HSCT Trial Shows Positive Results for RRMS

Research Summary: Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis: A Randomized Clinical Trial

In mid-January, we provided an update on another major piece of multiple sclerosis research that was published.  This trial provided insights into how HSCT compared to other currently available MS treatment options. You can read the text summary below or watch Brett provide a video summary of this research at the base of this article.

WHAT?

A clinical trial investigating the impact of haematopoietic stem cell transplantation (HSCT) as a treatment option for people living with relapsing-remitting MS (RRMS) and comparing it to current disease modifying therapies (DMTs) for multiple sclerosis.

WHO?

The research was led by Northwestern University, but involved four clinical centres across the US, Europe and South America.  

HOW?

The study involved 110 people with RRMS, who were randomly chosen to either be included in the group that received HSCT or a currently available MS treatment.  

WHERE/WHEN?

The study was published in JAMA (the third highest ranked medical journal in the world) in January, 2019.

FINDING #1:  The group that received the HSCT treatment had delayed disease progression compared to the group taking disease-modifying therapies (see Thought #2 for why we need to be careful about this comparison).

FINDING #2:  Only 8 out of 53 people that had HSCT experienced a relapse within the first 5 years after the treatment was undertaken.

FINDING #3:  There was a suggestion that HSCT treatment actually reduced the EDSS within the first 6-12 months after the therapy and resulted in a reported improvement in quality of life.

THOUGHT #1:  This study provides further evidence to suggest that HSCT can be an effective therapy for people living with RRMS, especially those with highly active, inflammatory disease.  It will be important to confirm these results with larger trials in the future, though the number of studies showing this is now quite extensive.

THOUGHT #2: It is difficult to really assess how HSCT compares to other MS therapies from this trial.  This is due to the fact that the treatment group contained people on many different therapies, which we know will have different levels of effectiveness.  Similarly, the participants weren’t blinded to which group they were in (something that is very difficult for a trial of HSCT) and so the possibility of placebo effect, especially for some of the participant reported outcomes, can’t be ruled out.

The abstract for this study can be read in full here.

VIDEO SUMMARY