The Gap
Tysabri is a therapy that has shown efficacy for reducing disease activity in patients diagnosed with pediatric-onset multiple sclerosis (POMS). Previous investigations have indicated that up to 58% of trial participants achieved (NEDA)-3, or no evidence of disease activity. For a patient to receive (NEDA)-3 they must have no MRI activity, no worsening of disability, and no clinical relapses. While these results have been amazing for the MS community, the effect of Tysabri on cognition (otherwise known as NEDA-3 plus) has yet to be explicitly examined.
This investigation is important because the onset of cognitive disability often accompanies a POMS diagnosis. The effect of Tysabri on improved cognition has been indicated in the adult MS population, but its effect has not been studied in the pediatric patient.
A team of researchers decided to change that.
A New Study
The results from this investigation were published in the Journal of Neurology.
The Italian team of scientists enrolled 20 POMS patients who had were an average of 14 years old. This trial took place at a single center where patients received the therapy for two years. During this time, each patient received an average of 42 infusions.
Results
This study found a significantly reduced EDSS score after the two years of treatment which indicates a reduction in disability.
MRI disease activity was found in 2 patients. Additionally, 2 patients were found to have a “mild decline in cognition.” However, this decline did not get any worse within the next year. As POMS patients without treatment have a high risk of experiencing decline in adulthood, this finding is significant.
Additionally, none of the trial participants experienced a relapse, with 80% of patients maintaining NEDA-3 plus status.
What it Means
The results from this study are important because they confirm that Tysabri should not only be used for POMS patients, but should be used as the first choice of treatment.
Early and aggressive treatment is important.
These clinical findings in addition to the fact that no serious adverse events were documented show the benefits that this therapy could have for the pediatric population’s cognition, filling a gap in the knowledge we already had.
You can read more about these findings here.
