Area of Science:
- Cardiovascular Medicine
- Hematology
- Regenerative Medicine
Background:
- Peripheral arterial disease (PAD) is characterized by endothelial dysfunction and reduced exercise capacity.
- Mobilizing progenitor cells is a potential therapeutic strategy for PAD.
Purpose of the Study:
- To evaluate the safety and efficacy of granulocyte-macrophage colony-stimulating factor (GM-CSF) in improving endothelial function and exercise capacity in PAD patients.
- To assess GM-CSF's ability to mobilize progenitor cells.
Main Methods:
- A randomized, placebo-controlled trial involving 45 PAD patients.
- Patients received escalating doses of GM-CSF or placebo, with measurements of CD34+ cells, endothelial function, and exercise capacity.
Main Results:
- GM-CSF administration was safe and significantly increased leukocytes, CD34+ cells, and colony-forming units.
- At 12 weeks, GM-CSF treatment improved endothelial function (flow-mediated vasodilation) and treadmill walking times.
- No significant improvements were observed in the placebo group.
Conclusions:
- GM-CSF therapy is a safe and effective strategy for mobilizing progenitor cells, improving endothelial function, and enhancing exercise capacity in PAD patients.
- Further research into bone marrow progenitor mobilization strategies for PAD is warranted.
Background:
We hypothesized that granulocyte macrophage colony-stimulating factor (GM-CSF) administration will be safe and will improve endothelial dysfunction and exercise capacity by mobilizing progenitor cells in patients with peripheral arterial disease (PAD).
Methods:
Forty-five patients with PAD received thrice-weekly injections for 2 weeks of 3, 6, or 10 microg/kg per day of GM-CSF or placebo in successive cohorts of 15 subjects randomized 2:1 to drug or placebo. CD34+ mononuclear cell subsets and colony formation assay, endothelial function, ankle-brachial index, and walking capacity were measured.
Results:
Granulocyte macrophage colony-stimulating factor administration was safe. After pooling data from GM-CSF cohorts, at 2 weeks, there was a significant increase in total leukocytes (43%, P < .0001), CD34+ cells (46%, P = .035), and colony-forming units (31%, P = .026, week 1). At 12 weeks, endothelial function improved with GM-CSF (flow-mediated vasodilation increased by 59%, P < .01) as did pain-free treadmill walking time (38 seconds, P = .008) and total treadmill walking time (55 seconds, P = .016). Corresponding changes were not observed in the placebo group.
Conclusions:
Granulocyte macrophage colony-stimulating factor therapy in patients with PAD was associated with mobilization of progenitor cells, improvement of endothelial dysfunction, and exercise capacity. The efficacy of strategies designed to mobilize bone marrow progenitors warrants further study in patients with PAD.
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