Aggressive multiple sclerosis: a single center, real‐world treatment experience

BACKGROUND: The best therapeutic approach for aggressive relapsing-remitting multiple sclerosis (RRMS) remains unknown. OBJECTIVE: To compare efficacy and safety of autologous haematopoietic stem cell transplantation (aHSCT) and alemtuzumab in aggressive RRMS. METHODS: Time-to-first relapse, time-to-confirmed disability worsening, time-to-first evidence of MRI activity and time-to-first evidence of disease activity were compared between the two treatment groups. Secondary outcomes included the 12,24 and 36 months annualized-relapse-rate (ARR) and the 6-months confirmed EDSS changes at month 12 and 24. RESULTS: Fifty-seven patients treated with aHSCT (n=25) or alemtuzumab (n=32) were included. At baseline, aHSCT patients had higher EDSS (median score of 6 versus 3; p<0.001), higher ARR (mean ARR of 3.2 versus 1.7; p=0.001) and a higher number of baseline T1-gadolinium enhancing lesions at MRI (mean number of 15.5 versus 1.6; p<0.001). NEDA-3 status was more frequently achieved in aHSCT-treated patients than in alemtuzumab-treated patients (75% versus 56% of patients at the end of the observation period; HR[95%CI]=0.27 [0.08-0.84]; p=0.023). aHSCT significantly reduced the risk of relapse (relapse-free survival of 84% versus 69%; HR[95%CI]=0.13 [0.02-0.63]; p=0.012) and MRI activity (MRI activity-free survival of 85% versus 59%; HR[95%CI]=0.13 [0.03-0.59] ; p=0.009). The ARR at 36 months was significantly lower in the aHSCT group (0.05 vs 0.35 p=0.02). A significant effect of aHSCT in promoting EDSS improvement compared with alemtuzumab was noted (p=0.035). CONCLUSIONS: Alemtuzumab and aHSCT are effective treatment choices for aggressive MS. aHSCT seems to be superior to alemtuzumab in inducing complete disease control and in promoting short-term disability improvement.