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CFTR Modulator Use Is Associated with Higher Hemoglobin Levels in Individuals with Cystic Fibrosis

Alex H Gifford1,2, Sonya L Heltshe3,4, Christopher H Goss3,4,5

  • 11 Division of Pulmonary and Critical Care Medicine, Dartmouth-Hitchcock Medical Center, Lebanon, New Hampshire.

Annals of the American Thoracic Society
|December 25, 2018
PubMed

Insights

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators like ivacaftor and lumacaftor/ivacaftor are linked to increased hemoglobin levels in patients with CF. This finding is significant for managing anemia in the cystic fibrosis community.

Area of Science:

  • Pulmonary Medicine
  • Genetics
  • Hematology

Background:

  • Cystic fibrosis transmembrane conductance regulator (CFTR) modulators represent a significant advancement in CF treatment.
  • The impact of CFTR modulators on comorbid conditions, such as anemia, is an area of growing interest.
  • Understanding these effects is crucial for comprehensive patient care.

Purpose of the Study:

  • To investigate the association between CFTR modulators and hemoglobin (Hgb) levels in individuals with cystic fibrosis.
  • To test the hypothesis that CFTR modulator therapy leads to elevated Hgb levels.

Main Methods:

  • Retrospective analysis of annualized Hgb and clinical data from the U.S. CF Foundation Patient Registry (2010-2016).
  • Inclusion of adult and pediatric patients treated with ivacaftor (IVA) or lumacaftor/ivacaftor (LUM/IVA).
  • Application of linear mixed models and linear regression to assess the effect of CFTR modulators on Hgb levels.

Main Results:

  • Ivacaftor (IVA) use was associated with increased Hgb levels in G551D-CFTR patients (males: +0.54 gm/dl, females: +0.18 gm/dl).
  • Lumacaftor/ivacaftor (LUM/IVA) use showed significant Hgb increases in F508del-CFTR homozygotes (males: +0.58 gm/dl, females: +0.26 gm/dl).
  • Multivariate analysis confirmed positive Hgb effects of IVA in males and LUM/IVA in both sexes.

Conclusions:

  • Both ivacaftor (IVA) and lumacaftor/ivacaftor (LUM/IVA) therapies are associated with higher hemoglobin levels in cystic fibrosis patients.
  • These findings suggest a potential benefit of CFTR modulators in addressing anemia in CF.
  • Further research may explore the mechanisms behind this hematological improvement.
Abstract

Keywords:
anemiacystic fibrosishemoglobinivacaftorlumacaftor

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