Real World Application of AHSCT in MS

Written by Brett Drummond (MStranslate co-founder), in collaboration with Joey Lambert (MStranslate intern)

Autologous haematopoietic stem cell transplantation (AHSCT) is an exciting, recently developed treatment for multiple sclerosis (MS). AHSCT aims to ‘reboot’ the immune system of a person living with MS, in order to stop the autoimmune attack of the myelin sheath.  We have previously published an overview of the process that you can access here.  As we know that this treatment is of great interest to our community, we will continue to bring you updates on new research in this field.  A summary of one recently published study can be found below.

Research Summary: Real‑world application of autologous hematopoietic stem cell transplantation in 507 patients with multiple sclerosis

What?

The study investigated the results of AHSCT in people living with MS up to five years post treatment to better understand the associated risks and to help determine who may benefit the most from this therapeutic approach.

WHO?

The research was conducted by a team from Northwestern University, Chicago, USA.

WHERE?

The study was published in the Journal of Neurology. The full article is currently open-access and can be viewed here.

WHEN?

The study was published on the 11th of October, 2021.

BACKGROUND

1. Previous trials of AHSCT have suggested that it can be an effective potential treatment for some people living with MS.

2. Studies have indicated that the treatment may be most beneficial in individuals that are early in their disease course, have highly active disease and have failed other powerful treatment options.

FINDINGS

1.  Participants with relapsing-remitting multiple sclerosis (RRMS) were shown to have a slowing, and possible reversal, of disease progression over a 5 year period after AHSCT treatment.  This was measured through measuring their Expanded Disability Status Scale (EDSS).  The average EDSS went from 3.87 before treatment to 2.19 after 5 years post-treatment.

2.  There was no significant reduction in EDSS in participants with secondary progressive multiple sclerosis (SPMS) across the 5 years post-treatment.  The average EDSS in this group was 5.09 before treatment and 4.72 after 5 years post-treatment.  

3.  The safety of the procedure was similar to what has been observed in other AHSCT studies.  The most common side-effect was infection, which is to be expected with this treatment.  Unfortunately, there was one death due to an infection picked up in hospital.  No other deaths were reported in relation to the treatment.  There was some incidences of secondary autoimmune diseases developing, particularly thyroid issues, although these resolved with treatment.

THOUGHTS

1.  This study adds further evidence to the growing body of trial data that suggests that AHSCT is a therapy that may be effective at providing long-term stability (and maybe improvement) over an extended period of time for some people living with multiple sclerosis, that have failed other treatments.

2.  This data also suggests that the treatment may deliver better results for people living with relapsing-remitting multiple sclerosis than those with secondary progressive MS.  As mentioned above, this is in line with other studies that have suggested that people earlier in their disease course, and with active disease, are most likely to derive the greatest benefits.  However, I think we should not completely discount the lack of disease progression across 5 years in a group of people living with SPMS.  While their EDSS may not have improved, the fact that they have not gotten worse across this period of time is worth investigating further.  As this study does not have any controls, it is hard to know exactly how much to read into these results.  A trial comparing participants undergoing AHSCT with other participants on highly-effective therapies, as well as potentially no treatment at all, would allow us to better understand these findings.

3.  The idea that people may see some improvement in EDSS after AHSCT is interesting.  I think we need some more information before making too many conclusions about this.  We need to try and understand whether or not that this reduction in EDSS is just due to a normal process that may be occurring in RRMS, or is an actual response to the treatment.

If you are interested in watching a video explanation of this same study, you can do so on our YouTube channel by clicking here.