Going the Extra Mile: Why Clinical Research in Cystic Fibrosis Must Include Children

Dobra, Rebecca; Bentley, Siân; Edmondson, C.; Ovens, Maxine; Saunders, Clare; Short, Christopher; Wilson, Gemma; Davies, Jane C.; Bush, Andy

doi:10.3390/children9071080

Cited by 4 publications

(2 citation statements)

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“…57 58 Research in children of all ages is challenging for multiple reasons. [59][60][61] These include ethical considerations; for example, neither a child nor parent can give consent to a procedure of more than minimal risk purely for research purposes, limiting the use of, for example, CT scanning or bronchoscopy. Physiological testing (eg, spirometry) might not be able to be performed by young children.…”

Section: Open Access Open Accessmentioning

confidence: 99%

Advances in the pathogenesis and personalised treatment of paediatric asthma

Scotney

Fleming

Saglani

et al. 2023

bmjmed

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The diversity of pathology of severe paediatric asthma demonstrates that the one-size-fits-all approach characterising many guidelines is inappropriate. The term “asthma” is best used to describe a clinical syndrome of wheeze, chest tightness, breathlessness, and sometimes cough, making no assumptions about underlying pathology. Before personalising treatment, it is essential to make the diagnosis correctly and optimise basic management. Clinicians must determine exactly what type of asthma each child has. We are moving from describing symptom patterns in preschool wheeze to describing multiple underlying phenotypes with implications for targeting treatment. Many new treatment options are available for school age asthma, including biological medicines targeting type 2 inflammation, but a paucity of options are available for non-type 2 disease. The traditional reliever treatment, shortacting β2 agonists, is being replaced by combination inhalers containing inhaled corticosteroids and fast, longacting β2 agonists to treat the underlying inflammation in even mild asthma and reduce the risk of asthma attacks. However, much decision making is still based on adult data extrapolated to children. Better inclusion of children in future research studies is essential, if children are to benefit from these new advances in asthma treatment.

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Section: Open Access Open Accessmentioning

confidence: 99%

Advances in the pathogenesis and personalised treatment of paediatric asthma

Scotney

Fleming

Saglani

et al. 2023

bmjmed

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“…Children have the same right to evidence-based therapy as adults but data extrapolation from adults may be inappropriate or misleading [27], therefore it is important to elaborate well-accepted patient-oriented research tools to predict CF treatment response in the pediatric population as well.…”

Section: Alternatives To Conventional Clinical Trialsmentioning

confidence: 99%

Organoid Technology and Its Role for Theratyping Applications in Cystic Fibrosis

2022

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Cystic fibrosis (CF) is a autosomal recessive, multisystemic disease caused by different mutations in the CFTR gene encoding CF transmembrane conductance regulator. Although symptom management is important to avoid complications, the approval of CFTR modulator drugs in the clinic has demonstrated significant improvements by targeting the primary molecular defect of CF and thereby preventing problems related to CFTR deficiency or dysfunction. CFTR modulator therapies have positively changed the patients’ quality of life, especially for those who start their use at the onset of the disease. Due to early diagnosis with the implementation of newborn screening programs and considerable progress in the treatment options, nowadays pediatric mortality was dramatically reduced. In any case, the main obstacle to treat CF is to predict the drug response of patients due to genetic complexity and heterogeneity. Advances in 3D culture systems have led to the extrapolation of disease modeling and individual drug response in vitro by producing mini organs called “organoids” easily obtained from nasal and rectal mucosa biopsies. In this review, we focus primarily on patient-derived intestinal organoids used as in vitro model for CF disease. Organoids combine high-validity of outcomes with a high throughput, thus enabling CF disease classification, drug development and treatment optimization in a personalized manner.

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