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Clinical Trial
. 2020 Dec;55(12):3364-3370.
doi: 10.1002/ppul.25059. Epub 2020 Sep 19.

Patients with cystic fibrosis and advanced lung disease benefit from lumacaftor/ivacaftor treatment

Linnea Chika Kristensen Ejiofor  1 , Inger Hee Mabuza Mathiesen  1 , Søren Jensen-Fangel  2 , Hanne Vebert Olesen  3 , Marianne Skov  4 , Lue Katrine Drasbaek Philipsen  5 , Camilla Lundgren Pedersen  6 , Tacjana Pressler  5
Affiliations
Clinical Trial

Patients with cystic fibrosis and advanced lung disease benefit from lumacaftor/ivacaftor treatment

Linnea Chika Kristensen Ejiofor et al. Pediatr Pulmonol. 2020 Dec.

Abstract

Background: Several studies have assessed safety and efficacy outcomes for lumacaftor/ivacaftor therapy. We report on lumacaftor/ivacaftor's impact on lung function, physical performance, and health-related quality of life (HRQOL) in a subpopulation of Danish people with Cystic Fibrosis (CF; PWCF) with advanced pulmonary disease who would not fulfill inclusion criteria for these studies.

Methods: This follow-up study examined lumacaftor/ivacaftor's effect in a highly selected CF population. Inclusion criteria included low percent predicted forced expiratory volume in one second (ppFEV1 ), fast deteriorating ppFEV1 , low body mass index (BMI), and difficult-to-treat infections. Primary endpoints included change in ppFEV1 slope, cardiopulmonary exercise testing (CPET), and all domains of the Cystic Fibrosis Questionnaire-Revised (CFQ-R). Secondary outcomes included change in ppFEV1 , BMI Z-score, and sweat chloride concentration.

Results: A total of 21 patients homozygous for the F508del mutation and a median ppFEV1 of 38.7 were included. We found significant improvements in ppFEV1 (+4.2 p < .01, +5.8 p < .01, +4.8 p < .01 and +3.8 p = .03 ppFEV1 after 3, 6, 9, and 12 months of treatment compared to baseline), ppFEV1 slope (+6.84 ppFEV1 /year between the year before and the year after treatment initiation; p = .02), and saturation at CPET initiation (+1.4%, p < .02) and termination (+2.6%, p < .01) after 6 months of treatment. Finally, HRQOL improved significantly in all CFQ-R domains except Emotion and Treat.

Conclusions: Our findings suggest that lumacaftor/ivacaftor reduces lung function decline, improves lung function, physical performance, and HRQOL to a greater extent in PWCF with severe lung disease than previously recognized.

Keywords: CPET; HRQOL; cystic fibrosis; lumacaftor/ivacaftor; ppFEV1 slope.

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References

REFERENCES

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