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Association of CFTR activity in sweat test, NPD, and ICM with ivacaftor and lumacaftor serum levels in Phe508del homozygous patients with cystic fibrosis
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  • Christian Dopfer,
  • Rainer Trittler,
  • Simon Y. Graeber,
  • Jesús Mozo Casado,
  • Carmen Diaz Enriquez,
  • Mirjam Stahl,
  • Anna Maria Dittrich,
  • Lutz Naerlich,
  • Marcus Mall,
  • Burkhard Tümmler,
  • Martin J. Hug
Christian Dopfer
Medizinische Hochschule Hannover Klinik fur Padiatrische Pneumologie Allergologie und Neonatologie

Corresponding Author:[email protected]

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Rainer Trittler
Universitatsklinikum Freiburg
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Simon Y. Graeber
Medizinische Hochschule Hannover
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Jesús Mozo Casado
Universitatsklinikum Freiburg
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Carmen Diaz Enriquez
Universitatsklinikum Freiburg
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Mirjam Stahl
Medizinische Hochschule Hannover
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Anna Maria Dittrich
Medizinische Hochschule Hannover Klinik fur Padiatrische Pneumologie Allergologie und Neonatologie
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Lutz Naerlich
Universitatsklinikum Giessen und Marburg GmbH Standort Giessen
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Marcus Mall
Medizinische Hochschule Hannover
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Burkhard Tümmler
Medizinische Hochschule Hannover Klinik fur Padiatrische Pneumologie Allergologie und Neonatologie
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Martin J. Hug
Universitatsklinikum Freiburg
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Abstract

Combination therapy with the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) corrector lumacaftor and the CFTR potentiator ivacaftor has demonstrated significant impact on clinical parameters in Phe508del homozygous people with CF. Whether these changes under treatment are correlated to serum levels of both drugs had yet to be investigated. We therefore analyzed data from our previous study (OrkambiFacts, ClinicalTrials.gov Identifier: NCT02807415). In summary, we did not find statistically significant correlations between serum drug levels and changes in clinical parameters and biomarkers of CFTR function such as FEV1, BMI, sweat chloride, nasal potential difference (NPD) and intestinal current measurement (ICM). Absolute blood levels of lumacaftor or ivacaftor do not seem to be informative biomarkers to predict clinical improvement or the attenuation of the basic defect.
28 Aug 2022Submitted to Pediatric Pulmonology
28 Aug 2022Submission Checks Completed
28 Aug 2022Assigned to Editor
01 Sep 2022Reviewer(s) Assigned
10 Oct 2022Review(s) Completed, Editorial Evaluation Pending
11 Oct 2022Editorial Decision: Revise Major
11 Dec 20221st Revision Received
12 Dec 2022Review(s) Completed, Editorial Evaluation Pending
12 Dec 2022Submission Checks Completed
12 Dec 2022Assigned to Editor
12 Dec 2022Reviewer(s) Assigned
07 Jan 2023Editorial Decision: Revise Minor
24 Mar 20232nd Revision Received