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Clinical Trials in Vasculitis

Seerapani Gopaluni1, David Jayne1

  • 1Lupus and Vasculitis Clinic, Addenbrooke's Hospital, Cambridge University Hospitals, Cambridge, CB2 0QQ UK.

Current Treatment Options in Rheumatology
|September 23, 2017
PubMed

Insights

Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) treatment involves induction and maintenance phases. Clinical trials guide options like cyclophosphamide, rituximab, and glucocorticoids, improving patient outlooks.

Area of Science:

  • Rheumatology and Immunology
  • Clinical Trials and Therapeutics

Background:

  • Systemic vasculitides are rare, complex inflammatory diseases of blood vessels with limited treatment evidence.
  • International collaboration has enabled clinical trials, particularly in Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV).
  • Understanding AAV pathogenesis and biomarkers remains incomplete, highlighting the need for evidence-based treatment strategies.

Purpose of the Study:

  • To review current treatment options for AAV based on clinical trial evidence.
  • To discuss induction and maintenance therapy strategies tailored to disease severity.
  • To highlight advancements and remaining questions in optimizing AAV management.

Main Methods:

  • Review of clinical trial data for Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) treatments.
  • Analysis of evidence for induction therapies including cyclophosphamide, rituximab, plasma exchange, and IVIGs.
  • Evaluation of maintenance therapies such as azathioprine, methotrexate, mycophenolate mofetil, and rituximab.

Main Results:

  • Cyclophosphamide and high-dose glucocorticoids are standard for AAV induction in generalized disease.
  • Rituximab is an effective alternative to cyclophosphamide, especially for relapsing or intolerant patients.
  • Azathioprine or methotrexate are preferred for maintenance; mycophenolate mofetil is an alternative. Rituximab use in maintenance is increasing.

Conclusions:

  • Treatment for AAV is stratified into induction and maintenance phases, guided by disease severity and clinical trial outcomes.
  • Significant progress has been made, with rituximab offering a key alternative and increasing use in both induction and maintenance.
  • Optimal AAV treatment strategies require further research to address remaining unanswered questions.
Keywords:
ANCAANCA-associated vasculitisAzathioprineClinical trialsCyclophosphamideGlucocorticoidsMycophenolate mofetilPlasma exchangeRituximabTreatmentVasculitis

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