TY - JOUR T1 - Deoxyspergualin in relapsing and refractory Wegener’s granulomatosis JF - Annals of the Rheumatic Diseases JO - Ann Rheum Dis SP - 1125 LP - 1130 DO - 10.1136/ard.2008.092429 VL - 68 IS - 7 AU - O Flossmann AU - B Baslund AU - A Bruchfeld AU - J W Cohen Tervaert AU - C Hall AU - P Heinzel AU - B Hellmich AU - R A Luqmani AU - K Nemoto AU - V Tesar AU - D R W Jayne Y1 - 2009/07/01 UR - http://ard.bmj.com/content/68/7/1125.abstract N2 - Objectives: Conventional therapy of Wegener’s granulomatosis with cyclophosphamide and corticosteroids is limited by incomplete remissions and a high relapse rate. The efficacy and safety of an alternative immunosuppressive drug, deoxyspergualin, was evaluated in patients with relapsing or refractory disease.Methods: A prospective, international, multicentre, single-limb, open-label study. Entry required active Wegener’s granulomatosis with a Birmingham vasculitis activity score (BVAS) ⩾4 and previous therapy with cyclophosphamide or methotrexate. Immunosuppressive drugs were withdrawn at entry and prednisolone doses adjusted according to clinical status. Deoxyspergualin, 0.5 mg/kg per day, was self-administered by subcutaneous injection in six cycles of 21 days with a 7-day washout between cycles. Cycles were stopped early for white blood count less than 4000 cells/mm3. The primary endpoint was complete remission (BVAS 0 for at least 2 months) or partial remission (BVAS <50% of entry score). After the sixth cycle azathioprine was commenced and follow-up continued for 6 months.Results: 42/44 patients (95%) achieved at least partial remission and 20/44 (45%) achieved complete remission. BVAS fell from 12 (4–25), median (range) at baseline to 2 (0–14) at the end of the study (p<0.001). Prednisolone doses were reduced from 20 to 8 mg/day (p<0.001). Relapses occurred in 18 (43%) patients after a median of 170 (44–316) days after achieving remission. Severe or life-threatening (⩾ grade 3) treatment-related adverse events occurred in 24 (53%) patients mostly due to leucopaenias.Conclusions: Deoxyspergualin achieved a high rate of disease remission and permitted prednisolone reduction in refractory or relapsing Wegener’s granulomatosis. Adverse events were common but rarely led to treatment discontinuation. ER -