Emerging Therapies in Antineutrophil Cytoplasm Antibody-Associated Vasculitis

Shunsuke Furuta; David Jayne


Curr Opin Rheumatol. 2014;26(1):1-6. 

In This Article

Emerging Therapies With 'Conventional'-Type Drugs

Mycophenolate mofetil, an oral immunosuppressant, has been used as an alternative agent for relapsing disease and in remission maintenance.[6] As an induction agent mycophenolate mofetil has been studied in myeloperoxidase (MPO)-ANCA vasculitis in which it appears to compare well to cyclophosphamide in small studies.[7,8] A larger induction study with mycophenolate mofetil in AAV, the MYCYC trial (ClinicalTrials.gov number; NCT00414128) has finished and is under analysis now. It will provide further information.

Gusperimus (15-deoxyspergualin) inhibits mainly T-cell maturation and cytotoxic T-cell proliferation but also B cells. Two open label studies reported high response rates in refractory GPA.[9] Administered in 28-day cycles of daily subcutaneous injection with washout periods, it has required close monitoring to avoid cytopaenias. The SPARROW trial (ClinicalTrials.gov number; NCT01446211) is evaluating the efficacy of gusperimus in relapsing and refractory GPA.

Plasma exchange is a treatment option for severe AAV. Confirmation of the pathogenicity of ANCA has contributed to a therapeutic rationale for plasma exchange in AAV, although removal of other factors may also be important. Meta-analysis of plasma exchange trials suggests a beneficial effect on the risk of end-stage renal disease but no effect on mortality.[10] Current practice recommends plasma exchange for those presenting with severe renal disease or alveolar haemorrhage with little evidence supporting the latter indication. The ongoing PEXIVAS trial (ClinicalTrials.gov number; NCT00987389) is examining the effect of plasma exchange on patient and renal survival in those with a GFR below 50 ml/min or severe alveolar haemorrhage.[11]