Schmidt, K. J. et al. Clinical trial: cyclophosphamide pulse therapya promising therapeutic alternative in refractory Crohn's disease. Aliment. Pharmacol. Ther. 29, 1230–1239 (2009).

Cyclophosphamide pulse therapy can induce remission of severe Crohn's disease in patients whose symptoms are refractory to traditional steroid treatment, a new study reveals. Research, led by Klaus Schmidt from the University Hospital of Schleswig-Holstein, Germany, found a “high efficacy of cyclophosphamide pulse therapy in these severely affected patients.”

“Therapeutic options in steroid-refractory, active Crohn's disease are still limited,” comments Schmidt. Antibodies to tumor necrosis factor are used as first-line therapy in such patients; however, “a considerable number of patients remain refractory, lose response or [become] intolerant to these biologicals,” Schmidt adds. Recent, noncontrolled studies indicated that cyclophosphamide pulse therapy, used to treat patients with vasculitis, can induce remission of steroid-refractory IBD without severe adverse effects.

Credit: Credit: K. Schmidt, University Hospital of Schleswig-Holstein

Schmidt and colleagues evaluated the efficacy and safety of cyclophosphamide pulse therapy—once-monthly pulses of 750 mg for 2–6 months (median 3 months)—in 15 patients with severe, steroid-refractory Crohn's disease. “The anti-inflammatory effects of cyclophosphamide pulse therapy could be observed after one infusion, [as] 47% of the patients [entered] remission at week 4,” says Schmidt. After 8 weeks, 10 patients (67% of the study population) entered remission, which lasted up to 40 months (median 16 months), and steroid-free remission was achieved in eight of the patients (54%). Overall, a clinical response was observed in 13 patients (87%). Treatment was well tolerated and no adverse effects were noted.

The researchers conclude that randomized trials are needed to confirm their encouraging results in relation to cyclophosphamide pulse treatment for IBD.