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Introduction <p>Spinal muscular atrophy (SMA) is a central nerve system disease characterized by a broad spectrum of clinical presentations, mainly manifested as muscle atrophy and weakness. The effectiveness and safety of nusinersen in Chinese adults with SMA have not been well reported. This analysis aimed to describe the effectiveness and safety of nusinersen among Chinese adults with 5q-SMA.</p> Methods <p>This analysis leveraged a longitudinal, multicenter disease registry including both prospective and retrospective data from adults with 5q-SMA in China. Nusinersen's effectiveness was assessed by the changes in the Hammersmith Functional Motor Scale&#xa0;Expanded (HFMSE), Revised Upper Limb Module (RULM), and 6-Minute Walk Test (6-MWT) from baseline and at 6, 10, and 14&#xa0;months among participants receiving nusinersen as their first disease-modifying therapy (DMT). Nusinersen's safety was evaluated among prospective participants who used nusinersen as their first DMT and initiated nusinersen on or after the enrollment date.</p> Results <p>A total of 171 and 19 participants were included in the effectiveness and safety analysis, respectively. For HFMSE, the mean [standard deviation (SD)] change from baseline at 14&#xa0;months after treatment initiation was 3.4 (5.34) among 17 sitters and 3.4 (4.03) among 25 walkers. For RULM, the mean (SD) change from baseline at 14&#xa0;months after treatment initiation was 2.6 (3.07) among 11 non-sitters, 1.6 (4.06) among 20 sitters, and 1.3 (1.84) among 13 walkers. For 6-MWT, the mean (SD) change from baseline at 14&#xa0;months after treatment initiation was 15.0 (35.80) m among 13 walkers. No death was reported in the registry. Three participants reported five mild adverse events, none of which were considered related to nusinersen by the investigators.</p> Conclusion <p>Despite the limited sample size, the analysis appears to show the benefit of nusinersen among Chinese adults of a wide range of ages and disease severity with 5q-SMA. The registry was registered on Clinicaltrials.gov (NCT05618379).</p>

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Effectiveness and Safety of Nusinersen Among Adults with 5q-Spinal Muscular Atrophy: A Multicenter Disease Registry in China

  • 期刊论文

  • 发表日期：

    2025年12月29日

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  • Yi Dai,
  • Xiaoli Yao,
  • Wenhua Zhu,
  • Yuying Zhao,
  • Huifang Shang,
  • Juanjuan Chen,
  • Qiang Meng,
  • Yaling Liu,
  • Wanjin Chen,
  • Liqiang Yu,
  • Lu Shen,
  • Daojun Hong,
  • Ken Chen,
  • Li Zhang,
  • Maerhaba Mai,
  • Liying Cui

摘要

Introduction

Spinal muscular atrophy (SMA) is a central nerve system disease characterized by a broad spectrum of clinical presentations, mainly manifested as muscle atrophy and weakness. The effectiveness and safety of nusinersen in Chinese adults with SMA have not been well reported. This analysis aimed to describe the effectiveness and safety of nusinersen among Chinese adults with 5q-SMA.

Methods

This analysis leveraged a longitudinal, multicenter disease registry including both prospective and retrospective data from adults with 5q-SMA in China. Nusinersen's effectiveness was assessed by the changes in the Hammersmith Functional Motor Scale Expanded (HFMSE), Revised Upper Limb Module (RULM), and 6-Minute Walk Test (6-MWT) from baseline and at 6, 10, and 14 months among participants receiving nusinersen as their first disease-modifying therapy (DMT). Nusinersen's safety was evaluated among prospective participants who used nusinersen as their first DMT and initiated nusinersen on or after the enrollment date.

Results

A total of 171 and 19 participants were included in the effectiveness and safety analysis, respectively. For HFMSE, the mean [standard deviation (SD)] change from baseline at 14 months after treatment initiation was 3.4 (5.34) among 17 sitters and 3.4 (4.03) among 25 walkers. For RULM, the mean (SD) change from baseline at 14 months after treatment initiation was 2.6 (3.07) among 11 non-sitters, 1.6 (4.06) among 20 sitters, and 1.3 (1.84) among 13 walkers. For 6-MWT, the mean (SD) change from baseline at 14 months after treatment initiation was 15.0 (35.80) m among 13 walkers. No death was reported in the registry. Three participants reported five mild adverse events, none of which were considered related to nusinersen by the investigators.

Conclusion

Despite the limited sample size, the analysis appears to show the benefit of nusinersen among Chinese adults of a wide range of ages and disease severity with 5q-SMA. The registry was registered on Clinicaltrials.gov (NCT05618379).