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Patient-Reported Outcomes Reinforce Efficacy of Natalizumab for MS


Researchers gathered data on patient-reported outcomes to support evidence for the continuation of natalizumab, a disease-modifying treatment, in the management of multiple sclerosis (MS) progression.

The use of natalizumab (Tysabri) in the treatment of multiple sclerosis (MS) exhibited both short- and long-term improvement in terms of patient-reported outcomes (PRO), according to a recent study published in the Journal of Neurological Science.

PROs are typically reported in the form of questionnaires and cover aspects about a specific disease itself or aspects of an individual’s quality of life. Although the provided information is not interpreted by a medical professional and comes directly from patients, these data have been increasingly included in clinical assessments and research.

For MS specifically, the LIFEware System, which largely covers daily functioning and physical or psychosocial limitations (where higher scores indicate better outcomes), has been recognized as a valuable tool in patient assessments. As the authors of the present study note, PRO data reported here, such as early measures of fatigue or lower-limb components, have been linked to degrees of MS progression and disability.

Patient Questionnaire Concept | image credit: jannoon028 - stock.adobe.com

Patient Questionnaire Concept | image credit: jannoon028 - stock.adobe.com

To expand on the efficacy of PRO in clinical data, researchers conducted a study to investigate the effects of disease-modifying treatments, specifically the continuation or discontinuation of natalizumab for MS, on PRO measures. They hypothesized that natalizumab discontinuation would be associated with worse PROs in patients and that natalizumab would contribute to substantial PRO improvements in the initial week of intervention. Patient disability was measured according to the Expanded Disability Status Scale (EDSS) and PROs were assessed with the LIFEware questionnaire.

Data from a total of 242 patients with MS treated with natalizumab, gathered from the New York State MS Consortium, were included in the analysis. Participants began natalizumab at approximately 43 years of age on average, with an average symptom onset age of 29.6 years, and average disability score of 3.5 EDSS. Of this group, 141 patients discontinued natalizumab, whereas the remaining 101 continued with the drug.

In the discontinuation group, researchers observed their EDSS scores change from 3.3 prior to treatment to 3.6 at the start of treatment and 4.1 following treatment discontinuation (P < .001). The continuation group also trended in this direction, however, at a reduced rate—going from 2.9 prior to initiation to 3.3 at the start and 3.5 at the time of their most recent follow-up (P = .002).

Over the course of the study period, more PRO domains worsened in the discontinuation group compared with patients who continued treatment (10 vs 2 PRO domains). Among these, in the discontinuation group, significant declines were reported in PROs encompassing bladder and bowel control (P < .001; P = .049), limitations in lower extremities (“getting up from low seat”: P = .009; “standing for more than 30 minutes”: P < .001; “climbing stairs”: P = .026), and feelings of lonesomeness (P = .034). Furthermore, the authors noted that a decision to continue natalizumab treatment was associated with better reports regarding upper extremity limitation (P = .023) and feelings of panic (P = .04).

Additionally, 67 patients from the cohort were prospectively analyzed in the first 7 days following natalizumab initiation. Among this group, significant improvements were observed in PROs regarding feelings of lonesomeness (P = .009), fatigue (P = .019), and bladder limitations (P = .012).

Overall, continuation of natalizumab was associated with long-term stabilizations or improvements in PROs compared with the significant reductions that were reported from those who discontinued this treatment. Furthermore, the researchers’ findings provide evidence for the short-term benefits of natalizumab treatment in regard to patient fatigue, bladder issues, and lonesome feelings. As data gathered on PROs continues to expand across clinical assessments, the authors conclude with confidence that natalizumab has a positive effect on the subjective well-being of patients with MS.


Jakimovski D, Kavak KS, Zakalik K, et al. Patient-reported outcomes based on discontinuation or continuous treatment with natalizumab: New York State Multiple Sclerosis Consortium (NYSMSC) study. J Neurol Sci. 2023 Nov 14;455:122781. doi: 10.1016/j.jns.2023.122781

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