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  • S93 Effect of lumacaftor/ivacaftor on total, bronchiectasis, and air trapping computed tomography (ct) scores in children homozygous for f508del-cftr: exploratory imaging substudy

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Cystic fibrosis: disease trajectory and evolving therapies
S93 Effect of lumacaftor/ivacaftor on total, bronchiectasis, and air trapping computed tomography (ct) scores in children homozygous for f508del-cftr: exploratory imaging substudy
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  1. AS Brody1,
  2. S Nagle2,
  3. C Hug3,
  4. G Marigowda3,
  5. D Waltz3,
  6. J Goldin4,
  7. F Ratjen5,
  8. L Wang3
  1. 1Cincinnati Children’s Hospital Medical Centre, Cincinnati, US
  2. 2University of Wisconsin, Madison, US
  3. 3Vertex Pharmaceuticals Incorporated, Boston, US
  4. 4David Geffen School of Medicine, Los Angeles, US
  5. 5University of Toronto, Toronto, Canada

Abstract

Objective To evaluate the effects of lumacaftor/ivacaftor (LUM/IVA) combination therapy on lung morphology and physiology with computed tomography (CT) scanning in patients aged 6 to 11 years with cystic fibrosis (CF) homozygous for the F508del-CFTR mutation.

Methods Baseline CT scans were obtained in 19 patients (12 active treatment, 7 placebo) from the phase 3 LUM/IVA trial (NCT02514473), and 24 week CT scans were completed in 7 active treatment and 3 placebo patients. CT scans were obtained at total lung capacity and at residual volume (RV). CT scans were scored by 2 independent readers blinded to all patient and time point information using the Brody score, which evaluates the extent and severity of multiple aspects of CF lung disease, including bronchiectasis and air trapping. Scores are presented as mean (SD); no statistical testing was performed for this preliminary study.

Results Mean total CT score (sum of the subcomponent scores) decreased from 20.6 to 12.5 (mean change [SD], 8.1 [13.6]) in the LUM/IVA group and increased from 32.8 to 41.4 (8.6 [14.6]) in the placebo group. The mean bronchiectasis score decreased from 3.2 to 2.5 (0.7 [1.3]) in the LUM/IVA group and increased from 6.4 to 8.1 (1.7 [2.1]) in the placebo group. Additionally, the diameter of ectatic bronchi decreased in several patients on active treatment. The mean air trapping score decreased from 7.8 to 5.9 (mean difference [SD], 1.9 [6.8]) in the active group and increased from 9.8 to 14.5 (4.7 [11.7]) in the placebo group, as shown for 1 patient (figure 1).

Conclusion This is the first report to describe CT lung findings after CFTR corrector/potentiator therapy in patients aged 6 to 11 years homozygous for F508del. In this 24 week exploratory analysis, bronchiectasis and air trapping scores improved in patients treated with LUM/IVA and worsened in the placebo group. These data suggest that LUM/IVA treatment may reduce CF disease-related changes in lung morphology and/or physiology and support the need for further study.

Please refer to page A257 for declarations of interest in relation to abstract S93.

Abstract S93 Figure 1
Abstract S93 Figure 1

Computed tomography images (residual volume) showing air trapping (arrows) at baseline and 24 weeks in a patient treated with lumacaftor/ivacaftor.

https://doi.org/10.1136/thoraxjnl-2017-210983.99

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