| アブストラクト | BACKGROUND: Spinal muscular atrophy (SMA) has become a manageable chronic condition with the advent of three major disease-modifying therapies. However, real-world safety data in children aged 0-3 years remain limited. This study aimed to characterize safety signals using long-term pharmacovigilance data. METHODS: Adverse drug event reports related to SMA therapies submitted to the FDA Adverse Event Reporting System were extracted from the first quarter of 2015 to the third quarter of 2025. Disproportionality analyses were conducted to detect signals. RESULTS: A total of 1,155; 408; and 957 Adverse drug event reports were identified for onasemnogene abeparvovec (OA), risdiplam, and nusinersen as the primary suspected drugs, respectively, with balanced sex distributions across the three therapies. Reports involving OA more frequently included abnormalities in liver function-related parameters. Signals related to respiratory failure, atelectasis, and pneumothorax were identified across all three therapies. Reports involving OA also showed a broader spectrum of renal and urinary preferred terms, including proteinuria, hematuria, and glycosuria. CONCLUSIONS: Distinct safety signal patterns were identified among the three SMA disease-modifying therapies in children 0-3 years. These findings provide postmarketing insights into their safety profiles. Further prospective studies are warranted to validate these findings. |
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| 組織名 | West China School of Pharmacy, Sichuan University, Chengdu, China; Department of;Pharmacy, West China Second Hospital, Sichuan University, Chengdu, China;;Evidence-Based Pharmacy Center, West China Second Hospital, Sichuan University,;Chengdu, China; Key Laboratory of Birth Defects and Related Diseases of Women and;Children (Sichuan University), Ministry of Education, Chengdu, China.;Department of Pharmacy, West China Second Hospital, Sichuan University, Chengdu,;China; Evidence-Based Pharmacy Center, West China Second Hospital, Sichuan;University, Chengdu, China; Key Laboratory of Birth Defects and Related Diseases;of Women and Children (Sichuan University), Ministry of Education, Chengdu,;China. Electronic address: chenl_hxey@scu.edu.cn. |
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