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Intramyocardial Cell Delivery: Observations in Murine Hearts
Published on: January 24, 2014
Granulocyte colony-stimulating factor for stem cell mobilisation in acute myocardial infarction: a randomised
Felice Achilli1, Stefano Maggiolini2, Fabiana Madotto3
1Department of Cardiology, ASST Brianza, Desio, Italy.
Insights
Granulocyte colony-stimulating factor (G-CSF) did not improve outcomes in ST-elevation myocardial infarction (STEMI) patients. Further research is needed to identify patient subgroups who may benefit from bone marrow cell mobilization therapies.
Area of Science:
- Cardiology
- Regenerative Medicine
- Clinical Trials
Background:
- Investigating the role of granulocyte colony-stimulating factor (G-CSF) in improving clinical outcomes after ST-elevation myocardial infarction (STEMI).
- Focusing on patients with left ventricular (LV) dysfunction post-percutaneous coronary intervention (PCI).
Purpose of the Study:
- To determine if early G-CSF administration improves 2-year cardiac mortality and morbidity in STEMI patients with reduced LV ejection fraction (≤45%) after PCI.
- Assessing the efficacy and safety of G-CSF in this patient population.
Main Methods:
- A prospective, multicentre, nationwide, randomised, open-label, phase III trial (NCT01969890).
- Patients received either G-CSF or standard of care (SOC).
- The primary outcome was a composite of all-cause death, myocardial infarction recurrence, and heart failure hospitalization.
Main Results:
- No significant difference in the primary composite outcome between G-CSF and SOC groups (HR 1.20; 95% CI 0.63 to 2.28).
- Similar 2-year mortality rates (2.31% vs. 2.68%) and adverse event profiles between groups.
- Post hoc analyses suggested potential benefits in patients with severe LV systolic dysfunction and trends towards better outcomes with low bone marrow cell mobilization.
Conclusions:
- The trial, though inconclusive due to low recruitment and event rates, is the largest study on cell-based cardiac repair post-STEMI.
- G-CSF treatment demonstrated tolerability and long-term safety.
- Further studies are warranted to identify patient subgroups that benefit most from bone marrow cell mobilization.
Background:
To determine whether granulocyte colony-stimulating factor (G-CSF) improves clinical outcomes after large ST-elevation myocardial infarction (STEMI) when administered early in patients with left ventricular (LV) dysfunction after successful percutaneous coronary intervention (PCI).
Methods:
STEM-AMI OUTCOME was designed as a prospective, multicentre, nationwide, randomised, open-label, phase III trial (ClinicalTrials.gov ID: NCT01969890) to demonstrate the efficacy and safety of early G-CSF administration in reducing 2-year cardiac mortality and morbidity in patients with STEMI with LV ejection fraction ≤45% after PCI. The primary outcome was a composite of all-cause death, recurrence of myocardial infarction and hospitalisation for heart failure. Due to low recruitment and event rates, the study was discontinued and did not achieve adequate statistical power to verify the hypothesis.
Results:
Patients were randomly allocated to G-CSF (n=260) or standard of care (SOC; n=261). No difference was found in the composite primary outcome between study groups (HR 1.20; 95% CI 0.63 to 2.28). The 2-year mortality was 2.31% in the G-CSF and 2.68% in the control group (HR 0.88; 95% CI 0.29 to 2.60). Adverse events did not differ between the G-CSF (n=65) and SOC groups (n=58; OR 1.17; 95% CI 0.78 to 1.75). In post hoc analyses on the intervention group, we observed a trend towards fewer composite primary outcomes in patients with low bone marrow (BM) cell mobilisation (n=108) versus those with high mobilisation (n=152, with peak leucocyte count >50×109/L; HR 2.86; 95% CI 0.96 to 8.56). Primary outcomes were lower in patients with severe LV systolic dysfunction at discharge treated with G-CSF than in controls (interaction β±SE, -0.08±0.04; p=0.034).
Conclusions:
Although inconclusive, this is the largest trial in the field of cell-based cardiac repair after STEMI providing evidence of the tolerability and long-term safety of G-CSF treatment. The results prompt further studies to understand which patient can benefit most from BM cell mobilisation.
Trial Registration Number:
NCT01969890.
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