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The rationale and the existing clinical data using haematopoietic stem cells in MS
ECTRIMS Online Library. Muraro P. Oct 12, 2018; 232036
Paolo A. Muraro
Paolo A. Muraro
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Abstract: 283

Type: Educational Session

Abstract Category: N/A

Introduction: In recent years, autologous haematopoietic stem cell transplantation (AHSCT) has been increasingly used to treat MS by 'rebooting' the immune system and stopping it from attacking nervous tissue. Recent evidence demonstrates that AHSCT markedly reduces relapse rates, lesion development and improves disability.
Objectives: To inform and educate the participants on the immunological principles and on the clinical evidence underlying the use of AHSCT as treatment for people with MS.
Aims:
1. To review the immunological rationale for using and developing AHSCT as one form of treatment strategy for MS
2. To summarise the clinical evidence of AHSCT with MS
3. To introduce treatment schemes and optimal patient selection criteria
4. To discuss the recent and ongoing progress in randomized clinical trials of AHSCT in MS
Methods: Literature review and meta-analysis of immunological and clinical data
Results:
1. Studies on the reconstitution of adaptive immune repertoires support the notion of an immune resetting after AHSCT and have identified mechanisms potentially underlying the treatment effect: depletion of proinflammatory T cells (Darlington et al. Annals of Neurology 2013; Abrahamsson et al Brain 2013) and regeneration of new T cells (Muraro et al J Clin Invest 2014)
2. Clinical studies have demonstrated profound suppression of MS activity (Burt et al. JAMA 2014, Atkins et al Lancet 2015, Nash et al Neurology 2017) and long-term durability of clinical stabilisation (Muraro et al JAMA Neurology 2017). Refinement of treatment criteria and protocols has reduced the toxicities and treatment-related mortality to currently below 0.5% (Sormani, Muraro et al. Neurology 2017).
3. Clinically based-criteria have recently defined the optimal patient profile for who AHSCT could be considered an appropriate treatment option (Muraro et al Nature Reviews Neurology 2017).
4. Randomised clinical trials (RCTs) against standard therapy reported superior effectiveness of AHSCT (Mancardi et al. Neurology 2014; Burt RK et al, AAN 2018 URL: http://n.neurology.org/content/90/15_Supplement/S36.004). RCTs of AHSCT against current highly effective therapy are being developed.
Conclusions: Increasing clinical and mechanistic evidence supports considering AHSCT as a treatment for patients with aggressive, inflammatory forms of MS. Further clinical and mechanistic work is underway to improve the indication for treatment and the understanding of the mode of action.
Disclosure: Professor Muraro discloses travel support and speaker honoraria from Bayer HealthCare, Bayer Pharma, Biogen Idec, Merck-Serono and Sanofi Aventis.

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