Frau J, et al. - In this study, long-term effect and safety of hematopoietic stem cell transplantation (HSCT) in multiple sclerosis (MS) were evaluated. The researchers confirmed the long-term efficacy of HSCT in reducing relapses and disability progression. The risk/benefit profile was better for intermediate intensity regimens

Methods

• The researchers included patients referred to the MS centre of Cagliari and undergoing HSCT.
• By using Wilcoxon test, variations in relapses and EDSS before and after HSCT were evaluated.
• They made a descriptive analysis for other clinical data.

Results

• From 1999-2006, 9 patients underwent HSCT(female 6, males 3; 5 relapsing-remitting, 2 secondary progressive, 1 primary progressive, and 1 progressive relapsing).
• In this study, the median follow-up was 11 years (11-18).
• The researchers performed aHSCT in 8 patients, 7 using a low intensity conditioning regimen, and 1 an intermediate intensity.
• Due to onco hematological disease, the primary progressive underwent allogeneic HSCT.
• Compared to the 2 preceding years, the relapses number decreased in the 2 years following the procedure (p=0.041).
• They observed new relapses or disease progressions after a range of 7 (low intensity regimen)-118 (intermediate intensity) months.
• The EDSS was stable in 2 patients, improved in 2, and worse in 5 (maximum 2 EDSS in 1 patient), at last follow-up.
• After a mean of 23.3 and 19.8 months, 6 patients showed new lesions, and 7 gadolinium-enhancing on brain MRI, respectively.
• Melanoma, and progressive multifocal leukoencephalopathy were the 2 serious adverse events.