Tysabri Treatment Leads to Disease Activity-free Status in Patients with Pediatric-onset MS

Tysabri Treatment Leads to Disease Activity-free Status in Patients with Pediatric-onset MS
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Early treatment with Tysabri (natalizumab) of patients with aggressive pediatric-onset multiple sclerosis is highly effective at achieving disease activity-free status and preventing cognitive decline, a new study shows.

The study, “No evidence of disease activity including cognition (NEDA-3 plus) in naïve pediatric multiple sclerosis patients treated with natalizumab,” was published in the Journal of Neurology.

Patients with pediatric-onset multiple sclerosis (POMS) experience significant brain inflammation, and develop early-onset physical and cognitive disability. This has a negative impact on patients’ overall quality of life.

Several studies have shown that Tysabri, developed by Biogen, consistently reduces disease activity in POMS patients. Up to 58% of patients reach the status of no evidence of disease activity (NEDA)-3, which is defined as no clinical relapses, no increase in disability (as evaluated by the EDSS score), and no MRI activity.

Additionally, Tysabri has been shown to be effective in preventing cognitive decline in the adult population.

“However, no study has addressed the achievement of NEDA-3 including cognition (NEDA-3 plus) in naïve POMS treated with NTZ [natalizumab],” the researchers wrote.

So, to address this gap, a group of Italian researchers evaluated the safety and effectiveness of Tysabri in a group of 20 patients with POMS (average age of 13.8 years), who were followed at a single MS center.

Patients received Tysabri for at least 24 months (two years). The average number of infusions per patient during this period was 42.

Results indicated that after two years of Tysabri treatment, there was a significant reduction in the mean EDSS score in the whole group, meaning less disability.

During the follow-up, evidence of disease activity on MRI was observed in only two patients (10%). A mild decline in cognition was observed in two other patients, which did not worsen in the subsequent year. This is particularly important because POMS patients are at high risk of developing cognitive impairments in adulthood and don’t gain the normal age-related cognitive functions.

Additionally, no patient experienced clinical relapse, and at the time of the last visit the NEDA-3 plus status was maintained in 80% (16 patients) of the group analyzed.

In terms of safety, no major adverse side effects were observed. The treatment was well-tolerated and well-accepted.

Overall, the team concluded, “early treatment of aggressive POMS with NTZ [Tysabri] proved to be highly effective in achieving and maintaining the NEDA-3 plus status.”

The data “strongly support the use of NTZ as a first-choice treatment in this unique MS population,” they added.

Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queen’s University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimer’s disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.
Total Posts: 1,053
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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Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queen’s University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimer’s disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.
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