Hematopoietic stem cell transplantation (HSCT) for primary systemic vasculitis and related diseases

Primary systemic vasculitis (PSV) as well as the related disorders behcet disease (BD) and relapsing polychondritis (RP) are a heterogenous group of autoimmune diseases with the potency for a severe and life threatening course. Patients with PSV seem to be ideal candidates for HSCT: First, when first line treatment has failed the patients are in high risk of severe organ damage or death due to active disease or treatment toxicity. Moreover, the chance to achieve complete remission with standard doses of cyclophosphamide-still standard treatment of severe manifestations of PSV- predicts that dose escalation to high dose chemotherapy will increase the effectiveness. The experience with HSCT in patients with severe PSV as published in case reports and from EULAR and EBMT-databases gives preliminary evidence that HSCT might be an effective treatment option in refractory cases of PSV and related diseases. Validated scores of disease activity and damage such as the Birmingham vasculitis activity score (BVAS) and the vasculitis activity damage index (VDI) could help to identify patients which might profit from HSCT.