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Longitudinal improvements in clinical and functional outcomes following initiation of elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis.
Vijaykumar, Kadambari; Leung, Hui Min; Barrios, Amilcar; Wade, Justin; Hathorne, Heather Y; Nichols, David P; Tearney, Guillermo J; Rowe, Steven M; Solomon, George M.
Afiliación
  • Vijaykumar K; Department of Medicine, University of Alabama at Birmingham, AL, United States.
  • Leung HM; Gregory Fleming James CF Research Center, Birmingham, AL, United States.
  • Barrios A; Wellman Center for Photomedicine, Massachusetts General Hospital, Boston, MA, United States.
  • Wade J; Wellman Center for Photomedicine, Massachusetts General Hospital, Boston, MA, United States.
  • Hathorne HY; Gregory Fleming James CF Research Center, Birmingham, AL, United States.
  • Nichols DP; Gregory Fleming James CF Research Center, Birmingham, AL, United States.
  • Tearney GJ; University of Washington, Seattle, WA, United States.
  • Rowe SM; Wellman Center for Photomedicine, Massachusetts General Hospital, Boston, MA, United States.
  • Solomon GM; Department of Medicine, University of Alabama at Birmingham, AL, United States.
Heliyon ; 10(8): e29188, 2024 Apr 30.
Article en En | MEDLINE | ID: mdl-38681615
ABSTRACT

Background:

Use of elexacaftor/tezacaftor/ivacaftor (ETI) for treatment of cystic fibrosis (CF) has resulted in unprecedented clinical improvements necessitating development of outcome measures for monitoring disease course. Intranasal micro-optical coherence tomography (µOCT) has previously helped detect and characterize mucociliary abnormalities in patients with CF. This study was done to determine if µOCT can define the effects of ETI on nasal mucociliary clearance and monitor changes conferred to understand mechanistic effects of CFTR modulators beyond CFTR activation.

Methods:

26 subjects, with at least 1 F508del mutation were recruited and followed at baseline (visit 1), +1 month (visit 2) and +6 months (visit 4) following initiation of ETI therapy. Clinical outcomes were computed at visits 1, 2 and 4. Intranasal µOCT imaging and functional metrics analysis including mucociliary transport rate (MCT) estimation were done at visits 1 and 2.

Results:

Percent predicted forced expiratory volume in 1 s (ppFEV1) showed a significant increase of +10.9 % at visit 2, which sustained at visit 4 (+10.6 %). Sweat chloride levels significantly decreased by -36.6 mmol/L and -41.3 mmol/L at visits 2 and 4, respectively. µOCT analysis revealed significant improvement in MCT rate (2.8 ± 1.5, visit 1 vs 4.0 ± 1.5 mm/min, visit 2; P = 0.048).

Conclusions:

Treatment with ETI resulted in significant and sustained clinical improvements over 6 months. Functional improvements in MCT rate were evident within a month after initiation of ETI therapy indicating that µOCT imaging is sensitive to the treatment effect of HEMT and suggests improved mucociliary transport as a probable mechanism of action underlying the clinical benefits.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Heliyon Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Heliyon Año: 2024 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Reino Unido