Patients included in the analyses had a mean age of 35 years, and the majority had been diagnosed with a relapsing form of MS, such as relapsing-remitting MS (RRMS) or active secondary progressive MS, according to a press release from the National Multiple Sclerosis Society. Most of them had also received BEAM+ATG, an intense chemotherapy combination as part of the immunoablation regimen that preceded the transplant itself.

At the study’s start, participants had a mean expanded disability status scale (EDSS) score of 6, corresponding to a moderate-to-high level of disability, in which patients need the help of a cane or crutch to move around.

When re-evaluated a decade after receiving the transplant, more than half (65.5%) of the patients continued showing no signs of disability worsening. These values were even higher (71.3%) in the subset of 122 RRMS patients included in the study.

The levels of disability in this subgroup of patients started to decrease since the transplant, with EDSS scores dropping a mean of 0.09 units per year.

Before 2007, three patients died within a period of 100 days following AHSCT, but after 2007, no patient died within 100 days of having the procedure.

Statistical analyses also found that patients who had immunoablation with BEAM+ATG and those who experienced a higher number of relapses before receiving the transplant, were more likely to show no signs of disability progression 10 years after the procedure.

“The BEAM+ATG conditioning protocol is associated with a more pronounced suppression of clinical relapses and MRI inflammatory activity,” the investigators wrote.

Taken together, the data provide “evidence that for people with MS, AHSCT induces durable disease remission,” the team wrote. Specifically, “aHSCT prevents disability worsening in the majority of patients and induces durable improvement in disability in patients with RRMS.”

These findings are in line with those of previous studies suggesting that a stem cell transplant may be particularly effective for specific groups of patients with active forms of MS who have failed to respond adequately to DMTs.

Based on data from these studies, the National MS Society recently released a set of recommendations on who would be the best candidates for this procedure. According to the experts, patients with relapsing forms of the disease, who are younger than 50, and who continue showing signs of disease activity despite treatment with high-efficacy DMTs, are likely to benefit most from a stem cell transplant.

A large Phase 3 trial, called BEAT-MS (NCT04047628), is ongoing to explore the therapeutic potential of AHSCT for those with MS. The trial, still recruiting participants, seeks to compare the safety and efficacy of a transplant with the most effective DMT in 156 adults with relapsing forms of MS.

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.

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About the Author:
Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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