To evaluate nusinersen's effect on respiratory function in SMA patients and provide a clinical basis for respiratory management.
Approach:
Study Design: Single-center prospective study collecting clinical data from SMA patients in a pediatric department.
Participants: 59 SMA patients (39 Type II; 20 Type III) were included, with 40 completing baseline respiratory tests.
Assessment: Respiratory function and active cough strength were evaluated at baseline and during follow-up after nusinersen treatment.
Key Findings:
67.5% of patients had abnormal respiratory function at baseline.
Type II patients had significantly lower VC %pred, FVC %pred, FEV1%pred, PEF %pred, FEF25%pred, and FEF50%pred compared to Type III patients (all P < 0.05).
After 6 months, absolute value of FVC improved significantly in Type III patients (P < 0.05).
By 18 months, significant improvements in absolute VC values in Type II patients, and absolute values and %pred of VC, FVC, FEF25, FEF50, and FEF75 in Type III patients were observed (all P < 0.05).
The proportion of patients with effective cough increased from 39.1% at baseline to 56.5% after treatment (P < 0.05).
Interpretation:
Nusinersen improved respiratory function and active cough strength in SMA patients, but did not fully reverse the progression of restrictive ventilatory dysfunction, particularly in Type II patients.
Limitations:
The study was conducted at a single center, which may limit generalizability.
Only partial patients completed serial pulmonary function tests during follow-up.
Conclusion:
Most SMA patients had impaired respiratory function, with improvements noted after nusinersen treatment, particularly in Type III patients.
