Hematopoietic stem cell transplantation in multiple sclerosis

Intensive immunosuppresion followed by hematopoietic stem cell transplantation (HSCT) has been suggested as potential treatment in severe forms of multiple sclerosis (MS). Since 1995 ca. 400 patients have been treated with HSCT. Stabilization or improvement occurred in almost 70% of cases at least for 3 years post-transplant. Magnetic resonance revealed the capacity of autologous HSCT to suppress or markedly reduce gadolinium-enhancing lesions. The progression of brain atrophy declined after two years post-HSCT. The profound immunological changes following autologous HSCT may result in restoration of self-tolerance. Relatively young patients with active inflammatory lesions of relatively short duration and rapidly progressive disease, but still low disability scores, unresponsive to conventional therapy seem the best candidates for transplantation. Transplant-related mortality was 6% in the first EBMT report and 5.3% in the second one. No deaths were reported since 2001. Very high-intensity conditioning regimen is associated with higher risk of toxicity without significant increase in efficacy. The effects of transplantation and transplantation-related morbidity are dependent on patient-selection, time of transplantation and conditioning regimens used. This review is a comprehensive study of the results obtained in several single-center and multicenter studies. Patient characteristics, transplantations steps, toxicity and clinical outcome have been monitored and compared.