Frontiers in pediatrics · 2026
Motor function and safety of nusinersen for spinal muscular atrophy: a systematic review and meta-analysis among Chinese patients.
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He Guanlan, Feng Zilu, Li Na, Wei Shasha, Liu Yunyuan, Tang Xiuneng
Аннотация
BACKGROUND: This study aimed to perform a systematic review and meta-analysis to evaluate motor function and safety outcomes in Chinese patients with spinal muscular atrophy (SMA) treated with nusinersen.
METHODS: We searched across multiple databases, including PubMed, Cochrane, Embase, Web of Science, CNKI, CBM, Wanfang, and VIP, for real-world studies (RWS) published before 15 February 2026. Data extraction is performed based on predefined study selection and eligibility criteria, while excluding studies involving non-Chinese populations.
RESULTS: 24 RWS were included in this analysis, comprising 1,273 Chinese patients with SMA Types 1-4 treated with nusinersen. Statistically significant improvements were found in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) [Mean Difference (MD) = 6.57 (95% CI: 4.35-8.80)], Hammersmith Infant Neurological Examination Section 2 (HINE-2) [MD = 2.05 (95% CI: 0.94-3.16)], Hammersmith Functional Motor Scale Expanded (HFMSE) [MD = 4.71 (95% CI: 3.10-6.32)], Revised Upper Limb Module (RULM) [MD = 3.70 (95% CI: 2.47-4.93)], and the 6-minute walk test (6MWT) [MD = 24.96 m (95% CI: 16.20-33.72)] at the end of the follow-up period. In terms of clinical response rate, CHOP-INTEND (≥4 points) was 71.80% (95%CI: 56.14%-85.65%), HFMSE (≥3 points) was 59.71%(95%CI: 43.79%-74.74%), RULM(≥2 points) was 60.35% (95%CI:48.67%-71.51%), respectively. As treatment duration increased, motor function scores and clinical response rates either improved or stabilized. When the treatment duration reached ≥14 months, the pooled mean change from baseline was as follows: CHOP-INTEND [MD = 9.45 (95% CI: 5.03-13.87)], HFMSE [MD = 4.37 (95% CI: 3.34-5.40)]], and RULM [MD = 2.83 (95% CI: 1.97-3.68)]. Clinical response rates were: 76.41% (95% CI: 52.89%-94.57%) for CHOP-INTEND, 69.83% (95% CI: 48.60%-87.61%) for HFMSE, and 63.23% (95% CI: 45.86%-79.13%) for RULM. The overall adverse event (AE) rate was 41.27% (95% CI: 29.65%-53.38%), with serious AEs occurring in 1.26% (95% CI: 0%-5.85%) of Chinese patients. The most common AEs were headache, back pain, and post-lumbar puncture syndrome, associated with the lumbar puncture administration method.
CONCLUSION: Our meta-analysis indicates that nusinersen is associated with a statistically significant and clinically meaningful improvement in motor function among Chinese patients with types 1-4 SMA in real-world studies. The reported adverse events align with the expected safety profile of nusinersen. However, the majority of studies included in this review had a follow-up period of less than 24 months. Therefore, further long-term follow-up is necessary to assess the sustained therapeutic effects of nusinersen in Chinese patients with SMA.
SYSTEMATIC REVIEW REGISTRATION: https://www.crd.york.ac.uk/PROSPERO/view/CRD42023403580, PROSPERO CRD42023403580.
METHODS: We searched across multiple databases, including PubMed, Cochrane, Embase, Web of Science, CNKI, CBM, Wanfang, and VIP, for real-world studies (RWS) published before 15 February 2026. Data extraction is performed based on predefined study selection and eligibility criteria, while excluding studies involving non-Chinese populations.
RESULTS: 24 RWS were included in this analysis, comprising 1,273 Chinese patients with SMA Types 1-4 treated with nusinersen. Statistically significant improvements were found in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) [Mean Difference (MD) = 6.57 (95% CI: 4.35-8.80)], Hammersmith Infant Neurological Examination Section 2 (HINE-2) [MD = 2.05 (95% CI: 0.94-3.16)], Hammersmith Functional Motor Scale Expanded (HFMSE) [MD = 4.71 (95% CI: 3.10-6.32)], Revised Upper Limb Module (RULM) [MD = 3.70 (95% CI: 2.47-4.93)], and the 6-minute walk test (6MWT) [MD = 24.96 m (95% CI: 16.20-33.72)] at the end of the follow-up period. In terms of clinical response rate, CHOP-INTEND (≥4 points) was 71.80% (95%CI: 56.14%-85.65%), HFMSE (≥3 points) was 59.71%(95%CI: 43.79%-74.74%), RULM(≥2 points) was 60.35% (95%CI:48.67%-71.51%), respectively. As treatment duration increased, motor function scores and clinical response rates either improved or stabilized. When the treatment duration reached ≥14 months, the pooled mean change from baseline was as follows: CHOP-INTEND [MD = 9.45 (95% CI: 5.03-13.87)], HFMSE [MD = 4.37 (95% CI: 3.34-5.40)]], and RULM [MD = 2.83 (95% CI: 1.97-3.68)]. Clinical response rates were: 76.41% (95% CI: 52.89%-94.57%) for CHOP-INTEND, 69.83% (95% CI: 48.60%-87.61%) for HFMSE, and 63.23% (95% CI: 45.86%-79.13%) for RULM. The overall adverse event (AE) rate was 41.27% (95% CI: 29.65%-53.38%), with serious AEs occurring in 1.26% (95% CI: 0%-5.85%) of Chinese patients. The most common AEs were headache, back pain, and post-lumbar puncture syndrome, associated with the lumbar puncture administration method.
CONCLUSION: Our meta-analysis indicates that nusinersen is associated with a statistically significant and clinically meaningful improvement in motor function among Chinese patients with types 1-4 SMA in real-world studies. The reported adverse events align with the expected safety profile of nusinersen. However, the majority of studies included in this review had a follow-up period of less than 24 months. Therefore, further long-term follow-up is necessary to assess the sustained therapeutic effects of nusinersen in Chinese patients with SMA.
SYSTEMATIC REVIEW REGISTRATION: https://www.crd.york.ac.uk/PROSPERO/view/CRD42023403580, PROSPERO CRD42023403580.