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HSCT Shows Benefits in Some People with Secondary Progressive MS in Small Study

February 10, 2020

Researchers from Florence, Italy report on a small, open-label study involving 26 people with moderate to severe secondary progressive MS who underwent HSCT (Hematopoietic Stem Cell Transplantation). Most showed signs of clinical or MRI inflammatory activity in the year prior to the procedure. Five years after the procedure, 42% of participants were stable with no further progression of disability that was reduced to 30% 10 years after transplant. No relapses or inflammatory activity occurred on MRI scans after treatment. These results suggest that HSCT might be appropriate in a subgroup of people with SPMS that have significant inflammatory activity as measured by MRI. Further study in larger numbers are needed to understand who among those with secondary progressive MS might benefit from HSCT.
  • HSCT  attempts to “reboot” the immune system, which is involved in damaging the brain and spinal cord in MS. In HSCT for MS, hematopoietic (blood cell-producing) stem cells, which are derived from a person’s own (scientifically referred to as “autologous”) bone marrow, are collected and stored, prior to depleting much of the immune system using chemotherapy drugs. Then the stored hematopoietic stem cells are reintroduced to the body and over time reconstitute the immune system.
  • The primary outcome of this study was absence of further disease progression, which was achieved in 42% of participants at 5 years, and 30% at 10 years. In another 10 participants, disease progressed once, and then stabilized. The other 6 experienced disease progression to severe disability.
  • No deaths or life-threatening complications occurred. Two people developed autoimmune thyroid inflammation, and one person who had previously been treated with cell-inhibiting therapies developed myelodysplastic syndrome (a type of cancer). The most common adverse events were fever, gastrointestinal effects, and viruses/infections.
  • In HSCT, conditioning (immunosuppressive) regimes can vary from low to medium to high intensity. This study used an intermediate-intensity regimen known as BEAM. A new HSCT trial using the BEAM regimen is recruiting participants with relapsing-remitting MS.
"Impact of autologous haematopoietic stem cell transplantation on disability and brain atrophy in secondary progressive multiple sclerosis" was published by Drs. Alice Mariottini, Luca Massacesi, and colleagues at Careggi University Hospital, Florence, Italy, in the MS Journal.

About Multiple Sclerosis

Multiple sclerosis is an unpredictable, often disabling disease of the central nervous system. Symptoms range from numbness and tingling to blindness and paralysis, and there is currently no cure for MS. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are leading to better understanding and moving us closer to a world free of MS. An estimated 1 million people live with MS in the United States. Most people with MS are diagnosed between the ages of 20 and 50, and it affects women three times more than men.


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