Impact of Elexacaftor-Tezacaftor-Ivacaftor in lung transplantation for Cystic Fibrosis in the United States

Tahuanty A. Pena; Brittany Wright; Kalpaj R. Parekh; Julia Kleney-Tait

doi:10.1016/j.jhlto.2024.100171

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Impact of Elexacaftor-Tezacaftor-Ivacaftor in lung transplantation for Cystic Fibrosis in the United States

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Impact of Elexacaftor-Tezacaftor-Ivacaftor in lung transplantation for Cystic Fibrosis in the United States

Tahuanty A. Pena, Brittany Wright, Kalpaj R. Parekh and Julia Kleney-Tait

JHLT Open, Vol.7, 100171

02/2025

DOI: 10.1016/j.jhlto.2024.100171

PMCID: PMC11935324

PMID: 40144821

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Abstract

Cystic Fibrosis (CF) is an autosomal recessive condition leading to progressive lung disease and often necessitating lung transplantation. Historically, CF has been one of the leading indications for lung transplants in the US. The advent of CFTR modulators, particularly ETI, has significantly improved clinical outcomes for pwCF, offering potential alterations in disease progression and transplantation needs. Data on lung transplants performed in the US since 1988 were retrieved from the Organ Procurement & Transplantation Network (OPTN). Custom reports were generated to compare the number of lung transplants and waitlist additions before and after ETI approval in 2019. The analysis focused on trends from 2009-2019 (pre-ETI) and 2021-2023 (post-ETI). The average annual lung transplants for CF decreased significantly from 243 (2009-2019) to 56.7 (2021-2023) post-ETI approval. Similarly, the average number of pwCF added to the lung transplant waitlist per year dropped from 295 to 55.6. Despite an overall increase in lung transplants and waitlist additions in the US, the proportion involving pwCF has markedly declined post-ETI. The introduction of ETI has dramatically reduced the need for lung transplants among pwCF, reflecting significant improvements in lung function and disease management. These findings underscore the transformative impact of CFTR modulators like ETI on the natural history of CF, highlighting the importance of continued advancements in precision medicine for genetic disorders. Future studies should investigate long-term outcomes and sustained trends in lung transplantation needs among pwCF.

Cystic Fibrosis

Elexacaftor

Ivacaftor

lung transplant

Tezacaftor

Details

Title: Subtitle: Impact of Elexacaftor-Tezacaftor-Ivacaftor in lung transplantation for Cystic Fibrosis in the United States
Creators: Tahuanty A. Pena - Department of Internal Medicine, Stead Family Department of Pediatrics, University of Iowa Carver College of Medicine, University of Iowa Healthcare
Brittany Wright - Department of Pharmacy, University of Iowa Healthcare
Kalpaj R. Parekh - University of Iowa
Julia Kleney-Tait - Department of Internal Medicine, University of Iowa Carver College of Medicine, University of Iowa Healthcare
Resource Type: Journal article
Publication Details: JHLT Open, Vol.7, 100171
DOI: 10.1016/j.jhlto.2024.100171
PMID: 40144821
PMCID: PMC11935324
NLM abbreviation: JHLT Open
ISSN: 2950-1334
Publisher: Elsevier Inc
Language: English
Electronic publication date: 10/2024
Date published: 02/2025
Academic Unit: Pulmonary, Critical Care, and Occupational Medicine; Anatomy and Cell Biology; Stead Family Department of Pediatrics; Cardiothoracic Surgery; Internal Medicine
Record Identifier: 9984740956302771

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