Effect of nusinersen on pulmonary function in children with spinal muscular atrophy in the plateau region: A pilot study
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. Method...
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Elsevier
2025-01-01
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| author | Jicai Zhu Xiaofang Chen Haoke Sang Minming Ma Chunhui Tang |
| author_facet | Jicai Zhu Xiaofang Chen Haoke Sang Minming Ma Chunhui Tang |
| author_sort | Jicai Zhu |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-c5be4902e07146d79b0fb46eabf7a257 |
| institution | Kabale University |
| issn | 2405-8440 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | Elsevier |
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| series | Heliyon |
| spelling | doaj-art-c5be4902e07146d79b0fb46eabf7a2572025-01-17T04:51:07ZengElsevierHeliyon2405-84402025-01-01111e41388Effect of nusinersen on pulmonary function in children with spinal muscular atrophy in the plateau region: A pilot studyJicai Zhu0Xiaofang Chen1Haoke Sang2Minming Ma3Chunhui Tang4Department of Pediatrics, The First People's Hospital of Yunnan Province, Medical School & Affiliated Hospital, Kunming University of Science and Technology, Kunming, Yunnan, ChinaDepartment of Pediatrics, The First People's Hospital of Yunnan Province, Medical School & Affiliated Hospital, Kunming University of Science and Technology, Kunming, Yunnan, ChinaDepartment of Pediatrics, The First People's Hospital of Yunnan Province, Medical School & Affiliated Hospital, Kunming University of Science and Technology, Kunming, Yunnan, ChinaDepartment of Pediatrics, The First People's Hospital of Yunnan Province, Medical School & Affiliated Hospital, Kunming University of Science and Technology, Kunming, Yunnan, ChinaCorresponding author. Department of Pediatrics, The First People's Hospital of Yunnan Province, Kunming, 650021, Yunnan, China.; Department of Pediatrics, The First People's Hospital of Yunnan Province, Medical School & Affiliated Hospital, Kunming University of Science and Technology, Kunming, Yunnan, ChinaBackground: 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.http://www.sciencedirect.com/science/article/pii/S2405844024174191Spinal muscular atrophyPulmonary functionNusinersen |
| spellingShingle | Jicai Zhu Xiaofang Chen Haoke Sang Minming Ma Chunhui Tang Effect of nusinersen on pulmonary function in children with spinal muscular atrophy in the plateau region: A pilot study Heliyon Spinal muscular atrophy Pulmonary function Nusinersen |
| title | Effect of nusinersen on pulmonary function in children with spinal muscular atrophy in the plateau region: A pilot study |
| title_full | Effect of nusinersen on pulmonary function in children with spinal muscular atrophy in the plateau region: A pilot study |
| title_fullStr | Effect of nusinersen on pulmonary function in children with spinal muscular atrophy in the plateau region: A pilot study |
| title_full_unstemmed | Effect of nusinersen on pulmonary function in children with spinal muscular atrophy in the plateau region: A pilot study |
| title_short | Effect of nusinersen on pulmonary function in children with spinal muscular atrophy in the plateau region: A pilot study |
| title_sort | effect of nusinersen on pulmonary function in children with spinal muscular atrophy in the plateau region a pilot study |
| topic | Spinal muscular atrophy Pulmonary function Nusinersen |
| url | http://www.sciencedirect.com/science/article/pii/S2405844024174191 |
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