Treatment with nusinersen (Spinraza®) improves motor function in adult and pediatric patients with type 2 and 3 spinal muscular atrophy (SMA), according to a new meta-analysis study published in the Orphanet Journal of Rare Diseases.
All of the studies reviewed reported an increase in Hammersmith Function Motor Scale Expanded (HFMSE) for treated patients, and the pooled mean change across all of the studies was 2.27 (95% CI, 1.41–3.13). This was in comparison to untreated patients which showed a negative trend (pooled mean change, −1.00; 95% CI, -1.33; -.67). The 2 datasets were statistically different from each other (P <.0001).
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Some studies reported Revised Upper Limb Module (RULM) scores for patients as well. All but 1 of the manuscripts reviewed showed an increase in RULM with nusinersen treatment, and pooled analysis yielded a pooled mean change of 1.11 (95% CI, .53-1.69). Untreated patients had a smaller change in RULM scores (pooled mean change, .47; 95% CI, -.79-1.74) although the difference between untreated and treated patients was not significant (P =.370).
Results of the 6-Minute Walk Test (6MWT) also showed significant differences (P <.0001) for treated vs untreated patients with pooled mean changes of 19.80 (95% CI, 6.70–32.89) and -8.29 (95% CI, −19.10-2.52), respectively.
“In conclusion our review highlights that improved motor function can be observed in all the type 2 and 3 cohorts of nusinersen treated patients, in contrast to the negative changes found in studies reporting untreated cohorts,” the authors of the review said.
“These real-world results obtained in clinical settings in different countries confirm and expand the positive results reported in more selected cohorts in the clinical trials,” the authors added.
The meta-analysis included 19 manuscripts with real-world results of nusinersen use in pediatric and adult patients with type 2 or 3 SMA. Since no control or placebo groups were included in these studies, the meta-analysis compared results to 12 papers that reported functional assessment data in untreated patients.
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 J Rare Dis. 2021;16(1). doi:10.1186/s13023-021-02065-z