A new French retrospective study published in Revue Neurologique reveals the importance of using both subjective and objective methods to assess treatment efficacy in adults with spinal muscular atrophy (SMA) who use a wheelchair.

The research team evaluated the efficacy of nusinersen, an intrathecally administered drug, by means of the conventional 32-item Motor Function Measurement (MFM) and the more subjective Canadian Occupational Performance Measure (COPM) performance and satisfaction scores.

The 18 patients, who were treated at Raymond-Poincaré ́University Hospital in Garches, France from September 2018 to August 2020, were divided at baseline into low-MFM and high-MFM groups, based on whether their score was less than or equal or greater than the mean of the overall population. The patients received nusinersen injections over a 303-day period according to the manufacturer’s protocol.


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“Interestingly, no improvements or reductions occurred in the objective MFM scores in either patient group over the treatment time,” the researchers wrote. “However, significant improvements were found in the perceived satisfaction and perceived performance measures of the COPM, particularly in the high baseline MFM group.”

The team believes this result might indicate that patients with less severe disease can benefit the most from treatment with nusinersen. In fact, other published studies on nusinersen have shown significant improvements in motor function in those with less severe disease.

The results of this study also reinforce the importance of even small improvements to patients in terms of their perceived satisfaction and feelings of self-sufficiency, as assessed by the COPM. In addition, while objective improvements in MFM were not found, the finding of stability or a lack of decline in function is probably also positive, given that the motor function of patients with SMA typically worsens over time.

Studies such as this help clinicians assess the potential benefits of a treatment to their patients as well as identify criteria for treatment initiation or withdrawal.

Reference

Lefeuvre C, Brisset M, Sarlon M, et al. Nusinersen treatment in adults with severe spinal muscular atrophy: a real-life retrospective observational cohort study. Rev Neurol (Paris). Published online January 6, 2022. doi:10.1016/j.neurol.2021.10.010