| 摘要: |
| 目的:探讨脊髓性肌萎缩症(SMA)患儿应用诺西那生钠注射液的临床疗效及安全性。 方法:选取 2022 年 1-6 月于我院 接受诺西那生钠鞘内注射治疗的 SMA 患儿 26 例,于治疗当天(T0)、第 14 天(T1)、第 28 天(T2)及第 63 天(T3)评估并比较 Hammersmith 扩展功能运动量表(HFMSE)、上肢模块修订版(RULM)、6 分钟步行试验(6MWT)及不良反应发生情况。 结果:与 T0 比较,T3 时 HFMSE 评分升高[(20. 69± 14. 64 ) 分 vs. ( 23. 38± 15. 69) 分, P< 0. 01]。 与 T0 比较,T3 时 RULM 评分升高 [(18. 27±10. 15)分 vs. (19. 88±10. 21)分,P<0. 05]。 结论:SMA 患儿应用诺西那生钠注射液治疗后,HFMSE 和 RULM 评分显 著提高,近端肢体运动功能明显改善,且无明显不良反应发生 |
| 关键词: 脊髓性肌萎缩症 诺西那生钠 Hammersmith 扩展功能运动量表 上肢模块修订版 |
| DOI：10.13407/j.cnki.jpp.1672-108X.2023.11.013 |
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| 基金项目: |
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| Efficacy of Nusinersen Sodium Injection in the Treatment of Children with Spinal Muscular Atrophy |
| Wang Han, Zhang Jinhong, Tian Xiaoyu, Bai Yan, Yu Bo, Wang Xiuxia |
| (The Second Hospital of Hebei Medical University, Shijiazhuang 050000, China) |
| Abstract: |
| Objective: To probe into the clinical efficacy and safety of norcyclosporine sodium injection in the treatment of children with spinal muscular atrophy (SMA). Methods: From Jan. 2022 to Jun. 2022, 26 children with SMA who received intrathecal injection of nusinersen sodium in our hospital were enrolled. Hammersmith Functional Motor Scale Expanded ( HFMSE) scores, Revised Upper Limb Module (RULM), and 6-Minute Walk Test ( 6MWT) and adverse drug reactions were evaluated and compared on the day of treatment (T0), the 14th day of treatment ( T1), the 28th day of treatment ( T2), and the 63rd day of treatment ( T3). Results: Compared with T0, HFMSE scores at T3 was higher ((20. 69±14. 64) points vs. (23. 38±15. 69) points, P<0. 01). Compared with T0, RULM scores at T3 was higher ((18. 27±10. 15) points vs. (19. 88±10. 21) points, P<0. 05). Conclusion: The HFMSE and RULM scores and motor function of proximal limb are significantly improved after treatment with nocinazone sodium injection in children with SMA. |
| Key words: spinal muscular atrophy nusinersen sodium Hammersmith Functional Motor Scale Expanded Revised Upper Limb Module |
