New Tic Disorder in a Child With Cystic Fibrosis Treated With Elexacaftor/Tezacaftor/Ivacaftor.

Q2 Medicine
Stephanie R Duehlmeyer, E Claire Elson, Christopher M Oermann
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引用次数: 0

Abstract

The widespread use of highly effective cystic fibrosis transmembrane-conductance regulator -modulator therapy has dramatically altered the lives of individuals with cystic fibrosis. Clinical trials leading to -modulator approval by the US Food and Drug Administration demonstrated improvements in major -outcome measures including pulmonary function, gastrointestinal symptoms, and quality of life. Subsequent clinical experience has confirmed significant improvement across these domains. Adverse effects reported -during clinical trials included headache and dizziness amongst others including upper respiratory infections, abdominal pain, diarrhea, rash, and elevated serum transaminases. Post marketing clinical experience has suggested that there may be additional central nervous system adverse effects resulting from modulator therapy. Reported events after initiation of cystic fibrosis transmembrane-conductance regulator modulator treatment include headaches and increased prevalence of mental health concerns including anxiety and depression. We report a new tic disorder in a 7-year-old girl with cystic fibrosis treated with elexacaftor/tezacaftor/ivacaftor.

一名接受 Elexacaftor/Tezacaftor/Ivacaftor 治疗的囊性纤维化患儿出现新的抽搐症。
高效囊性纤维化跨膜传导调节剂调节剂疗法的广泛应用极大地改变了囊性纤维化患者的生活。美国食品和药物管理局批准使用调节剂的临床试验显示,肺功能、胃肠道症状和生活质量等主要结果指标均有所改善。随后的临床经验证实,这些方面均有显著改善。临床试验期间报告的不良反应包括头痛和头晕,还包括上呼吸道感染、腹痛、腹泻、皮疹和血清转氨酶升高。上市后的临床经验表明,调节剂治疗可能会导致其他中枢神经系统不良反应。据报道,开始接受囊性纤维化跨膜电导调节剂调节剂治疗后发生的事件包括头痛以及包括焦虑和抑郁在内的精神健康问题发病率增加。我们报告了一名接受 elexacaftor/tezacaftor/ivacaftor 治疗的 7 岁囊性纤维化女孩新出现的抽搐障碍。
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来源期刊
Journal of Pediatric Pharmacology and Therapeutics
Journal of Pediatric Pharmacology and Therapeutics Medicine-Pediatrics, Perinatology and Child Health
CiteScore
2.40
自引率
0.00%
发文量
90
期刊介绍: The Journal of Pediatric Pharmacology and Therapeutics is the official journal of the Pediatric Pharmacy Advocacy Group. JPPT is a peer-reviewed multi disciplinary journal that is devoted to promoting the safe and effective use of medications in infants and children. To this end, the journal publishes practical information for all practitioners who provide care to pediatric patients. Each issue includes review articles, original clinical investigations, case reports, editorials, and other information relevant to pediatric medication therapy. The Journal focuses all work on issues related to the practice of pediatric pharmacology and therapeutics. The scope of content includes pharmacotherapy, extemporaneous compounding, dosing, methods of medication administration, medication error prevention, and legislative issues. The Journal will contain original research, review articles, short subjects, case reports, clinical investigations, editorials, and news from such organizations as the Pediatric Pharmacy Advocacy Group, the FDA, the American Academy of Pediatrics, the American Society of Health-System Pharmacists, and so on.
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