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Open research questions in Cystic Fibrosis Research Advances

34 unresolved questions extracted from the limitations and future-work sections of 324 Cystic Fibrosis Research Advances papers in our library. Each links back to the study that raised it.

What the literature leaves open

  • Introduction Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have transformed cystic fibrosis care, but their role in CFTR-related disorders is not well defined.

    Resolution of recurrent pancreatitis and nutritional recovery with CFTR modulator therapy in CFTR-related disorder: a case report · 2026 · DOI

  • aeruginosa isolates adapted for years to the specific CF lung environment efficiently colonize and survive in the non-CF allograft environment remains unclear.

    Adaptation of Pseudomonas aeruginosa to the lung allograft environment in cystic fibrosis lung transplant recipients · 2026 · DOI

  • IntroductionPostal sputum sampling represents a potential strategy for patient-led, efficient, and regular sampling, yet little is known about the validity of posting samples for clinical and research purposes in bronchiectasis.

    Postal sputum samples for clinical and research applications in chronic lung infections caused by Pseudomonas aeruginosa · 2026 · DOI

  • Importantly, they suggest {beta}-lactams may be effective in treating MRSA infections in CF patients, warranting further investigation in relevant in vivo systems.

    Clinically-relevant altered antibiotic responses and mechanisms of β-lactam sensitization of MRSA in cystic fibrosis artificial sputum · 2026 · DOI

  • RationaleThe CFTR function-phenotype relationship remains incompletely understood, with prior work yielding heterogeneous findings suggesting linear and nonlinear associations.

    Clinical and primary cell evidence reveals complex CFTR function-phenotype relationships · 2026 · DOI

  • Cystic fibrosis is traditionally framed as a dichotomy between affected individuals and clinically unaffected carriers, yet the systemic immune consequences across the spectrum of CFTR functionality remain incompletely defined.

    Multimodal mapping of systemic inflammation and immunity acrossthe spectrum of CFTR dysfunction · 2026 · DOI

  • This study has several limitations. First, the relatively small sample size may limit statistical power and the gen- eralizability of the findings. Second, asthma diagnosis was based on a combination of clinical and spirometric assessment; however, inconsistent objective confirma- tion, along with predominantly non-obstructive baseline spirometry and the absence of variability assessment, may have introduced a risk of misclassification and lim- ited diagnostic validity. In addition, the use of bronchodilator reversibility testing in a predominantly non-obstructive population reduces its diagnostic utility, particularly in the absence of consistently reported absolute FEV₁ changes (mL). The cross-sectional design and lack of longitudinal follow- up also limit the ability to assess disease progression and causal relationships. Furthermore, potential recall bias in determining dis- ease chronology and recruitment from a single respira- tory center may introduce selection bias and affect the external validity of the results. The primarily descriptive nature of the analysis, without multivariable adjustment for potential confounders, may limit deeper interpreta- tion of the observed associations. Finally, variability in the application of standardized assessment tools may affect reproducibility.

    Clinical and spirometric characteristics of patients with asthma and bronchiectasis: a comparative observational study · 2026 · DOI

  • Introduction: Fungal organisms are increasingly isolated from respiratory samples of patients with cystic fibrosis (CF), yet their clinical relevance remains incompletely understood.

    Fungal colonization and its clinical associations in pediatric cystic fibrosis: A 12-year single-center cohort stud · 2025 · DOI

  • Abstract Rationale Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and effective in people with cystic fibrosis (CF) aged ≥6 years with at least one F508del-CFTR allele but has not been studied in younger children.

    Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2–5 Years with Cystic Fibrosis and at Least One <i>F508del</i> Allele · 2023 · DOI

  • While vancomycin poses a significant risk of nephrotoxicity, the relationship between vancomycin exposure and nephrotoxicity in PwCF has not been characterized.

    Population Pharmacokinetic and Exposure‐Response Analysis of Vancomycin Nephrotoxicity in Cystic Fibrosis Patients · 2026 · DOI

  • A substantial body of evidence from clinical trials of uncontrolled asthmasubstantiates the necessity for further investigation into the infl uence of additional adverse environmental factors, including electronic smoking.

    DIAGNOSTIC CRITERIA AND RISK FACTORS FOR PULMONARY FIBROSIS IN PEDIATRIC PATIENTS WITH BRONCHIAL ASTHMA · 2024 · DOI

  • ABPA is mostly seen in steroid dependent adult asthma patients and has rarely been reported in pediatric asthma patients.

    Two different diseases in two adolescent girls with the same clinic: Allergic bronchopulmonary aspergillosis · 2024 · DOI

  • Explanations for this gender disparity are only beginning to be unraveled and further investigation into mechanisms is needed to help develop therapies that may narrow this gender gap.

    Gender Differences in Outcomes of Patients with Cystic Fibrosis · 2014 · DOI

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34 open questions have been extracted from the limitations and future-work passages of 324 Cystic Fibrosis Research Advances papers in our library. Each one below links back to the study that raised it, so you can read the original claim in context.

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