Multiple sclerosis, Stem transplants would delay disability.
According to the new study, stem cell transplantation would allow disability to be delayed longer than drugs.
According to the study funded by the Italian Multiple Sclerosis Foundation, published in Neurology®, the medical journal of the American Academy of Neurology, hematopoietic stem cell transplantation can delay disability longer than other drugs for multiple sclerosis (MS). The study involved autologous transplants (or self-transplants, in which the donor and recipient are the same people) of hematopoietic stem cells, which use healthy blood stem cells to replace diseased cells.
While most people with MS are first diagnosed with relapsing-remitting multiple sclerosis, characterized by symptom flare-ups followed by periods of remission, many people with relapsing-remitting MS (RRMS) eventually transition to MS secondarily. progressive (SP): it is the evolution of the relapsing-remitting form, many of the people initially diagnosed with the RR form will be able to pass to a secondary progressive form, characterized by a persistent disability that gradually progresses over time, which does not present large fluctuations in symptoms but a slow and steady worsening of the disease.
“Haematopoietic stem cell transplantation has been shown to delay disability in people with relapsing-remitting MS in the past , but it is not known whether such transplantation could help delay disability during the more advanced stage of the disease,” he said. study author Matilde Inglese, of the University of Genoa and member of the American Academy of Neurology. “Our results are encouraging, because while current treatments for secondary progressive MS have little or no benefit, our study found that stem cell transplantation not only can delay disability longer than many other MS drugs but may also provide a slight improvement in symptoms“.
The study included 79 people with secondary progressive MS who received a stem cell transplant and 1,975 people from the Italian MS Registry treated with MS drugs. All received treatment after their secondary MS diagnosis. The two groups were matched for age, gender, and level of disability. Drugs included beta-interferons, azathioprine, glatiramer acetate, mitoxantrone, fingolimod, natalizumab, methotrexate, teriflunomide, cyclophosphamide, dimethyl fumarate, and alemtuzumab .
Participants’ level of disability was measured with the Expanded Disability Status Scale , a common method for quantifying disability with scores ranging from 0 (no symptoms) to 10 points (death due to MS). Participants were evaluated at various time points over 10 years. At the start of the study, participants had a median score of 6.5 for both those receiving the transplant and those receiving the medications. The score of 6.0 is defined as “ the need to use a cane or brace intermittently – or on its side – to walk approximately 100 meters with or without rest ”. For scores of 6.5 we mean “la 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 experienced no worsening of their disability from MS, compared with 46% of those who took medication. Additionally, at five years, the researchers found that people who received the stem cell transplant were more likely to see lasting improvements over time, with 19% experiencing less disability than at the start of the study, compared with just 4% of people taking drugs.
Over 10 years, the disability score of people receiving stem cell transplants decreased by an average of 0.01 points per year, signifying less disability, while the average score of people taking medications increased by 0.16 points per year, signifying an increase in disability.
“Our study demonstrates that hematopoietic stem cell transplantation is associated with a slower progression of disability and a greater likelihood of disability improvement compared to other therapies,” said Matilde Inglese. “Although these results are encouraging, they are not applicable to patients with secondary progressive MS who have no signs of inflammatory disease activity; More research on larger groups of people is needed to confirm our findings.” A limitation of the study is that it is retrospective (a type of study in which the disease is already present and one proceeds backwards to understand what the possible determining factors were) and observational and does not prove cause and effect but suggests an association.