中国药业2026，Vol.35Issue(11)：119-129,11.DOI:10.3969/j.issn.1006-4931.2026.11.023
基于FAERS挖掘曲非奈肽用于18岁以下雷特综合征患者的药品不良事件信号
Signal Mining of Adverse Drug Events Induced by Trofinetide in the Treatment of Patients with Rett Syndrome Under 18 Years Old Based on the FAERS
摘要
Abstract
Objective To mine the drug adverse event(ADE)signals of trofinetide in the treatment of patients with Rett syndrome(RTT)under 18 years old,and to provide a reference for the clinical rational and safe use of trofinetide.Methods ADE reports related to trofinetide in the treatment of patients with RTT under 18 years old from the first quarter of 2023 to the first quarter of 2025 in the US Food and Drug Administration Adverse Event Reporting System(FAERS)were retrieved,and they were classified and described according to the preferred term(PT)and system organ classification(SOC)in the Medical Dictionary for Regulatory Activities 27.0(MedDRA 27.0).The Reporting Odds Ratio(ROR)method,the Medicines and Healthcare Products Regulatory Agency(MHRA)method,the Bayesian Confidence Propagation Neural Network(BCPNN)method,and the Multi-Item Gamma Poisson Shrinker(MGPS)method were used for signal mining and analysis.The subgroup analysis was conducted based on gender,age,and dose of trofinetide.The combination medication,comorbidities,and timing of the ADE ccurrence were analyzed.Results A total of 849 ADE reports were screened with trofinetide as the primary suspected(PS)drug.Among them,the patients were mainly female(95.17%),the patients' ages were mostly concentrated in the range of 5-12 years old(50.77%),the ADEs were mainly reported in 2024(76.44%),all reports originated from the United States of America(USA,100.00%),the majority of reporters were consumers(97.17%),the primary clinical outcome of severe ADEs was hospitalization(21.91%).A total of 3 682 cases of ADEs were identified,involving 11 SOCs and 132 PTs.Among the ADE positive signals,the top three SOCs reported cases were gastrointestinal disorders(741 cases),various injury,poisoning and procedural complications(612 cases),and infectious and infestations(441 cases);the top three PTs in terms of signal intensity were faeces soft(ROR=248.91),developmental regression(ROR=245.88),and breath holding(ROR=174.97).Five new adverse drug reactions(ADRs)were identified,including drooling,pulmonary congestion,dyskinesia,infectious aspiration pneumonia,and insomnia.The subgroup analysis results showed that diarrhea was most frequently reported at the age of 5-12,the types of ADEs in female patients were more than that in male patients,and the incidence of ADEs in the mental system was higher in patients with high-dose trofinetide(60 mL).Trofinetide was often used in combination with antiepileptic,anxiolytic,and analgesic-antipyretic drugs.Patients treated with trofinetide frequently have comorbid conditions involving various neurological,gastrointestinal,and psychiatric disorders.The median onset time of trimethoprim-related ADE was 6 d,and most of them occurred within 30 d after drug administration.Conclusion Close monitoring is required when using trofinetide to treat patients with RTT under 18 years old in clinical practice,particularly during the initial phase of treatment and when high doses are administered.While monitoring for known ADRs,close attention should be paid to new suspected signals,and appropriate interventions should be implemented to ensure the patients' medication safety.关键词
曲非奈肽/雷特综合征/药品不良事件/美国食品和药物管理局不良事件报告系统/儿童/安全性Key words
trofinetide/Rett Syndrome/adverse drug event/FAERS/children/medication safety分类
医药卫生引用本文复制引用
林晓芳,张琳,冉婕..基于FAERS挖掘曲非奈肽用于18岁以下雷特综合征患者的药品不良事件信号[J].中国药业,2026,35(11):119-129,11.基金项目
重庆市临床药学重点专科建设项目[渝卫办发[2021]52号]. （）
