Clinical follow-up analysis of nusinersen in the disease-modifying treatment of pediatric spinal muscular atrophy
10.3760/cma.j.cn112140-20211223-01075
- VernacularTitle:诺西那生钠修正治疗儿童脊髓性肌萎缩症随访分析
- Author:
Shanshan MAO
1
;
Yijie FENG
;
Lu XU
;
Mei YAO
;
Yu XIA
;
Jianing JIN
;
Lingshuang WANG
;
Tingting CHEN
;
Xiaoyang CHEN
;
Yi ZHANG
;
Huan ZHANG
;
Yuan JIANG
;
Haibing LI
;
Qi LONG
;
Feng GAO
Author Information
1. 浙江大学医学院附属儿童医院神经内科 国家儿童健康与疾病临床医学研究中心，杭州 310052
- Keywords:
Spinal muscular atrophies of childhood;
Nusinersen;
Disease-modifying treatment;
Clinical effect
- From:
Chinese Journal of Pediatrics
2022;60(7):688-693
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To explore the clinical efficacy of disease-modifying drug nusinersen on children with spinal muscular atrophy.Methods:The baseline and longitudinal clinical data of 15 children who were treated with nusinersen in the Children′s Hospital, Zhejiang University School of Medicine from October 2019 to October 2021 were retrospectively collected. The general data (gender, age, genotype, and clinical classification, etc.), motor function, nutritional status, scoliosis and respiratory function were analyzed. Wilcoxon rank-sum test was used for comparing multi-system conditions before and after treatment.Results:The age of 15 cases (7 males, 8 females) was 6.8 (2.8, 8.3) years, with 2 cases of type 1, 6 cases of type 2, and 7 cases of type 3 respectively, and the course of disease was 55.0 (21.0, 69.0) months. After 9.0 (9.0, 24.0) months of treatment, the motor function scale evaluations of the Hammersmith neurological examination section 2 (13.0 (7.0, 23.0) vs. 18.0 (10.0, 25.0) scores, Z=-2.67, P=0.018) of 15 children, the Hammersmith functional motor scale expanded (38.0 (18.5, 45.5) vs. 42.0 (23.0, 51.0) scores, Z=-2.38, P=0.018), and the revised upper limb module (27.0 (19.5, 32.0) vs. 33.0 (22.5, 35.5) scores, Z=-2.52, P=0.012) of children with type 2 and 3 had significantly improved. Thirteen patients achieved clinically significant motor function improvement, and 2 of them had kept stable scale scores. Subjective reports also indicated that the muscle strength and daily exercise ability of these children improved after treatment, and no serious adverse reactions were reported. Supplemented by the multi-disciplinary team management, the levels of some indicators such as Cobbs angle of scoliosis and forced vital capacity all had significantly improved (all P<0.05). Conclusions:Nusinersen can improve the motor function of patients with 5q spinal muscular atrophy, which is also proved safe to be used in children. The drug treatment supplemented by the multi-disciplinary team management is helpful to improve the multi-system function of the children with spinal muscular atrophy.
