Evolution and Prognostic Variables of Cystic Fibrosis in Children and Young Adults: A Narrative Review
Mădălina Andreea Donos1, Elena Țarcă2, Elena Cojocaru3
1Pediatrics Department, "Grigore T. Popa" University of Medicine and Pharmacy, 700115 Iasi, Romania.
Insights
Predicting cystic fibrosis (CF) outcomes requires personalized care. Clinical and biological markers, alongside genetics, help tailor treatments for improved survival in CF patients.
Area of Science:
- Medical Genetics
- Pulmonology
- Pediatrics
Background:
- Cystic fibrosis (CF) is a genetic disorder impacting multiple organ systems.
- Increasing life expectancy in CF necessitates improved prognostic tools.
- A growing number of CFTR variants influences CF population statistics.
Purpose of the Study:
- To review recent findings on clinical parameters for predicting CF outcomes.
- To assess the impact of various factors on CF prognosis from prenatal to adult stages.
- To highlight the importance of tailored treatment strategies in CF care.
Main Methods:
- Conducted a narrative review of existing literature.
- Searched PubMed using terms: prognostic factors, children, cystic fibrosis, mucoviscidosis.
- Evaluated articles focusing on CF evolution in pediatric and young adult populations.
Main Results:
- Customized CF patient care is essential, considering clinical, biological, genetic, and comorbidity factors.
- Dynamic clinical markers offer realistic targets for individualized CF treatment.
- Improvements in diagnostics and care over the last decade have positively impacted CF prognosis.
Conclusions:
- Genotyping aids CF research and treatment categorization but doesn't encompass all prognostic factors.
- Epigenetics, genetic modifiers, environment, and socioeconomic status influence CF outcomes.
- Early identification, evidence-based research, and multidisciplinary care enhance long-term CF management.
Abstract:
Introduction: Cystic fibrosis (CF) is a genetic condition affecting several organs and systems, including the pancreas, colon, respiratory system, and reproductive system. The detection of a growing number of CFTR variants and genotypes has contributed to an increase in the CF population which, in turn, has had an impact on the overall statistics regarding the prognosis and outcome of the condition. Given the increase in life expectancy, it is critical to better predict outcomes and prognosticate in CF. Thus, each person's choice to aggressively treat specific disease components can be more appropriate and tailored, further increasing survival. The objective of our narrative review is to summarize the most recent information concerning the value and significance of clinical parameters in predicting outcomes, such as gender, diabetes, liver and pancreatic status, lung function, radiography, bacteriology, and blood and sputum biomarkers of inflammation and disease, and how variations in these parameters affect prognosis from the prenatal stage to maturity. Materials and methods: A methodological search of the available data was performed with regard to prognostic factors in the evolution of CF in children and young adults. We evaluated articles from the PubMed academic search engine using the following search terms: prognostic factors AND children AND cystic fibrosis OR mucoviscidosis. Results: We found that it is crucial to customize CF patients' care based on their unique clinical and biological parameters, genetics, and related comorbidities. Conclusions: The predictive significance of more dynamic clinical condition markers provides more realistic future objectives to center treatment and targets for each patient. Over the past ten years, improvements in care, diagnostics, and treatment have impacted the prognosis for CF. Although genotyping offers a way to categorize CF to direct research and treatment, it is crucial to understand that a variety of other factors, such as epigenetics, genetic modifiers, environmental factors, and socioeconomic status, can affect CF outcomes. The long-term management of this complicated multisystem condition has been made easier for patients, their families, and physicians by earlier and more accurate identification techniques, evidence-based research, and centralized expert multidisciplinary care.
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