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Old 09-09-2019, 11:40 AM
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Dave @ ActiveMSers
Join Date: Jun 2008
Location: Albuquerque, NM
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Default STUDY: HSCT vs Alemtuzumab (Lemtrada)

Therapeutic strategies for aggressive multiple sclerosis: a single center experience with alemtuzumab and autologous hematopoietic stem cells transplantation

G. Boffa1, C. Lapucci1, E. Sbragia1, E. Capello1, D. Currò1, A. Laroni1, L. Roccatagliata2, A. Uccelli1, F. Gualandi3, G. Mancardi1, M. Inglese1 1Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DiNOGMI), University of Genova, 2Department of Health Science (DISSAL), University of Genoa, Genoa, Italy/IRCCS AOU San Martino-IST, Genoa, Italy, 3Bone Marrow Transplantation Unit, San Martino Hospital, Genova, Italy, Genova, Italy

Background: Aggressive multiple sclerosis (MS) represents almost 10% of all MS cases. Treatment of these patients is challenging and optimal strategies have yet to be defined. Alemtuzumab and autologous hematopoietic stem cells transplantation (aHSCT) represent ideal candidates for managing aggressive MS because of their profound and immediate immune-ablative effects. However, experience is limited and uncertainty remains on correct patient selection.

Aims: To characterize and compare therapeutic strategies for aggressive MS in a real life single center experience.

Methods: 77 patients with aggressive MS, according to the criteria of Rush et al. [CA Rush et al. Nat Rev Neurol 2015], were retrospectively collected from the MS center of Genova (mean age=33 (±7) years, median EDSS=6 (1-8,5), RRMS 74%, females 68%). 30 patients underwent treatment with alemtuzumab and 47 with aHSCT (conditioning regimen: carmustine/etoposide/ara-C/melphalan (BEAM) or cyclophosphamide). The proportion of patients with complete disease remission (no evidence of activity -NEDA-) was calculated at 2 years.

Results: At baseline, aHSCT-treated patients showed higher ARR (p=0,02), worse EDSS (p< 0,001) and more pronounced MRI activity (number of Gd+ lesions at baseline, p< 0,001) than alemtuzumab treated patients. Proportion of patients achieving NEDA at 2 years was 78% and 70% for the aHSCT and alemtuzumab group, respectively. Relapses occurred more frequently in the alemtuzumab group (p=0,01), with no differences in EDSS at 2 years. Early adverse events were more frequent in the aHSCT group (p< 0,001). One death occurred in the aHSCT group, although the relation with the procedure is not clear. Late adverse events, especially autoimmune disorders were more frequent in the alemtuzumab group (p=0,04).

Conclusions: Alemtuzumab and aHSCT are effective treatment choices for aggressive MS, providing complete disease remission in a large proportion of patients at 2 years. aHSCT induces earlier disease remission compared to alemtuzumab.
Dave Bexfield
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