[Clinical efficacy of nusinersen sodium in the treatment of children with spinal muscular atrophy]. / 诺西é£çé æ²»çèé«æ§èè缩çå¿ç«¥ç临åºåæ.
Zhongguo Dang Dai Er Ke Za Zhi
; 26(7): 743-749, 2024 Jul 15.
Article
em Zh
| MEDLINE
| ID: mdl-39014952
ABSTRACT
OBJECTIVES:
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.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Oligonucleotídeos
Limite:
Child
/
Child, preschool
/
Female
/
Humans
/
Infant
/
Male
Idioma:
Zh
Ano de publicação:
2024
Tipo de documento:
Article