Stem cell transplants may delay disability longer than some MS drugs

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In people with active secondary progressive multiple sclerosis (MS), hematopoietic stem cell transplants may delay disability longer than other MS drugs, according to a study published online December 21, 2022, in Neurology®, the medical journal of the american academy of neurology. The study involved autologous hematopoietic stem cell transplants, which use healthy blood stem cells from a person’s own body to replace diseased cells.

Although most people with MS are first diagnosed with relapsing-remitting MS, which is characterized by symptom flares followed by periods of remission, many people with relapsing-remitting MS eventually go on to secondary progressive MS, which You don’t have big changes in symptoms, just a slow, steady worsening of the disease.

“Hematopoietic stem cell transplants have previously been found to delay disability in people with relapsing-remitting MS, but less is known about whether such transplants could help delay disability during the most advanced stage of the disease,” said Dr. study author Matilde Inglese, MD, PhD, of the University of Genoa in Italy and a member of the American Academy of Neurology. “Our results are encouraging, because while current treatments for secondary progressive MS have modest or small benefits, our study found that stem cell transplants can not only delay disability longer than many other MS drugs, but which may also provide a slight improvement in symptoms.”

The retrospective study included 79 people with active secondary progressive MS who received stem cell transplants and 1,975 people from the Italian MS registry who were treated with MS drugs. All received treatment after being diagnosed with active secondary progressive MS. The two groups were matched for age, sex, and level of disability. Drugs included beta interferons, azathioprine, glatiramer acetate, mitoxantrone, fingolimod, natalizumab, methotrexate, teriflunomide, cyclophosphamide, dimethyl fumarate, and alemtuzumab.

The level of disability of the participants was measured on the Expanded Disability Status Scale, a common method of quantifying disability with scores ranging from 0, no symptoms, to 10 points, death due to MS. Participants were evaluated at various times over 10 years.

At the start of the study, participants had a median score of 6.5 for both those who received transplants and those who received the drugs. Scores of 6.0 are defined as the need to use a cane or brace intermittently or on one side to walk about 100 meters with or without rest. Scores of 6.5 are defined as the need to constantly use a cane or brace on both sides to walk about 20 meters without resting.

Five years into the study, the researchers found that 62% of people who underwent stem cell transplants did not experience a worsening of their MS disability compared to 46% of those who took medication.

Additionally, at five years, the researchers found that people who received stem cell transplants were more likely to see sustained improvements over time, with 19% experiencing less disability than at the start of the study, compared to just 4%. of people taking medication.

Over 10 years, the disability score of people who received stem cell transplants decreased by an average of 0.01 points per year, which means less disability, while the average score of people taking medications increased by 0, 16 points per year, an increase in disability.

“Our study shows that hematopoietic stem cell transplants were associated with a slowing of disability progression and a greater likelihood of disability improvement compared with other therapies,” Inglese said. “While these results are encouraging, they are not applicable to patients with secondary progressive MS who do not have signs of inflammatory disease activity; further research in larger groups of people is needed to confirm our findings.”

One limitation of the study is that it was retrospective and observational, and did not prove cause and effect. It only suggests an association. The study also did not include people taking the MS drugs siponimod, cladribine, ocrelizumab, ofatumumab, or rituximab.

The study was funded by the Italian Multiple Sclerosis Foundation.

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