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aHSCT Shows Effectiveness When Compared with Three Other MS Therapies in Novel Study

May 22, 2023

The use of autologous hematopoietic stem cell transplantation (aHSCT) was as effective, and in some cases, more effective than the use of MS disease-modifying therapies (DMTs) in a novel study reviewing records of 4,915 people with MS. These results add to growing evidence of the value of this procedure for a subset of people with MS. Further resources for understanding the benefits and risks of this complex procedure are available below.
  • Background: There is growing evidence that aHSCT, commonly known as bone marrow transplant, can be highly effective for people with relapsing MS who meet specific characteristics. AHSCT attempts to “reboot” the immune system, which mistakenly attacks the brain, spinal cord and optic nerves in MS, damaging them.

    In this procedure, stem cells from a person’s own blood or bone marrow, are removed from the body, collected and frozen. Then, much of the person's immune system is depleted using chemotherapy drugs. Finally, the stored stem cells are reintroduced into the body where they rebuild the immune system. Research is ongoing to compare the effectiveness of aHSCT versus approved DMTs, to help determine its optimal place in managing MS.
  • This Study: The “gold standard” for comparing how effective therapies are is a clinical trial where participants are randomly assigned to one therapy or the other. This team used a unique study design called an “emulated” clinical trial, wherein they analyzed the medical records of individuals with MS who had been treated with aHSCT, fingolimod, natalizumab, or ocrelizumab over the course of five years.

    Data were obtained from six transplant centers (in Canada, Sweden, the United Kingdom, Norway and Australia), and from 94 centers in 27 countries contributing to the international MSBase registry.
  • Results: Investigators identified 4,915 people for this study, of whom 167 had received aHSCT. By comparing medical records, the team found suggestions that aHSCT prevented MS relapses significantly more than fingolimod, marginally more than natalizumab and similarly to ocrelizumab. In terms of preventing disability worsening, aHSCT showed similar effectiveness to the disease-modifying therapies. AHSCT reversed some disability (as measured by the standard EDSS scale of physical disability) significantly more than fingolimod and natalizumab, and similarly to ocrelizumab.

    Safety was evaluated in the aHSCT group. During treatment with chemotherapy drugs, common side effects were excessive loss of white blood cells and allergic reactions. One person died due to a blockage of veins in the liver caused by the medication busulfan. After transplant, 58 people experienced serious side effects, mainly infections.
  • The Meaning: This novel study adds to growing evidence of the value of aHSCT in the management of certain individuals with MS. However, randomized, controlled trials remain the best method for determining the optimal use of this procedure and how it compares with other high efficacy MS disease-modifying therapies. One such trial – the BEATMS study – is ongoing and recruiting people with active relapsing MS.
Comparative Effectiveness of Autologous Hematopoietic Stem Cell Transplant vs Fingolimod, Natalizumab, and Ocrelizumab in Highly Active Relapsing-Remitting Multiple Sclerosis” by the MSBase Study Group Collaborators is published in JAMA Neurology (Published online May 15, 2023).
Resources for Understanding aHSCT
Get the basics on aHSCT, including what happens during treatment and how to find an accredited treatment center
What to know about autologous hematopoietic stem cell transplantation” from Momentum Magazine
Watch a webinar, “Ask an MS Expert: Stem Cell Transplant in MS

About Multiple Sclerosis

Multiple sclerosis is an unpredictable disease of the central nervous system. Currently there is no cure. Symptoms vary from person to person and may include disabling fatigue, mobility challenges, cognitive changes, and vision issues. An estimated 1 million people live with MS in the United States. Early diagnosis and treatment are critical to minimize disability. Significant progress is being made to achieve a world free of MS.

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