ArticleThe journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG2024
New Tic Disorder in a Child With Cystic Fibrosis Treated With Elexacaftor/Tezacaftor/Ivacaftor.
Article in The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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Who cites it
2 citing papers in PubMed, 1 citations in OpenAlex.
- Mental health, sleep, and quality of life following CFTR modulator therapy: a longitudinal study of children with cystic fibrosis and caregivers.European journal of pediatrics · 2026Observational
- Early effect of CFTR modulators on the mental health of patients with cystic fibrosis and parents.European journal of pediatrics · 2026Observational
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Authors and funding
3 authors at 1 institution in 1 country.
Funding
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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.
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Registered trials
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