Statkute L et al. - Non-myeloablative autologous haematopoietic stem cell transplantation (HSCT) is an alternative therapy for select patients with systemic vasculitis (SV) refractory to conventional immunosuppressive therapies. Methods
A report of treating 4 pts with refractory SV employing non-myeloablative autologous HSCT
4 pts with refractory SV were involved in phase I clinical trial of high dose chemotherapy and autologous HSCT
Peripheral blood stem cells were mobilised with cyclophosphamide (Cy) and granulocyte-colony stimulating factor (G-CSF)
Conditioning regimen consisted of Cy 200 mg/kg and rabbit anti-thymocyte globulin 5.5 mg/kg intravenously (iv)
Results
All 4 pts tolerated HSCT well w/o transplant related mortality or any significant toxicity
At median f/u of 28 mos all pts were alive
3 pts entered a sustained remission at 6, 6 and 24 mos, respectively, after transplant
They had decrease in disease activity and disease or treatment related damage
All 3 pts who achieved remission discontinued immunosuppressive therapy at the time of transplant and have not required treatment since
1 patient with Behcet disease and positive for human leukocyte antigen (HLA)-B51 has not improved after HSCT