Impact of Nusinersen Treatment on the Independence and Mental Health of School-Aged Patients With Spinal Muscular Atrophy.

BACKGROUND

Emerging disease-modifying therapies for spinal muscular atrophy (SMA) have altered patients' mental health and daily functioning. This longitudinal study evaluated nusinersen's effects on independence and mental health in school-age SMA patients.

METHODS

Thirty-one SMA type 2/3 patients from a pediatric hospital were assessed using the SMA Independence Scale-Upper Limb Module (SMAIS-ULM), Screen for Child Anxiety-Related Emotional Disorders (SCARED), and Depression Self-Rating Scale for Children (DSRSC). Motor function was measured via Hammersmith Functional Motor Scale-Expanded (HFMSE) and Revised Upper Limb Module (RULM), with correlations analyzed.

RESULTS

Median treatment initiation age was 9.19 (8.73-11.90) years. After 14 months, SMAIS-ULM scores improved significantly (P < 0.01), while anxiety/depression rates declined (P < 0.01). Higher HFMSE scores correlated with better independence (P < 0.01) and reduced anxiety/depression (P < 0.01). Similarly, higher RULM scores predicted greater independence (P < 0.01) and lower anxiety (P < 0.05).

CONCLUSIONS

Nusinersen therapy enhances independence and mental health in school-age SMA patients, supported by patient-reported outcomes. These findings underscore its multifaceted therapeutic benefits.