PERIPHERAL-BLOOD STEM-CELL TRANSPLANTATION IN THE TREATMENT OF PROGRESSIVE MULTIPLE-SCLEROSIS - FIRST RESULTS OF A PILOT-STUDY

Several experimental autoimmune diseases (AID), including allergic enc ephalomyelitis, ie the multiple sclerosis (MS) model, respond to TBI a nd chemotherapy followed by BMT, Remissions of AID may also occur in p atients with concomitant malignancies treated with allogeneic or autol ogous BMT, These observations have emphasized the possibility of treat ing AID with high-dose therapy and haematopoietic stem cell transplant ation (HSCT). In a phase I/II pilot study, 15 patients with progressiv e MS were treated with BEAM followed by autologous blood SCT and antit hymocyte globulin (ATG), Patients were severely disabled, with median EDSS and SNRS scores of 6 (5-7.5) and 42 (33-62), respectively. Cyclop hosphamide (4 g/m(2)) and G/GM-CSF (5 mu g/kg/day) were used for stem cell mobilization, which caused no neurotoxicity, On days +1 and +2, A TG (2.5-5 mg/kg) was given for in vivo T cell-depletion. Allergy (93%) and infections (87%) were the principal toxic complications, Mild, tr ansient, neurotoxicity was observed in six patients in the immediate p ost-transplant period, The median follow-up time is 6 months (6-18). D urable neurologic improvements have been detected on both the EDSS (7/ 15) and SNRS (15/15) systems, One patient worsened at 3 months and two have relapsed, Autologous HSCT appears feasible in MS; it does not ag gravate disability and seems to offer a clinical benefit, However, the se observations need confirmation and long-term outcomes will show if benefits counterbalance toxicity and cost.