CFTR Modulators to the Rescue of Individuals with Cystic Fibrosis and Advanced Lung Disease

Myerburg, Mike M.; Pilewski, Joseph M.

doi:10.1164/rccm.202103-0674ed

Cited by 5 publications

(5 citation statements)

References 14 publications

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“…These observations also confirm previous radiological findings that showed areas with reduced mucus impaction following treatment and significantly augment research that identified the peripheral lung as an important site of therapy effect 8,9 .…”

Section: Discussionsupporting

confidence: 89%

Impact of elexacaftor/tezacaftor/ivacaftor combination therapy on body plethysmography in adults with cystic fibrosis: Beyond FEV<sub>1</sub>

Wollsching-Strobel,

Dieninghoff,

Mathes

et al. 2024

Pneumon

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INTRODUCTIONThe treatment approach involving a combination of three cystic fibrosis (CF) transmembrane conductance regulatory (CFTR) protein modulators (elexacaftor, tezacaftor, ivacaftor) in the treatment of CF patients has provided beneficial results on FEV 1 . METHODS The aim of the retrospective single-center observational study was to show the impact of elexacaftor/tezacaftor/ivacaftor with special emphasis on full body plethysmography, exercise capacity and quality of life. Adult cystic fibrosis patients were evaluated after the initiation of triple CFTR therapy between July 2020 and March 2021. A two-tailed t-test for dependent samples with Bonferroni correction was used to compare outcomes between the two visits. RESULTS A total of 38 adult CF-patients (20 females), aged 37.2 ± 11.6 years were included in the study. The analysis shows significant improvements in FEV 1 and vital capacity (VC). The Tiffeneau-Index (FEV 1 /VC) only showed a minor increase from 59 ± 14% to 62 ± 13%. Hyperinflation (RV) and effective specific airway resistance (sRAWeff) significantly improved such as exercise capacity (6MWD) and CFQ-R. sRAWeff showed a strong significant association with an improvement in exercise capacity (Pearson's r=0.593; 95% CI: 0.296-0.786, p<0.001) and FEV 1 (Pearson's r=0.560; 95% CI: 0.292-0.746, p<0.001) while all other parameters did not show significant correlations. CONCLUSIONS Triple CFTR modulator therapy improves not only FEV 1 but also airway resistance and pulmonary hyperinflation. This is most reliably assessed by measuring sRAWeff, which is associated with exercise capacity, representing the work of breathing. Futures studies should incorporate body plethysmography assessments especially in patients with limited ability to perform spirometry or with non-reproducible spirometry results.

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Section: Discussionsupporting

confidence: 89%

Impact of elexacaftor/tezacaftor/ivacaftor combination therapy on body plethysmography in adults with cystic fibrosis: Beyond FEV<sub>1</sub>

Wollsching-Strobel,

Dieninghoff,

Mathes

et al. 2024

Pneumon

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“…However, the study was performed at the time when the coronavirus disease (COVID-19) pandemic was surging in France and was having a profound effect on the ability to perform lung transplantation ( 7 – 9 ). Thus, it was suggested that at least some of the reduction in lung transplantation observed in our study could have been related to the COVID-19 pandemic ( 10 ). Indeed, data from the United Network for Organ Sharing registry also reported a decrease in lung transplant volume at the onset of the COVID-19 pandemic ( 11 ).…”

mentioning

confidence: 76%

Major Decrease in Lung Transplantation for Patients with Cystic Fibrosis in France

Martin

Legeai²,

Regard

et al. 2022

Am J Respir Crit Care Med

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“…29 As a result, we expect that there will be a continued decline or postponement in the need for LTX in pediatric and adult CF patients alike. 4 There have been anecdotal reports from several centers around the world of individuals with CF being able to postpone listing for LTX or even be removed from the transplant wait list, [30][31][32] which is corroborated by our experience at Canadian and Swiss pediatric LTX programs. Although confounded by the COVID-19 pandemic, one observational study noted that Trikafta® use may have led to approximately 50 fewer patients aged ≥12 years on the transplant path.…”

Section: Effect Of Cftr Modulators On Transplantationmentioning

confidence: 62%

The changing landscape of pediatric lung transplantation

Avdimiretz

Benden

2022

Clinical Transplantation

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There has been a shift over decades in the diagnostic indications for lung transplantation in children; in particular, there has been a reduction in the proportion of pediatric cystic fibrosis (CF) patients undergoing lung transplantation early in life, and more transplants occurring in other diagnostic groups. Here, we examine trends in pediatric lung transplantation with regards to indications by analyzing data from the United Network of Organ Sharing, the International Society for Heart and Lung Transplantation Thoracic Transplant Registry, and other sources. Over the past two years, there has been a precipitous decline in both the number of transplants due to CF and the proportion of CF cases relative to the total number of transplants, likely not solely due to the COVID-19 pandemic. In 2020, primary pulmonary arterial hypertension for the firsttime surpassed CF as main indication for pediatric lung transplantation in the United States, a finding that is also reflected in international data. We discuss the effect of novel CFTR modulator therapies as a major factor leading to this shifting landscape.Based on our trending, pulmonary hypertension-related diagnoses and pediatric interstitial lung diseases are rising indications, for which we suggest adjustments of consensus guidelines around candidate selection criteria.

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CFTR Modulators to the Rescue of Individuals with Cystic Fibrosis and Advanced Lung Disease

Cited by 5 publications

References 14 publications

Impact of elexacaftor/tezacaftor/ivacaftor combination therapy on body plethysmography in adults with cystic fibrosis: Beyond FEV<sub>1</sub>

Impact of elexacaftor/tezacaftor/ivacaftor combination therapy on body plethysmography in adults with cystic fibrosis: Beyond FEV<sub>1</sub>

Major Decrease in Lung Transplantation for Patients with Cystic Fibrosis in France

The changing landscape of pediatric lung transplantation

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