诺西那生对高原地区脊髓性肌萎缩症患儿肺功能的影响：一项初步研究。
Effect of nusinersen on pulmonary function in children with spinal muscular atrophy in the plateau region: A pilot study.
作者信息
Zhu Jicai, Chen Xiaofang, Sang Haoke, Ma Minming, Tang Chunhui
机构信息
Department of Pediatrics, The First People's Hospital of Yunnan Province, Medical School & Affiliated Hospital, Kunming University of Science and Technology, Kunming, Yunnan, China.
出版信息
Heliyon. 2024 Dec 19;11(1):e41388. doi: 10.1016/j.heliyon.2024.e41388. eCollection 2025 Jan 15.
BACKGROUND
The drug nusinersen is applied to improve motor function in patients with spinal muscle atrophy (SMA). However, research on the effects of this treatment on lung function is lacking.
AIM
To investigate the effect of nusinersen on lung function in children with SMA in the Plateau.
METHODS
A total of 20 patients with SMA (types 1, 2, or 3) who started nusinersen treatment at the Department of Pediatrics at Yunnan First People's Hospital from March 2022 and February 2024 were studied. A retrospective study was conducted to investigate changes in lung function parameters (including forced vital capacity, forced expiratory volume at 1 s, forced expiratory volume at 1 s/forced vital capacity, and peak expiratory flow) in patients with SMA treated with nusinersen.
RESULTS
20 patients (13 male, 7 female; aged 5-16 years) were enrolled, including 2, 9, and 9 with SMA types 1, 2, and 3, respectively. The mean value of FVC % and FEV1/FVC % did not decrease further following nusinersen treatment in any patients. The mean value of FEV1% was 4.4 % and 5.0 % higher than before treatment in all patients (P = 0.03), and those with type 2 SMA (P = 0.04), respectively. The overall mean PEF % did not decrease any further after treatment. However, the average level in the type 2 group increased by 2.9 % (P = 0.03).
CONCLUSION
Patients with SMA, particularly those classified as type 2, showed a trend of improvement in lung function following nusinersen treatment.
背景
药物诺西那生用于改善脊髓性肌萎缩症（SMA）患者的运动功能。然而，关于这种治疗对肺功能影响的研究尚缺。
目的
探讨诺西那生对高原地区SMA患儿肺功能的影响。
方法
对2022年3月至2024年2月在云南省第一人民医院儿科开始接受诺西那生治疗的20例SMA患者（1型、2型或3型）进行研究。采用回顾性研究，调查接受诺西那生治疗的SMA患者肺功能参数（包括用力肺活量、第1秒用力呼气量、第1秒用力呼气量/用力肺活量和呼气峰值流速）的变化。
结果
纳入20例患者（男13例，女7例；年龄5至16岁），其中1型、2型和3型SMA患者分别为2例、9例和9例。接受诺西那生治疗后，所有患者的用力肺活量百分比（FVC%）和第1秒用力呼气量/用力肺活量百分比（FEV1/FVC%）均值均未进一步下降。所有患者的第1秒用力呼气量百分比（FEV1%）均值比治疗前分别高4.4%和5.0%（P = 0.03），2型SMA患者中FEV1%均值比治疗前高5.0%（P = 0.04）。治疗后总体呼气峰值流速百分比（PEF%）均值未进一步下降。然而，2型组的平均水平提高了2.9%（P = 0.03）。
结论
SMA患者，尤其是2型患者，接受诺西那生治疗后肺功能有改善趋势。
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