Long-term HSCT Outcomes in Norway

Research Summary: Autologous hematopoietic stem cell transplantation for multiple sclerosis: Long-term follow-up data from Norway

Although not a new treatment approach, haematopoietic stem cell transplantation (HSCT) has gained a lot of attention over recent years as a potential therapy for multiple sclerosis (MS). However, as there are significant risks associated with this treatment, it is important that we have a clear understanding of who will be the most likely to benefit and the long-term outcomes that can be generated.

What?

The study looked to assess the long-term outcomes of people living with relapsing-remitting multiple sclerosis (RRMS) that received a haematopoietic stem cell transplant (HSCT) between January 2015 and January 2018 at a single centre in Norway.

Who?

This research was conducted by a team at the Haukeland University Hospital and the University of Bergen in Norway.

Where?

The article was published in the Multiple Sclerosis Journal.  The full study is currently freely accessible and can be accessed here. 

When?

The study was first published online in the May 2024 edition of the journal.

BACKGROUND #1: HSCT works by using chemotherapy to eliminate an individual’s existing immune system and then using their own stem cells to reboot it.  It is thought that by removing the autoreactive immune cells, which are responsible for causing damage in MS, it may be possible to stop disease progression and provide long-term benefits.

BACKGROUND #2:  Although HSCT has shown benefits for some people living with MS, it is still unclear exactly where it fits into the MS treatment landscape.  In most places around the world, it is used in people that have a highly aggressive disease course that does not respond to other high-efficacy therapies.

FINDING #1:  The study involved 29 people with RRMS that had HSCT between January 2015 and January 2018.  The participants had an average disease duration of around 5.5 years and moderate disability (average EDSS of 2.9).  Most of the participants had failed 2 or more therapies before receiving HSCT.  All participants received an intermediate intensity (non-myeloablative) chemotherapy regimen.

FINDING #2:  69% (20/29) of participants experienced no evidence of disease activity after receiving HSCT.  As well as this, 11 participants also had an overall reduction in disability, as measured by the EDSS.  It was found that having a higher EDSS before the treatment was more likely to result in experiencing relapses, new lesions on MRI or disease progression after the transplant. 

FINDING #3: The study also looked at any outcomes relating to the employment status of the participants.  While only one person was working full-time prior to HSCT, this number had increased to 10 after 2 years and 15 by 5 years post-transplant.

FINDING #4: The study also reported some adverse events following HSCT.  There were 5 cases of autoimmune thyroid conditions that developed.  As well as this, 13 of the 21 female participants experienced ovarian failure.  However, 5 children were also born to women who had undergone the treatment (4 of which were naturally conceived).

THOUGHT #1:  This study provides further evidence for the long-term benefits that can be obtained from the use of HSCT in some people living with relapsing-remitting multiple sclerosis.  Importantly, this study saw sustained effects using a lower intensity chemotherapy approach and there were no deaths as a result of the treatment.  This suggests that it is possible to still generate the desired outcomes without needing the strongest chemotherapy approach (which when used increases the risks associated with the procedure).

THOUGHT #2: The findings of this long-term follow-up also continue to indicate that there are certain characteristics that make a person living with MS more likely to obtain benefits from receiving HSCT.  As shown in the results of other trials, this seems to be people that have a short disease duration, highly active disease and a lower EDSS.

THOUGHT #3:  The adverse events reported in this study are also important to keep in mind.  With all new trial results for HSCT, we get a better understanding of the benefits and risks that come with the procedure.  By having as much information as possible about all of the potential outcomes, it gives greater clarity to neurologists and people living with MS, allowing an informed decision to be made.

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Other articles we have published on the topic of HSCT can be revisited here.