诺西那生钠治疗脊髓性肌萎缩症儿童的临床分析

doi:10.7499/j.issn.1008-8830.2401082

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Abstract Objective To investigate the efficacy and safety of nusinersen sodium in the treatment of children with spinal muscular atrophy (SMA). Methods A retrospective analysis was conducted on the clinical data of 50 children with 5q SMA who received nusinersen sodium treatment and multidisciplinary treatment management in Shanxi Children's Hospital from February 2022 to February 2024. Results Compared with the baseline data, 67% (8/12), 74% (35/47), and 74% (35/47) of the SMA children had a clinically significant improvement in the scores of Philadelphia Infant Test of Neuromuscular Disorders, Hammersmith Functional Motor Scale Expanded, and Revised Upper Limb Module, respectively, and the distance of 6-minute walking test increased from 207.00 (179.00, 281.50) meters to 233.00 (205.25, 287.50) meters (P<0.05) after nusinersen sodium treatment. Of all 50 children with SMA, 24 (48%) showed good tolerability after administration, with no significant or persistent abnormalities observed in 2 034 laboratory test results, and furthermore, there were no serious or immunological adverse events related to the treatment. After treatment, there was a significant change in forced vital capacity as a percentage of the predicted value in 27 children with restrictive ventilatory dysfunction, as well as a significant change in the level of 25-(OH) vitamin D in 15 children with vitamin D deficiency (P<0.05). Conclusions For children with SMA, treatment with nusinersen sodium can continuously improve the response rates of motor function scales, with good tolerability and safety.

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	GUO Jin
	WU Yun-Hong
	ZHANG Lin-Xia
	JI Hui-Ru
	ZHOU Na
	HU Xiao-Yue

Key words： Spinal muscular atrophy Nusinersen sodium Efficacy Safety Child

Received: 16 January 2024

Cite this article:

GUO Jin,WU Yun-Hong,ZHANG Lin-Xia et al. Clinical efficacy of nusinersen sodium in the treatment of children with spinal muscular atrophy[J]. CJCP, 2024, 26(7): 743-749.

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http://www.zgddek.com/EN/10.7499/j.issn.1008-8830.2401082 OR http://www.zgddek.com/EN/Y2024/V26/I7/743

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