Publications by authors named "Erica Roesch"

This case report presents a comprehensive evaluation of the complex balance of therapeutic benefits and potential risks associated with the cystic fibrosis transmembrane conductance regulator (CFTR) modulator elexacaftor/tezacaftor/ivacaftor (ETI) therapy in managing an eight-year-old male with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI). While ETI therapy significantly enhanced exocrine pancreatic function, it led to hepatotoxicity, necessitating therapy discontinuation. Attempts to restart ETI at reduced doses were unsuccessful due to persistent hepatic dysfunction.

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Introduction: The availability of cystic fibrosis transmembrane conductance regulator (CFTR) modulators opens the possibility of discontinuing some chronic pulmonary therapies to decrease cystic fibrosis (CF) treatment burden. However, CFTR modulators may not adequately address neutrophilic inflammation, which contributes to a self-perpetual cycle of viscous CF sputum, airway obstruction, inflammation, and lung function decline.

Areas Covered: This review discusses the emerging role of neutrophil extracellular traps in CF and its role in CF sputum viscosity, airway obstruction, and inflammation, based on a literature search of PubMed (1990-present).

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Purpose Of Review: Cystic fibrosis is a genetic disease that increases risk of death from respiratory failure because of impairment in mucociliary clearance. Complex daily care regimens including medications and airway clearance techniques (ACTs) aim to preserve lung function and alleviate symptoms for people with cystic fibrosis (pwCF). The success of highly effective modulator therapy (HEMT) permits evaluation of treatment simplification.

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Human Mesenchymal Stem Cell (hMSC) immunotherapy has been shown to provide both anti-inflammatory and anti-microbial effectiveness in a variety of diseases. The clinical potency of hMSCs is based upon an initial direct hMSC effect on the pro-inflammatory and anti-microbial pathophysiology as well as sustained potency through orchestrating the host immunity to optimize the resolution of infection and tissue damage. Cystic fibrosis (CF) patients suffer from a lung disease characterized by excessive inflammation and chronic infection as well as a variety of other systemic anomalies associated with the consequences of abnormal cystic fibrosis transmembrane conductance regulator (CFTR) function.

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A 24-week, phase 3, open-label study showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in children aged 6-11 years with cystic fibrosis (CF) and one or more alleles. To assess long-term safety and efficacy of ELX/TEZ/IVA in children who completed the pivotal 24-week phase 3 trial. In this phase 3, two-part (part A and part B), open-label extension study, children aged ⩾6 years with CF heterozygous for and a minimal function mutation (/MF genotypes) or homozygous for ( genotype) who completed the 24-week parent study received ELX/TEZ/IVA based on weight.

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Article Synopsis
  • Mesenchymal stem cells (MSCs) from bone marrow are being explored as a treatment for cystic fibrosis (CF), potentially reducing lung inflammation and improving antibiotic effectiveness.
  • A phase 1 clinical trial enrolled 15 adults with CF to test the safety and tolerability of different doses of intravenous hMSCs, with no serious adverse events reported during the study period.
  • The preliminary results indicate that hMSC infusions were safe and well-tolerated, suggesting they are worth further investigation as a potential therapy for CF lung disease.
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Purpose: The prevalence of eosinophilic esophagitis (EoE) has been on the rise since it was first described in the 1990s. Several diseases and exogenous factors have been associated with EoE. Our aim was to investigate the epidemiology of EoE in cystic fibrosis (CF) patients.

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Challenging mealtime behaviors in young children and difficulties in meeting their dietary intake recommendations are sources of parenting stress and associated with negative quality of life. The gastrointestinal (GI) manifestations of cystic fibrosis (CF) can often present similarly to a GI pathology unrelated to CF. Specifically, this case series focuses on three toddlers with CF who presented with oral aversion and challenging mealtime behaviors and later were diagnosed with eosinophilic esophagitis (EoE).

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Introduction: is a common respiratory pathogen that contributes to chronic pulmonary infection in individuals with cystic fibrosis. Guidelines recommend early intervention upon positive culture. Tobramycin has in vitro activity against Gram-negative bacteria, including , and TOBI Podhaler is indicated for the management of individuals with cystic fibrosis with infection.

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Background: Individuals with cystic fibrosis (CF) experience elevated inflammation in multiple organs, but whether this reflects an inherent feature of CF cells or is a consequence of a pro-inflammatory environment is not clear.

Method: Using CRISPR/Cas9-mediated mutagenesis of CFTR, 17 subclonal cell lines were generated from Caco-2 cells. Clonal lines with functional CFTR (CFTR) were compared to those without (CFTR) to directly address the role of CFTR in inflammatory gene regulation.

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Inflammation plays a critical role in cystic fibrosis (CF) lung pathology and disease progression making it an active area of research and important therapeutic target. In this review, we explore the most recent research on the major contributors to the exuberant inflammatory response seen in CF as well as potential therapeutics to combat this response. Absence of functional cystic fibrosis transmembrane conductance regulator (CFTR) alters anion transport across CF airway epithelial cells and ultimately results in dehydration of the airway surface liquid.

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Recent discoveries and technical advances in genetic engineering, methods called gene or genome editing, provide hope for repairing genes that cause diseases like cystic fibrosis (CF) or otherwise altering a gene for therapeutic benefit. There are both hopes and hurdles with these technologies, with new ideas emerging almost daily. Initial studies using intestinal organoid cultures carrying the common, F508del mutation have shown that gene editing by CRISPR/Cas9 can convert cells lacking CFTR function to cells with normal channel function, providing a precedent that this technology can be harnessed for CF.

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