Treatment Helps Motor Function in Real-World Patients With Type 2, 3 SMA

A meta-analysis of data on the effect of nusinersen in real-world patients with later-onset spinal muscular atrophy (SMA) suggests the drug improves measures of motor function across a range of patients with type 2 and 3 SMA. Researchers published their findings in the Orphanet Journal of Rare Diseases.

“Although a direct comparison with studies reporting data from untreated patients cannot be made, the longitudinal changes in the treated cohorts (consistently positive) are divergent from those observed in the untreated cohorts (consistently negative),” researchers wrote.

The systematic review and meta-analysis included 19 studies reporting nusinersen’s motor function effects in patients with type 2 or 3 SMA. Thirteen studies focused on adults, four on children, and two included both adults and children. Twelve studies also provided data for untreated patients.

According to researchers, all studies reported improvement on at least one measure of motor function in patients treated with nusinersen at every time point up to 14 months. Patients not treated with nusinersen, however, showed negative changes in motor function.

Differences were apparent in both the global cohort of patients as well as in smaller groups categorized by age, SMA type, and functional status, the study found. Positive changes were most obvious in younger patients.

Although measures of fatigue and other aspects of daily living would have allowed for a more qualitative assessment of the effects of nusinersen, researchers pointed out, most studies did not include that information. Nevertheless, the real-world results confirm and expand previously reported positive findings from clinical trials.

“Patients, parents/caregivers, physicians, and payers for this expensive drug can benefit from the results of this review that, while confirming the importance of early treatment, suggests a positive response to nusinersen treatment across a broad spectrum of the SMA population,” researchers wrote.

Reference:
Coratti G, Cutrona C, Pera MC, et al. Motor function in type 2 and 3 SMA patients treated with nusinersen: a critical review and meta-analysis. Orphanet Journal of Rare Diseases. 2021;16(1):430. doi:10.1186/s13023-021-02065-z