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When triple therapy is not working: A reverse iceberg perspective

Published:March 05, 2023DOI:https://doi.org/10.1016/j.jcf.2023.02.012
      In most countries the elexacaftor / tezacaftor / ivacaftor compound (ETI) is approved for pwCF aged 6 and older and at least one F508del mutation. ETI is widely considered a life changer by most who use or prescribe it, to the point that there is now some discussion in the CF community about changes in the strategy of care and consideration on how the future framework of CF centers may be affected by long term ETI usage [

      Southern K.W., Castellani C., Lammertyn E., Smyth A., VanDevanter D., van Koningsbruggen-Rietschelet S., et al. Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis. Published online: 2022. DOI:10.1016/j.jcf.2022.10.002.

      ]. The achievements of ETI have indeed been so patent that CF drug development is now largely focused towards treatments for pwCF ineligible for CFTR modulator therapies [
      • De Boeck K.
      Cystic fibrosis in the year 2020: a disease with a new face.
      ].
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