Summary

This AI-generated content is provided without warranty, with no liability accepted for reliance on it. Learn more.

• Spinraza (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
• This summary is based on the review of 10 systematic review(s)/meta-analysis(es). ^[1-10]
• Motor Function Improvement: Nusinersen significantly improved motor function in multiple scales, including HFMSE (WMD: 4.90; 95% CI: 3.17, 6.63), RULM (WMD: 3.70; 95% CI: 3.30, 4.10), and HINE-2 (WMD: 5.21; 95% CI: 4.83, 5.60). Risdiplam showed lesser improvement in HFMSE (WMD: 0.87; 95% CI: 0.05, 1.68).
• Other Functional Scales: Nusinersen significantly improved motor milestone response (RD: 0.51; 95% CI: 0.39, 0.62) and HINE-2 score (RD: 0.26; 95% CI: 0.12, 0.40). Risdiplam showed improvement in the MFM32 scale (WMD: 1.48; 95% CI: 0.58, 2.38).
• Patient Survival and Respiratory Support: Nusinersen increased survival without permanent respiratory support in SMA type 1 and improved motor function across SMA types 1-3. Early treatment in presymptomatic children led to near-normal motor development.
• Comparison of Effectiveness with Other Drugs: Risdiplam may offer better survival and motor function in Type 1 SMA compared to nusinersen, though comparisons for types 2/3 were challenging due to differences in study populations. Other drugs (e.g., creatine, gabapentin) showed no clinically important effects on motor function in SMA types II and III.
• Overall Adverse Events (AEs): Nusinersen-related adverse events were reported in 0.57% (95% CI: 0%-3.97%) as definite and 7.76% (95% CI: 1.85%-17.22%) as probable. The overall adverse event rate was 83.51% (95% CI: 73.55%-93.46%), with serious adverse events occurring in 33.04% (95% CI: 18.15%-49.91%).
• Specific Adverse Events: The most common adverse events associated with nusinersen were fever (40.07%), upper respiratory tract infection (39.94%), and pneumonia (26.62%).
• Comparison with Placebo: There was a significant difference in overall adverse event rates favoring nusinersen (OR = 0.27, 95% CI: 0.08-0.95, p = 0.042), with a lower incidence of serious and fatal adverse events compared to placebo.
• Nusinersen shows significant motor function improvement across various age groups and SMA types, with better outcomes in younger children and those with a shorter disease duration. Adults older than 12 years with 5q-SMA also experience statistically significant improvements in HFMSE and RULM scores.