Therapeutic advances in the treatment of vasculitis

Abstract

Considerable therapeutic advances for the treatment of vasculitis of the young have been made in the past 10 years, including the development of outcome measures that facilitate clinical trial design. Notably, these include: a recognition that some patients with Kawasaki Disease require corticosteroids as primary treatment combined with IVIG; implementation of rare disease trial design for polyarteritis nodosa to deliver the first randomised controlled trial for children; first clinical trials involving children for anti-neutrophil cytoplasmic antibody (ANCA) vasculitis; and identification of monogenic forms of vasculitis that provide an understanding of pathogenesis, thus facilitating more targeted treatment. Robust randomised controlled trials for Henoch Schönlein Purpura nephritis and Takayasu arteritis are needed; there is also an over-arching need for trials examining new agents that facilitate corticosteroid sparing, of particular importance in the paediatric population since glucocorticoid toxicity is a major concern.

Fig. 1

Recommended clinical guideline for the management of Kawasaki disease. Adapted from reference 31. *Treatment can be commenced before 5 days of fever if sepsis excluded; treatment should also be given if the presentation is > 10 days from fever onset if there are signs of persistent inflammation; **Kobayashi risk score ≥5 points X Refer to paediatric cardiologist; ¶ Other specific interventions such as PET scanning, addition of calcium channel blocker therapy, and coronary angioplasty at discretion of paediatric cardiologist. + Other immunomodulators may include ciclosporin. ♥For infants, Z score for internal coronary artery diameter >7 based on Montreal normative http://parameterz.blogspot.co.uk/2010/11/montreal-coronary-artery-z-scores.html

Fig. 2

High power view of skin biopsy taken from an 8 week old infant with CANDLE syndrome caused by digenic mutation in PSMB4 and PSMB9. There is a florid leukocytoclastic cutaneous vasculitis with karyorrhectic debris. In most areas distinct vessels could not be identified due to the destructive vasculitic process

Fig. 3

Chronic scarring of the pinna in a 12-year old boy with SAVI