An Open-Label Pilot Study of Granulocyte Colony-Stimulating Factor for the Treatment of Severe Endoscopic Postoperative Recurrence in Crohn’s Disease

Abstract

Background/Aim: Recombinant human granulocyte colony-stimulating factor (rhG-CSF) promoted healing of Crohn’s disease (CD)-like intestinal lesions in chronic granulomatous disease and glycogen storage disease Ib, both characterized by defective neutrophil functions. We performed a prospective, open-label pilot study with rhG-CSF for the treatment of CD. Patients andMethods: Five patients with clinically inactive CD, but with severe endoscopic ileitis within 1 year after intestinal resection and ileocolonic anastomosis, received 300 µg of rhG-CSF (Filgrastim; Neupogen^®) subcutaneously, three times weekly for a total of 12 weeks. Safety was evaluated by assessment of clinical and laboratory data and disease activity. The primary parameter of efficacy was complete mucosal healing, as defined by the Rutgeerts score. Anti-inflammatory mediators were repeatedly measured during treatment. Results: All patients completed the protocol in clinical remission. In 1 subject transient headache resolved after halving the rhG-CSF dosage. Complete mucosal healing was observed in 2 patients: in 1 patient after 12 weeks of therapy and in 1 patient 9 months after treatment cessation. In a single patient, closure of an anovaginal and of a perianal fistula was noted. Neutrophil counts and interleukin-1 receptor antagonist and soluble tumor necrosis factor receptor p55 and p75 levels were found to be increased during drug administration. Conclusion: rhG-CSF seems to be safe, well tolerated, and might provide efficacy in CD.