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Livestreamed Session
Livestreamed Session

EPP

PTAC/CFTR
Session Type: Workshop

W17--Extrapulmonary Manifestations & Mechanisms

Friday, October 24, 2025
7:45 AM - 9:45 AM PDT
Location:427-429

High-efficiency CFTR modulator therapies (HEMTs) have revolutionized the treatment landscape for people with cystic fibrosis (CF) by targeting the underlying CFTR protein defect. These therapies have led to significant improvements in pulmonary function, quality of life, and life expectancy. However, their effects on extrapulmonary manifestations of CF are more variable and remain incompletely understood.
As individuals with CF live longer, new extrapulmonary complications may emerge, reflecting both the effects of chronic disease and aging, as well as unanticipated consequences of long-term HEMT use. While some extrapulmonary symptoms may improve, others may persist or even develop de novo.
Despite the substantial benefits of HEMTs, their long-term impact on specific organ systems—including the brain, liver, pancreas, kidneys, and reproductive tract—remains under active investigation. This underscores the critical need for ongoing research, longitudinal clinical monitoring, and mechanistic studies to fully understand the evolving phenotype of CF in the era of modulator therapy.

Learning Objectives:

  • Increase awareness of the evolving clinical phenotype of cystic fibrosis (CF) in the era of HEMTs.
  • Review the spectrum of extrapulmonary manifestations in people with CF.
  • Understand the potential effects of early exposure to HEMTs during development as well as the long-term impact of HEMT use in the aging CF population.

Presentations:

  • 7:45 AM - 9:45 AM PDT
    W17.1- Neurological outcomes of in-utero treatment with ETI

    Speaker: Paul M. Litman, n/a – Case Western Reserve University

  • 7:45 AM - 9:45 AM PDT
    W17.2- Disruption of hippocampal function by ETI therapy

    Speaker: Veronica A. Campanucci

  • 7:45 AM - 9:45 AM PDT
    W17.3- Antigen-experienced CD4+ T cell clonal expansion characterizes CF-related diabetes

    Speaker: Camilla Margaroli – University of Alabama at Birmingham

  • 7:45 AM - 9:45 AM PDT
    W17.4- Absent CFTR function is associated with progressive kidney injury and abnormal mineral metabolism in an in vivo mouse model

    Speaker: Hayrettin Yavuz, MD, PhD – University of Virginia

  • 7:45 AM - 9:45 AM PDT
    W17.5- Leaky gut, dysbiosis, and inflammation support inter-organ communication in Cystic Fibrosis

    Speaker: Alessandra Bragonzi, N/A, n/a – San Raffaele Scientific Institute