Patients with spinal muscular atrophy (SMA) treated with nusinersen (Spinraza®) showed improvements during the second year of treatment, albeit smaller when compared to the first 12 months, according to results of a new study published in Annals of Clinical and Translational Neurology.

“Our study confirms that an improvement in nusinersen-treated patients can also be observed in the second year, as suggested by the follow-up of clinical trials or in smaller cohorts of treated patients,” the authors said.

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Data collected from Italian centers showed a significant increase in the Hammersmith Functional Motor Scale Expanded (HFMSE) score from baseline to 24 months in patients with genetically confirmed SMA type 2 (n=46) and type 3 (n=65). The increase from baseline to 12 months was only significant in patients with SMA type 2.

The Revised Upper Limb Module (RULM) score also improved after 12 and 24 months in patients with type 2, but not in patients with type 3.

Subgroup analyses revealed:

  • 24-month changes in HFMSE score in both patients with type 2 and type 3 <5 years of age
  • 24-month changes in HFMSE and RULM scores in sitters, but not nonsitters, in patients with type 2
  • 24-month changes in HFMSE score in ambulant patients, but not nonambulant patients, in patients with type 3
  • A strong correlation between disease duration and 24-month changes in HFMSE score in both patients with type 2 and type 3 aged <5 years
  • A moderate correlation between disease duration and 24-month changes in HFMSE score in patients with type 3 aged 8 to 14 years
  • A small correlation between disease duration and 24-month changes in HFMSE and RULM scores in patients with type 2 and type 3 in all age groups

Moreover, the authors observed 12- and 24-month changes in both HFMSE and RULM scores between nusinersen-treated and untreated patients with SMA type 2. Similar findings were observed for patients with SMA type 3 at 12 months, while only HFMSE showed significant differences at 24 months.

Reference

Pane M, Coratti G, Pera MC, et al. Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy. Ann Clin Transl Neurol. Published online February 15, 2022. doi:10.1002/acn3.51514