Autologous stem cell transplantation for resistant systemic vasculitis

A recent report describes non-myeloablative autologous haematopoietic stem cell transplantation (HSCT) as an effective approach for selected patients with treatment resistant systemic vasculitis. The investigators describe their report as the first single-centre trial to use a standard protocol for HSCT treatment and follow-up of patients with systemic vasculitis. The protocol included mobilisation of peripheral blood stem cells with cyclophosphamide and granulocyte-colony stimulating factor (G-CSF) and a conditioning regimen of cyclophosphamide 200 mg/kg and rabbit anti-thymocyte globulin 5.5 mg/kg. All four patients were evaluated at three, six and 12 months after HSCT, then annually. No new significant toxicities were reported. Three patients, one with Behcet disease, one with Sjögren syndrome, and one with Wegener granulomatosis, entered a sustained remission at six, six and 24 months, respectively, after transplant. Each of these three subjects had a significant decrease in disease activity and disease or treatment related damage, as measured by the Birmingham Vasculitis Activity Score and Vasculitis Damage Index, respectively. Also all three patients discontinued immunosuppression at the time of transplant and have not required treatment since. A further patient with Behcet disease, who was positive for human leukocyte antigen (HLA)-B51 was lost to follow-up at 22 months after HSCT and when last seen was not in remission and was still receiving immunosuppressive therapy. The investigators suggest, "non-myeloablative autologous HSCT is an alternative therapy for select patients [with systemic vasculitis] refractory to conventional immunosuppressive therapies" and add, "the selection of patients for autologous HSCT should be aimed to potentially reversible symptoms, not to specific diagnoses per se." Reference Statkute, L. Oyama, Y. Barr, W. et al. 2008, ‘Autologous non-myeloablative haematopoietic stem cell transplantation for refractory systemic vasculitis’ Annals of the Rheumatic Diseases, vol. 67, pp. 991-997....