Sargramostim, granulocyte–macrophage colony-stimulating factor, a hematopoietic growth factor, stimulates cells of the intestinal innate immune system.
Preliminary studies suggest sargramostim may have activity in Crohn's disease.
Dr Korzenik and colleagues evaluated this novel therapeutic approach by conducting a randomized, placebo-controlled trial.
|The sargramostim group had significant improvements in quality of life|
|New England Journal of Medicine|
Using a 2 to 1 ratio, the researchers randomly assigned 124 patients with moderate-to-severe active Crohn's disease to treatment and control groups.
The patients were randomized to receive either 6 µg of sargramostim per kilogram per day or placebo subcutaneously for 56 days.
The research team reported that antibiotics and aminosalicylates were allowed, whilst immunosuppressants and glucocorticoids were prohibited.
The primary end point was a clinical response, defined by a decrease from baseline of at least 70 points in the Crohn's Disease Activity Index on day 57.
The researchers included other end points of changes in disease severity and adverse events.
Endpoints relating to change in quality of life included a decrease from baseline of 100 points in the Crohn's Disease Activity Index score on day 57, and remission, defined by a index score of 150 points or less on day 57.
The researchers found no significant difference in the rate of the primary end point of a clinical response between the sargramostim and placebo groups.
However, the team noted significantly more patients in the sargramostim group than in the placebo group reached the secondary end points of a clinical response of a decrease and remission of the Crohn's Disease Activity Index.
The rates of either type of clinical response and of remission were significantly higher in the sargramostim group on day 29 of treatment and 30 days after treatment.
The researchers also observed that the sargramostim group had significant improvements in the quality of life.
The research team found that mild-to-moderate injection-site reactions and bone pain were more common in the sargramostim group.
In addition, the team noted that 3 patients in the sargramostim group had serious adverse events possibly or probably related to treatment.
Dr Korzenik's team concluded, “This study was negative for the primary end point.”
“However, findings for the secondary end points suggest that sargramostim therapy decreased disease severity and improved the quality of life in patients with active Crohn's disease.”