Long-Term Treatment of Refractory Wegener's Granulomatosis (WG) with 15-Deoxyspergualin (DSG): An Open Study in Seven Patients.

Objective: A subset of patients with WG is refractory to treatment with cyclophosphamide (CYC) plus glucocorticosteroids (GC). A 6 months course of the immunosuppressant DSG has previously been successfully employed in refractory WG. However, there are no reports in the literature on long-term treatment with DSG. Methods: To document the effects of long-term DSG treatment this follow-up study of a previous pilot trial reports on 7 patients suffering of refractory WG, who were successfully treated with DSG over an average of 26.5 (range: 11-55.5) months. Results: Before administration of DSG, patients had received a mean of 5.4 (2-11) different therapeutic approaches including GC and CYC in all and experimental therapies in 4, without control of disease activity (6.6 [range: 3-12] relapses during a disease duration of 4.7 [1.5-9] years). Four patients were unresponsive to CYC, and 3 did not tolerate it. All six suffered of active WG when DSG treatment was initiated (mean Birmingham vasculitis activity score: 16.1 [4-25]). DSG (0.5 mg/kg/day s.c.) was given for 2 to 3 weeks until the WBC count dropped to 3,000/µl followed by a rest until a WBC of 4,000/µl was reached again. No other immunosuppressants besides steroids were given. All patients showed a long lasting, favorable response to DSG with partial (n = 2) or complete (n = 5) remission. Only one of the cases relapsed while being treated with DSG. Termination/interruption of DSG treatment in 3 cases was followed by relapse in 4 of 5 occasions. Resumption of DSG lead to complete remission. Currently, 5 of the 7 patients are still treated with DSG and are in remission. Infections, mainly of the respiratory tract, were observed in 5 cases and required hospitalisation in one. This case developed a 3rd degree heartblock that required pacing. Otherwise, infections resolved under adequate treatment without sequels. Conclusions: Long-term treatment with DSG seems safe and successful in patients with refractory WG under careful monitoring of WBC counts. [ABSTRACT FROM AUTHOR]