Granulocyte-colony-stimulating Factor for Acute Ischemic Stroke: A Randomized Controlled Trial (STEMTHER)

Granulocyte-colony-stimulating factor (G-CSF) functions both as a neuroprotectant and a stimulator of autologous bone marrow stem cell release. Therefore, administration of G-CSF should improve the outcome of stroke. Here, we examine the safety of using G-CSF to treat acute ischemic stroke using a randomized controlled trial involving 20 adult patients presenting with ischemia in the carotid region within 48 h of onset. The experimental group (n=10) received subcutaneous G-CSF injections (10 mg kg(-1) day(-1)) in addition to conventional therapy for 5 days. The primary outcome was motor function as measured by the modified Rankin Scale 180 days post-stroke. Safety was evaluated according to the frequency of hemorrhagic transformation of infarctions and serious adverse events. Only six patients in the experimental group completed full course of treatment, while four patients (three in the control and one in the experimental group) were lost to follow-up. We found the experimental and control groups did not differ significantly in either neurological impairment or degree of disability/dependence at 180 days post-stroke. We conclude that while adding G-CSF (10 mg kg(-1) day(-1)) to acute ischemic stroke therapy for 5 days is safe, its efficacy remains unproven.