Elexacaftor/tezacaftor/ivacaftor, sold under the brand names Trikafta (US) and Kaftrio (EU), is a fixed-dose combination medication used to treat cystic fibrosis. Elexacaftor/tezacaftor/ivacaftor is composed of a combination of ivacaftor, a chloride channel opener, and elexacaftor and tezacaftor, CFTR modulators.It is approved for use in the United States for people aged six years and older who have cystic fibrosis with a F508del mutation or other mutations in the CFTR gene. It is also approved for use in Canada, the European Union and Australia. Medical uses
The combination is indicated for the treatment of people aged six years and older who have cystic fibrosis with a F508del mutation or other mutations in the CFTR gene. Side effects
The most common side effects affecting more than 5% of patients are headache, upper respiratory tract infection, abdominal pain, diarrhea, rash, alanine aminotransferase increased, nasal congestion, blood creatine phosphokinase increased, aspartate aminotransferase increased, rhinorrhea, rhinitis, influenza, sinusitis and blood bilirubin increased. Interactions
Concomitant use with CYP3A inducers is not recommended. Dosage must be adjusted with moderate or strong CYP3A inhibitors.Other drugs with the potential for interaction include: warfarin, digoxin, statins, glyburide, nateglinide, repaglinide. Pharmacology
Cystic fibrosis and CFTR
Cystic fibrosis is an autosomal recessive genetic disorder of the CFTR protein which reduces chloride and sodium ion transport through the cell membrane, causing thicker than normal mucus secretions. The CFTR protein is found in epithelial cells of the lung, liver, pancreas, digestive tract, and reproductive tracts. CFTR has a role in the production of mucus, sweat, and digestive fluids.
This includes community activism, and multiple reports by international human rights and health institutions and national ethics bodies.In the cases where gonads may pose a cancer risk, as in some cases of androgen insensitivity syndrome, concern has been expressed that treatment rationales and decision-making regarding cancer risk may encapsulate decisions around a desire for surgical "normalization". Types
Feminizing and masculinizing surgeries: Surgical procedures depend on the diagnosis, and there is often a concern as to whether surgery should be performed at all. Typically, surgery is performed shortly after birth. Defenders of the practice argue that individuals must be clearly identified as male or female for them to function socially and develop "normally". Psychosocial reasons are often stated. This is criticised by many human rights institutions, and authors. Unlike other aesthetic surgical procedures performed on infants, such as corrective surgery for a cleft lip, genital surgery may lead to negative consequences for sexual functioning in later life, or feelings of freakishness and unacceptability. Hormone treatment: There is widespread evidence of prenatal testing and hormone treatment to prevent or eliminate intersex traits, associated also with the problematization of sexual orientation and gender non-conformity. Psychosocial support: All stakeholders support psychosocial support. A joint international statement by participants at the Third International Intersex Forum in 2013 sought, amongst other demands: "Recognition that medicalization and stigmatisation of intersex people result in significant trauma and mental health concerns.