Long-term treatment of relapsing Wegener’s granulomatosis with 15-deoxyspergualin

Abstract

Objective. To determine the safety and efficacy of prolonged treatment with 15-deoxyspergualin (DSG, gusperimus) in patients with relapsing WG.

Methods. Patients with relapsing WG treated with DSG were studied. Other immunosuppressants except corticosteroids were withdrawn and DSG, 0.5 mg/kg/day, self-administered subcutaneously for up to 21 days, in 28-day cycles. The cycle was terminated early for white blood cell count <4 × 10⁹/l. The prednisolone dose was adjusted according to the clinical state. End points were disease remission, relapse, Birmingham Vasculitis Activity Score (BVAS), prednisolone dose and safety.

Results. Eleven patients, five (45%) of whom were female, received a total of 15 treatment periods with DSG. The median (range) duration of each treatment period was 6.8 (3.3–15.9) months. Ten (90.9%) patients responded in 13/15 courses after a median of 1.7 (0.7–2.7) months and six (54.5%) achieved remission after 7.7 (1.9–13.5) months. Two (18.2%) patients relapsed while continuing to receive DSG. Remission was maintained in other patients while DSG was continued. However, 7/8 relapsed after DSG withdrawal. The median BVAS fell from 10 (3–22) at baseline to 3 (0–16) at the end of each treatment period (P = 0.002). Median prednisolone doses were reduced from 20 (5–30) mg/day at baseline to 10 (5–25) mg/day at the end of each treatment period (P = 0.052). Three severe adverse events occurred in two patients.

Conclusions. Extended treatment with DSG was effective in the majority of patients with relapsing WG and permitted prednisolone reduction. There was no unexpected toxicity associated with prolonged DSG administration.

Deoxyspergualin, Gusperimus, Wegener’s granulomatosis, Vasculitis, Refractory disease, Cyclophosphamide