english.prescrire.org > Spotlight > Archives : 2022 > Ivacaftor + tezacaftor + elexacaftor (Kaftrio°) in cystic fibrosis with at least one F508del mutation: respiratory symptoms alleviated in the short term

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Ivacaftor + tezacaftor + elexacaftor (Kaftrio°) in cystic fibrosis with at least one F508del mutation: respiratory symptoms alleviated in the short term

FEATURED REVIEW Is this triple therapy more effective than dual therapy at reducing the incidence of pulmonary exacerbations and alleviating symptoms? Does it slow disease progression and extend survival? What adverse effects are associated with the addition of elexacaftor?
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  •   OFFERS AN ADVANTAGE  In patients with cystic fibrosis and at least one F508del mutation in the CFTR gene, triple therapy comprising ivacaftor + tezacaftor + elexacaftor alleviated respiratory symptoms in comparative trials, none of which had a duration of more than 24 weeks. Triple therapy with these drugs was more effective by this measure than the comparator, which was tailored to patients' CFTR mutations. A reduced incidence of pulmonary exacerbations was demonstrated, mainly in a trial in patients heterozygous for the F508del mutation and a mutation resulting in minimal CFTR protein function. The adverse effects of this combination mainly include upper respiratory tract infections, hepatic disorders, rash, muscle disorders. It is also involved in many drug interactions. Any longer-term adverse effects, or the possible effect on disease progression, are not yet known.
      

©Prescrire 1 March 2022

Source: "Ivacaftor + tezacaftor + elexacaftor (Kaftrio°) in cystic fibrosis with at least one F508del mutation. Respiratory symptoms alleviated in the short term" Prescrire International 2022; 31 (235): 61-64. Subscribers only.

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 OFFERS AN ADVANTAGE 
 
 
 
See also:

"Kaftrio° (ivacaftor +
tezacaftor + elexacaftor):
cited as Noteworthy at the
2021 Prescrire Drug Awards"
(March 2022)
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