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.
The agency outlined early regulatory actions supporting nonanimal methods, including draft guidance, artificial intelligence tools, and expanded use of human-relevant data models.
Patients with preoperative vitamin D deficiency had higher postoperative pain scores and opioid use after mastectomy, including more than triple the odds of moderate to severe pain within 24 hours of surgery.
