沙丁胺醇治疗晚发性脊髓性肌萎缩症患儿的疗效和安全性。
Efficacy and safety of salbutamol in treatment of children with later-onset spinal muscular atrophy.
机构信息
Department of Neurology, Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou 310052, China.
Department of Developmental Behavior, Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou 310052, China.
出版信息
Zhejiang Da Xue Xue Bao Yi Xue Ban. 2023 Dec 17;52(6):714-720. doi: 10.3724/zdxbyxb-2023-0463.
OBJECTIVES
To investigate the clinical efficacy and safety of salbutamol in the treatment of children with later-onset spinal muscular atrophy (SMA).
METHODS
This study is a prospective single-arm phase Ⅲ clinical study. Pediatric patients with SMA type Ⅱ and Ⅲ who visited Department of Neurology, Children's Hospital, Zhejiang University School of Medicine from December 2020 to June 2022 were enrolled. All patients were evaluated with motor function scales, pulmonary function test and drug safety before study. Patients were treated with salbutamol tablets orally, with an initial dose of 1 mg (tid). If tolerable, the dose was increased to 1.5 mg (tid) in the second week, then increased to 2 mg (tid) from the third week and maintained for 6 months. Patients were followed up at 1, 3 and 6 months of treatment.
RESULTS
Twenty-six patients were enrolled, including 10 boys and 16 girls. There were 16 cases of SMA type Ⅱ and 10 cases of type Ⅲ with age at treatment initiation of 5.67 (3.13, 7.02) years and disease duration of 2.54 (1.31, 4.71) years. The Hammersmith Functional Motor Scale-Expanded (HFMSE) scores were increased from 14.0 (6.5, 43.0) before treatment to 26.0 (15.0, 46.5) after treatment (=－4.144, <0.01) in 25 cases. The Revised Upper Limb Module Scale scores were increased from 33.0 (25.5, 36.0) before treatment to 35.0 (31.0, 36.5) after treatment (=－2.214, <0.05) in 9 cases. In 7 ambulant children with SMA type Ⅲ, the six minutes walking distance was increased by 30 (15, 52) m after a 6-month treatment (=－2.366, <0.05). Compared with the baseline pulmonary functions the patients showed a significant increase in forced vital capacity (FVC), forced expiratory volume in one second (FEV), and peak expiratory flow (PEF) in 15 cases after treatment (all <0.05). According to patients and caregivers subjective reporting, there were various degrees of improvement in coughing, sputum production ability and exercise endurance. No serious adverse events were observed during the study.
CONCLUSIONS
Short-term oral administration of salbutamol may improve motor and pulmonary functions in later-onset SMA children with good safety.
目的
探讨沙丁胺醇治疗晚发型脊髓性肌萎缩症（SMA）患儿的临床疗效和安全性。
方法
本研究为前瞻性单臂Ⅲ期临床研究。纳入 2020 年 12 月至 2022 年 6 月在浙江大学医学院附属儿童医院神经内科就诊的 SMA Ⅱ型和Ⅲ型晚发型患儿。所有患儿在研究前均进行运动功能量表、肺功能检查和药物安全性评估。患儿给予沙丁胺醇片口服，起始剂量为 1mg（tid）。若可耐受，第 2 周剂量增加至 1.5mg（tid），第 3 周增加至 2mg（tid），维持治疗 6 个月。患儿在治疗后 1、3、6 个月进行随访。
结果
共纳入 26 例患儿，其中男 10 例，女 16 例；SMA Ⅱ型 16 例，Ⅲ型 10 例，起始治疗年龄为 5.67（3.13，7.02）岁，病程为 2.54（1.31，4.71）年。25 例患儿治疗后 Hammersmith 功能性运动量表-扩展版（HFMSE）评分由治疗前的 14.0（6.5，43.0）分增加至 26.0（15.0，46.5）分（=－4.144，<0.01）。9 例 SMA Ⅲ型可步行患儿治疗后修订上肢运动功能测试量表评分由治疗前的 33.0（25.5，36.0）分增加至 35.0（31.0，36.5）分（=－2.214，<0.05）。7 例 SMA Ⅲ型可步行患儿治疗后 6 分钟步行距离增加 30（15，52）m（=－2.366，<0.05）。与基线肺功能比较，15 例患儿治疗后用力肺活量（FVC）、第 1 秒用力呼气容积（FEV）和呼气峰流速（PEF）均有显著改善（均<0.05）。根据患儿及其照料者的主观报告，咳嗽、咳痰能力和运动耐力均有不同程度的改善。研究期间未观察到严重不良事件。
结论
沙丁胺醇短期口服可能改善晚发型 SMA 患儿的运动和肺功能，且安全性良好。
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