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

Rheumatology (Oxford). 2010 Mar;49(3):556-62. doi: 10.1093/rheumatology/kep411. Epub 2009 Dec 23.

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 Other immunosuppressants except corticosteroids were withdrawn and DSG, 0.5 mg/kg/day, self-administered subcutaneously for up to 21 days, in 28-day cycles.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 x 10(9)/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.

Publication types

  • Clinical Trial

MeSH terms

  • Adult
  • Aged
  • Drug Administration Schedule
  • Drug Therapy, Combination
  • Female
  • Glucocorticoids / administration & dosage
  • Granulomatosis with Polyangiitis / drug therapy*
  • Guanidines / administration & dosage*
  • Guanidines / adverse effects
  • Guanidines / therapeutic use
  • Humans
  • Immunosuppressive Agents / administration & dosage*
  • Immunosuppressive Agents / adverse effects
  • Immunosuppressive Agents / therapeutic use
  • Injections, Subcutaneous
  • Male
  • Middle Aged
  • Prednisolone / administration & dosage
  • Recurrence
  • Remission Induction
  • Treatment Outcome
  • Young Adult

Substances

  • Glucocorticoids
  • Guanidines
  • Immunosuppressive Agents
  • Prednisolone
  • gusperimus