Real-world assessment of LCI following lumacaftor-ivacaftor initiation in adolescents and adults with cystic fibrosis

Philippe Reix; Aurélie Tatopoulos; Iulia Ioan; Muriel Le Bourgeois; Stéphanie Bui; Marie Luce Choukroun; Katia Bessaci-Kabouya; Michele Gerardin; Plamen Bokov; Jennifer Da Silva; Jean-Louis Paillasseur; Pierre Regis Burgel; French Cystic Fibrosis Reference Network study group

doi:10.1016/j.jcf.2021.06.002

Real-world assessment of LCI following lumacaftor-ivacaftor initiation in adolescents and adults with cystic fibrosis

J Cyst Fibros. 2022 Jan;21(1):155-159. doi: 10.1016/j.jcf.2021.06.002. Epub 2021 Jun 26.

Affiliations

¹ Cystic Fibrosis Center, Hospices Civils de Lyon, Lyon, France; UMR 5558 CNRS Equipe EMET Université Claude Bernard Lyon 1 Lyon, France. Electronic address: [email protected].
² Pediatric Cystic Fibrosis Center, Hôpital d'Enfants, Centre Hospitalier Universitaire de Nancy, Nancy, France.
³ Service d'Explorations Fonctionnelles Pédiatriques, Hôpital d'Enfants, Centre Hospitalier Universitaire de Nancy, Nancy, France.
⁴ Pediatric Respiratory Disease and Cystic Fibrosis Center National Reference Cystic Fibrosis Reference Center, Hôpital Universitaire Necker Enfants Malades, Paris, France.
⁵ Pediatric Respiratory Disease and Cystic Fibrosis Center and CIC 1401, CHU de Bordeaux, Bordeaux, France.
⁶ Service d'explorations fonctionnelles respiratoires pédiatriques, CHU de Bordeaux, Bordeaux, France.
⁷ Department of Pediatrics A and Cystic Fibrosis Center, American Memorial Hospital, Reims, France.
⁸ Pediatric Cystic Fibrosis center, Hôpital Robert Debré, AP-HP, Paris, France.
⁹ Service de Physiologie Pédiatrique, AP-HP, Hôpital Robert Debré, Paris, France; Université de Paris, UMR1141, Equipe NeoPhen, INSERM co-tutelle, Paris, France.
¹⁰ Université de Paris, Institut Cochin, Inserm, Paris U1016, France; ERN-Lung CF network, France; URC-CIC Paris Descartes Necker Cochin, AP-HP, Hôpital Cochin, Paris, France.
¹¹ Effi-stat, Paris, France.
¹² Université de Paris, Institut Cochin, Inserm, Paris U1016, France; ERN-Lung CF network, France; Respiratory Medicine and National Cystic Fibrosis Reference Center, Cochin Hospital, Assistance Publique Hôpitaux de Paris (AP-HP), Paris, France.

PMID: 34183285
DOI: 10.1016/j.jcf.2021.06.002

Abstract

Lung clearance index (LCI) is a biomarker of ventilation inhomogeneity. Data are scarce on its usefulness in daily practice for monitoring the effects of treatments in older children and adults with CF. In this French observational study of lumacaftor-ivacaftor, 63 of 845 patients (7.5%) had available LCI performed at baseline and at six (M6; n=34) or 12 months (M12; n=46) after lumacaftor-ivacaftor initiation. At inclusion, median [IQR] age was 16 years [13-17], ppFEV₁ was 72.8 [59.6-80.7], and LCI was 12.3 [10.3-15.0]. At both M6 and M12, no statistically significant LCI increases of 0.13 units or 1.34% (95% CI: -4.85-7.53) and 0.6 units or 6.66% (95% CI: -0.03-13.5) were observed. Discordant results between LCI and ppFEV₁ were observed in one-third of the patients. In daily practice, LCI monitoring in adolescents and young adults with moderate lung disease gives results that are more heterogenous than those reported in children with milder disease.

Keywords: CFTR modulators; Cystic fibrosis; Lumacaftor; Multiple breath washout.

Publication types

Observational Study
Research Support, Non-U.S. Gov't

MeSH terms

Adolescent
Aminophenols / therapeutic use*
Aminopyridines / therapeutic use*
Benzodioxoles / therapeutic use*
Chloride Channel Agonists / therapeutic use
Cohort Studies
Cystic Fibrosis / drug therapy*
Cystic Fibrosis / physiopathology*
Cystic Fibrosis Transmembrane Conductance Regulator / therapeutic use
Drug Combinations
Humans
Quinolones / therapeutic use*
Respiratory Function Tests

Substances

Aminophenols
Aminopyridines
Benzodioxoles
Chloride Channel Agonists
Drug Combinations
Quinolones
lumacaftor, ivacaftor drug combination
Cystic Fibrosis Transmembrane Conductance Regulator