Efficacy and safety of salbutamol in treatment of children with later-onset spinal muscular atrophy / 浙江大学学报·医学版

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 (Z=－4.144, P<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 (Z=－2.214, P<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 (Z=－2.366, P<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 (FEV1), and peak expiratory flow (PEF) in 15 cases after treatment (all P<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.

Surveillance of schistosomiasis in People's Republic of China in 2015 / 中国血吸虫病防治杂志

Objective To analyze the investigation data of the national schistosomiasis surveillance sites in 2015,so as to provide scientific evidences for schistosomiasis control,elimination and surveillance. Methods According to National Schisto-somiasis Surveillance Programme(version 2014),457 surveillance sites were selected,and the investigation data in residents, floating population,domestic animals and Oncomelania hupensis snails were collected and analyzed from four types of endemic counties. Results A total of 4468 seropositive cases were detected from 133350 residents,among which 4457 residents with seropositive results received the etiological tests,and 71 of them were identified with positive results. Most of them were fisher-men and farmers in the middle and old-aged group. The schistosomiasis infection rate was 0.05% in local population. Totally 977 seropositive cases were examined from 85047 migrant individuals,and 16 positive cases were found out from 966 individuals who took etiological tests,which showed the schistosomiasis infection rate was 0.02% in floating population. Imported cases were found among floating people in four provinces,namely Zhejiang,Hunan,Hubei and Anhui provinces. No acute schistoso-miasis cases were reported. A total of 13406 head of cattle received examinations and only 5 were determined as stool positives. The cattle infection rate was 0.04%. The snail survey covered an area of 22295.13 hm2 and snails were found in an area of 7426.63 hm2,including 3.47 hm2 newly detected area with snails. No schistosome-infected snails were found. Conclusions Based on the collected data from 457 national schistosomiasis surveillance sites of China,the Schistosoma japonicum infection rate is 0.05% in local population which maintains a stably descending trend. In floating population,there are imported schistosome-in-fected persons. Cattle are still a vulnerable species infected with schistosome. Although no infected snails are found,snails are widely distributed in endemic areas. Some provinces detect areas with snails for the first time or the reproduction of snails. The staff in endemic provinces should carry out the surveillance work according to National Schistosomiasis Surveillance Programme (version2014)to improve the surveillance system,and enhance the sensibility and effectiveness of surveillance work.