Autologous hematopoietic stem cell transplantation (ahsct) in multiple sclerosis: a phase ull clinical trial

A phase I/II clinical trial on autologous hematopoietic stem cell transplantation (AHSCT) was conducted hy 15 Italian neurological and hematological centers after reaching a consensus on patient selection criteria, primary endpoint and treatment regimen. The study was designed to investigate magnetic resonance imaging (MRI) and laboratory changes following autologous PBPC transplantation in MS patients with an EDSS in the 5.0-6.5 range, with documented clinical signs of rapid progression during the previous year, who had been refractory to conventional treatments. Other endpoints were the evaluation of treatment safety and progression of disease. At the present time, nine patients have completed the autologous transplantation and one additional case has been included. MRI exams of seven transplanted patients revealed the complete disappearance of gadolinium-enhancing lesions in the months following the conditioning regimen. A marked decrease of the number of enhancing lesions was already apparent after the mobilization treatment, but clinical status remained unchanged or improved only slightly. Treatment was well tolerated and no serious adverse events were recorded, except for cytomegalovirus infection in three cases. The present data suggest that AHSCT abrogates MRI disease activity in severe secondary progressive MS cases.