| 吴计划,赵忠礼,胡学会,等.真实世界研究:诺西那生钠治疗 61例脊髓性肌萎缩症儿童的有效性与安全性[J].安徽医药,2025,29(8):1686-1691. |
| 真实世界研究:诺西那生钠治疗 61例脊髓性肌萎缩症儿童的有效性与安全性 |
| Real-world study: efficacy and safety of nusinersen in 61 children with spinal muscular atrophy |
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| DOI：10.3969/j.issn.1009-6469.2025.08.042 |
| 中文关键词: 脊髓性肌萎缩症 诺西那生钠 儿童 运动活动 真实世界研究 |
| 英文关键词: Spinal muscular atrophy Nusinersen Child Motor activity Real-world study |
| 基金项目:安徽省医疗卫生重点学科项目(皖卫函〔 2021〕273号) |
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| 中文摘要: |
| 目的研究诺西那生钠在脊髓性肌萎缩症( SMA)儿童真实世界的有效性和安全性。方法回顾性和前瞻性收集 2021年 5月至 2024年 3月安徽省儿童医院使用诺西那生钠治疗的 5q SMA病儿数据。在基线和治疗后 63 d、6个月、 14个月，通过费城儿童医院神经肌肉评估量表( CHOP INTEND)、汉默史密斯运动功能扩展量表(HFMSE)、修订上肢模块( RULM)评分、 6分钟步行试验( 6MWT)记录进行有效性分析，并分析治疗反应的相关影响因素，记录不良反应。结果共纳入 61例 SMA病儿， Ⅰ、 Ⅱ、Ⅲ型分别为 13例( 21.3%)、 31例( 50.8%)、 17例( 27.9%)。与基线相比，接受诺西那生钠治疗 14个月后， Ⅰ型 SMA病儿 CHOP INTEND评分由 20.0(9.5，28.0)分增至 28.0(22.0，43.0)分( P=0.008)。 Ⅱ型 SMA病儿 RULM评分由 11.0(8.0，16.0)分增至 17.5(10.8，25.8)分( P=0.001)。 Ⅲ型病儿 HMFSE评分由 45.0(35.5，48.5)分增至 50.0(38.0，56.0)分(P=0.007)。在 Ⅰ、Ⅱ型病儿中开始治疗年龄与运动功能改善呈负相关。治疗过程中，未发现导致停药的不良事件。结论诺西那生钠可显著改善 5q型 SMA儿童的运动功能， Ⅰ、Ⅱ型 SMA病儿早期治疗获益更大。 |
| 英文摘要: |
| Objective To investigate the real-world efficacy and safety of nusinersen in children with spinal muscular atrophy (SMA). Methods Data were retrospectively and prospctively collected from children with 5q SMA treated with nusinersen between May 2021and March 2024 at Anhui Provincial Children's Hospital. Patients were evaluated with the Children's Hospital of Philadelphia InfantTest of Neuromuscular Disorders (CHOP-INTEND),the Hammersmith Functional Motor Scale Expanded (HFMSE), the Revised Upper Limb Module (RULM) and 6-minute walk test (6MWT) at baseline and at Day 63, Month 6, and Month 14 post-treatment. The analysis of factors related to treatment response was performed, and adverse reactions were reviewed.Results A total of 61 children with SMA were enrolled, including 13 (21.3%) with type Ⅰ, 31 (50.8%) with type Ⅱ, and 17 (27.9%) with type Ⅲ. Compared to baseline, after14 months of treatment with nusinersen, the CHOP INTEND score increased from 20.0 (9.5, 28.0) to 28.0 (22.0, 43.0) in type Ⅰ SMAchildren (P=0.008). The RULM score increased from 11.0 (8.0, 16.0) to 17.5 (10.8, 25.8) in type Ⅱ SMA children (P=0.001). HMFSE score increased from 45.0 (35.5, 48.5) to 50.0 (38.0, 56.0) in type Ⅲ SMA children (P=0.007). The age of treatment initiation was nega-tively correlated with motor function improvement in types Ⅰ and Ⅱ SMA children. No adverse events leading to discontinuation of thedrug were observed during treatment.Conclusion Nusinersen can significantly improve the motor function of children with type 5qSMA, with greater benefit from early treatment in type Ⅰ and Ⅱ SMA children. |
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