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. 2024 May 8;11(1):4.
doi: 10.1186/s40348-024-00178-6.

Changes in vitamins and trace elements after initiation of highly effective CFTR modulator therapy in children and adults with cystic fibrosis - a real-life insight

Dorit Fabricius  1 , Tina Knieling  1 , Noelle Zurmuehl  1 , Leandra Makedon  1 , Joachim Freihorst  1 , Hanna Schmidt  1 , Sebastian Bode  2
Affiliations

Changes in vitamins and trace elements after initiation of highly effective CFTR modulator therapy in children and adults with cystic fibrosis - a real-life insight

Dorit Fabricius et al. Mol Cell Pediatr. .

Abstract

Background: Highly-effective CFTR-modulator therapy with elexa-/teza-/ivacaftor (ETI) has led to improvements in pulmonary outcomes, sweat chloride, body mass index (BMI) and quality of life in people with cystic fibrosis (CF). Improved uptake of fat-soluble vitamins and micronutrients has been reported for CFTR-modulators but data regarding ETI therapy is lacking.

Methods: This single-center retrospective study evaluated forced expiratory volume in one second (FEV-1), sweat chloride, BMI, transaminases (AST, ALT), bilirubin, vitamins A, D, E, zinc and selenium in children and adults eligible for ETI. Parameters were assessed before and up to one year after initiation of ETI.

Results: 58 patients (median age m = 28 years, SD ± 11.6 years, 51.7% female14 < 18 years old) were included. FEV-1 and sweat chloride improved significantly after ETI. There were no changes in BMI or AST. ALT was increased significantly after 4 weeks of ETI but returned to normal levels in further course. Bilirubin levels remained elevated after ETI. Vitamin A was significantly higher 12 months after ETI. No changes were found for vitamins D, E, zinc and selenium.

Conclusions: This study adds to the evidence that improvements of some fat-soluble vitamin levels can be found after ETI. No changes regarding micronutrients were noted. Individualized follow-up and supplementation are recommended.

Keywords: Cystic fibrosis; Highly effective CFTR modulator therapy; Individualized therapy; Micronutrients; Trace elements; Vitamins.

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Conflict of interest statement

The authors declare no competing interests.

Figures

Fig. 1
Fig. 1
A: No changes in aspartate-aminotransferase levels before and up to 12 months after start of ETI. B: Significant change of alanine-aminotransferase four weeks after initiation of ETI, no other changes. C: Significant and persistent elevation of total bilirubin levels after start of ETI at all timepoints compared to before start of ETI therapy. Reference ranges of AST (< 35 U/l), ALT (< 34 U/l) and bilirubin (2–21 µmol/l) are indicated in grey. AST = aspartate-aminotransferase, ALT = alanine-aminotransferase, ETI = elexa-/teza-/ivacaftor.
Fig. 2
Fig. 2
A: No changes in zinc plasma levels before and up to 12 months after start of ETI. B: No changes in selenium levels before and up to 12 months after start of ETI therapy in patients with cystic fibrosis. Missing data for zinc levels 4 weeks after start of ETI. Reference ranges of zinc (9–22 µmol/l) and selenium (50–120 µg/l) are indicated in grey. ETI = elexa-/teza-/ivacaftor.
Fig. 3
Fig. 3
A: Significant increase of Vitamin A 12 months after start of ETI therapy in patients with cystic fibrosis. B: No changes in serum vitamin D levels and C: No changes in vitamin E levels over the study period after initiation of ETI therapy. Missing data at timepoint 4 weeks after start of ETI for vitamins A & E. Reference ranges of vitamin A (1.05–2.8 µmol/l), vitamin D (20–50 µg/l) and vitamin E (12–42 µmol/l) are indicated in grey. ETI = elexa-/teza-/ivacaftor.
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