HSCT for MS - we are ready for prime time
ECTRIMS Online Library. Burman J. 09/11/19; 279382; 30
Dr. Joachim Burman
Dr. Joachim Burman

Abstract: 30

Type: Hot Topic

Abstract Category: Hot Topic 4: HSCT and stem cell treatment in MS

J. Burman

Department of Neuroscience, Uppsala University, Uppsala, Sweden

Autologous hematopoietic stem cell transplantation (AHSCT) was first used for MS in 1995 and since then more than 2,500 patients have undergone this procedure in Europe. Initially, it was reserved for treatment resistant progressive forms of MS, with disappointing results. In the last decade a shift towards treatment of relapsing-remitting MS has occurred. It is a one-time procedure and a majority of patients do not need alternative treatment after AHSCT. The proportion of patients with 'no evidence of disease activity' (NEDA) has consistently been reported in the range of 68-70% at five years in several uncontrolled single-centre studies and nationwide surveys, which is superior to what has been reported for conventional disease modifying drugs (DMD). However, this evidence is weakened by the absence of control groups.Safety has always been a concern, in a meta-analysis of AHSCT for MS, treatment related mortality was 0.3%, which is comparable to routine surgical procedures. Recently, the outcome of a phase III randomized controlled trial (RCT) of AHSCT versus approved DMD treatment was reported. The trial confirmed the earlier reports on NEDA, which after treatment with AHSCT was considerably higher (78.5% at five years) than with DMD treatment (2.97% at five years). Furthermore, one in two patients treated with AHSCT improved in EDSS, whereas only one in thirteen improved with DMD. In the same trial, there was no deaths or grade 4 adverse events in either group, suggesting that AHSCT can be performed safely at experienced centres.At this point it is justified to say that AHSCT is superior to standard DMD treatment in terms of efficacy and it seems to be on par with other advanced therapies in terms of safety. Current evidence supports a more widespread use of this procedure.
Disclosure: Dr Burman reports receipt of grants from the Swedish Society for Medical Research, the Swedish Society for Medicine, Neuro Sweden and the Bissen Brainwalk Foundation.

By clicking “Accept Terms & all Cookies” or by continuing to browse, you agree to the storing of third-party cookies on your device to enhance your user experience and agree to the user terms and conditions of this learning management system (LMS).

Cookie Settings
Accept Terms & all Cookies