Evidence map›Paper›PMID 42346180›Full record

ArticleBritish journal of clinical pharmacology2026

A descriptive case series of hepatotoxicity associated with CFTR modulators and possible relevance of pharmacogenetic polymorphisms in cystic fibrosis patients.

Clara Laffitte Redondo, Venceslas Bourdin, Abd El Kader Ait Tayeb, Sihem Benaboud, Inès Martins, Caroline Joyau, Myriam Benhamida, Isabelle Sermet-Gaudelus, Clémence Martin, Pierre Régis Burgel and 3 more

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In one paragraph

Article in British journal of clinical pharmacology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors.

Clara Laffitte RedondoService de Médecine Génomique et Pharmacogénomique, CHU Kremlin Bicêtre, APHP, Paris, France.
Venceslas BourdinCentre Régional de Pharmacovigilance, Service de Pharmacologie, Hôpital Cochin, AP-HP, Université de Paris; Réseau Français de Pharmacovigilance, Paris, France.
Abd El Kader Ait TayebService de Médecine Génomique et Pharmacogénomique, CHU Kremlin Bicêtre, APHP, Paris, France.
Sihem BenaboudService de Pharmacologie Clinique, Hôpital Cochin, AP-HP, Université de Paris-Cité, Paris, France.
Inès MartinsService de Médecine Génomique et Pharmacogénomique, CHU Kremlin Bicêtre, APHP, Paris, France.
Caroline JoyauCentre Régional de Pharmacovigilance, Service de Pharmacologie Clinique, Institut de Biologie, CHU Hôtel Dieu, Nantes, France.
Myriam BenhamidaCentre de Ressources et de Compétence pour la Mucoviscidose, Hôpital Mère-Enfant, Nantes, France.
Isabelle Sermet-GaudelusINSERM U1151, Université Paris Cité, Hôpital Necker Enfants Malades, APHP, Paris, France.
Clémence MartinInstitut Cochin, Université Paris Cité, Inserm U1016, Paris, France.
Pierre Régis BurgelInstitut Cochin, Université Paris Cité, Inserm U1016, Paris, France.
Céline VerstuyftService de Médecine Génomique et Pharmacogénomique, CHU Kremlin Bicêtre, APHP, Paris, France.
Laurent ChouchanaCentre Régional de Pharmacovigilance, Service de Pharmacologie, Hôpital Cochin, AP-HP, Université de Paris; Réseau Français de Pharmacovigilance, Paris, France.ORCID https://orcid.org/0000-0002-9626-3571
Estelle Ayme-DietrichService de Médecine Génomique et Pharmacogénomique, CHU Kremlin Bicêtre, APHP, Paris, France.ORCID https://orcid.org/0000-0002-0613-3803

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are widely used in patients with cystic fibrosis and significantly improve respiratory function and quality of life. However, their effectiveness may be limited by liver damage, which sometimes leads to treatment discontinuation, and the mechanisms underlying this remain poorly understood. This case series explores the role of genetic polymorphisms affecting the enzymes and transporters involved in their pharmacokinetics, as well as detoxification mechanisms (CYP3A4/CYP3A5/ABCB1/SLCO1B1/GSTP1/GSTM1/GSTT1). Six cases of hepatotoxicity under CFTR modulators were assessed using the RECAM score. Among the genetic polymorphisms assessed, CYP3A4*22 (rs35599367) allele had higher allele frequency than in the European population, which usually does not express CYP3A5. This genetic variant, reducing CYP3A activity, could decrease the elimination of CFTR modulators and increase the risk of liver toxicity. Other genetic factors may also be involved. Further studies are needed to confirm these results.

Indexed as

AminophenolsChemical and Drug Induced Liver InjuryCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorCytochrome P-450 CYP3AQuinolonesAdolescentAdultChildFemaleGene FrequencyHumansMalePolymorphism, GeneticYoung AdultAminophenolsCFTR protein, humanCYP3A4 protein, humanCystic Fibrosis Transmembrane Conductance RegulatorCytochrome P-450 CYP3AQuinolonesCFTR modulatorsCYP3A4hepatotoxicityivacaftorpharmacogenetics

Identifiers

PMID42346180
PMCPMC13519765

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.