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Old 10-01-2018, 06:47 PM
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Location: Albuquerque, NM
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Default HSCT as a first line disease modifying therapy in patients with 'aggressive' MS


The use of autologous haematopoietic stem cell transplantation as a first line disease modifying therapy in patients with 'aggressive' multiple sclerosis

J. Das, J. Snowden, J. Burman, et al

Introduction: Autologous haematopoietic stem cell transplantation (AHSCT) is a very effective treatment in patients with highly active relapsing remitting multiple sclerosis (MS) who failed to respond to standard disease modifying therapies (DMTs). European Society for Blood and Marrow Transplantation guidelines advocate its use as a first line treatment in patients with 'aggressive' MS.

Objectives/Aims: To assess the safety and efficacy of AHSCT as a first line DMT in patients with 'aggressive' MS.

Methods: A total of 20 patients with 'aggressive' MS received AHSCT in 5 centres [7 from Sheffield (UK), 7 from Uppsala (Sweden), 4 from Ottawa (Canada), 1 from Florence (Italy) and 1 from Chicago (USA)]. None received any standard DMTs for more than 3 months before AHSCT. BEAM (carmustine, etoposide, cytarabine, melphalan) with antithymocyte globulin (ATG), cyclophosphamide with ATG or combination of cyclophosphamide, ATG and busulphan were used as conditioning regimens.

Results: Median age at diagnosis was 33 (19 - 52) years. Median pre-treatment EDSS score was 6.5 (1.5 - 9.5). Median follow up was 30 (6 - 118) months. Median EDSS score at the last follow up was 2.0 (0 - 6.5). Median improvement between pre-treatment and last follow up EDSS scores was 2.5 (0 - 8), which was statistically significant (p < 0.05, Wilcoxon signed-rank test). None had any clinical relapse following AHSCT. Three patients were noted to have new T2 lesions with or without gadolinium enhancement during the first follow up MRI, but no further new or enhancing lesions were observed in any subsequent scans. There were routine toxicities, but no treatment related mortality.

Conclusions: AHSCT was safe and highly effective in inducing rapid and sustained remission in this cohort and was associated with a significant improvement of patients' level of disability. This demonstrates the potential role of AHSCT as first line therapy in 'aggressive' MS.
Dave Bexfield
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