Modulator treatment for people with cystic fibrosis: moving in the right direction

Elborn, J. Stuart

doi:10.1183/16000617.0051-2020

Cited by 8 publications

(7 citation statements)

References 4 publications

(6 reference statements)

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“…The majority of treatments available for CF treat the complications and symptoms associated with the disease, independent of the genetic defect. However, over the last decade, treatment has gradually been directed towards restoring CFTR protein function, thus targeting the underlying cause of the disease [8,9]. CFTR modulators are targeted therapies that increase the processing and delivery of CFTR to the cell surface (correctors) and increase the flow of ions through activated CFTR proteins at the cell surface (potentiators) [10].…”

Section: Introductionmentioning

confidence: 99%

“…The introduction of Ivacaftor for individuals with class III and residual function mutations (e.g., G551D), followed by Tezacaftor/Ivacaftor combinations in individuals homozygous for F508del, have evidenced significant improvements in sweat chloride, pulmonary function, body weight, and overall quality of life (QoL) [9,11,12]. As of June 2020, NHS England and the European Medicines Agency (EMA) approved the use of the highly effective triple modulator therapy Tezacaftor/Ivacaftor/Elaxacaftor in individuals over the age of 12-years who present with at least one F508del mutation [8].…”

Section: Introductionmentioning

confidence: 99%

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Evaluating the Effect of Kaftrio on Perspectives of Health and Wellbeing in Individuals with Cystic Fibrosis

Aspinall

Mackintosh

Hill

et al. 2022

IJERPH

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Background: Modulator therapy represents a significant step forward in CF care and is expected to have a significant impact on the health and mortality of many individuals with CF. Studies have predominantly explored the physiological effects of modulator therapy on clinical outcomes, with little consideration of the individual lived experience of modulator therapy among adults with Cystic Fibrosis. Methods: To explore this, semi-structured interviews were conducted with 12 individuals currently taking Kaftrio, which were subsequently thematically analysed. Results: Three overarching themes were identified: (i) positive perception of Kaftrio, (ii) negative perception of Kaftrio, and (iii) the relationships with the clinical team. The experience of modulator therapy should be recognised as being unique to the individual, with perceptions of illness, self-identity, and outcomes strongly dictating the lived experience. Conclusions: There is a consensus that, while for many, the quality of life is evidently increased through the use of Kaftrio, this is not without its own challenges. This highlights the need for both individuals with CF and their clinical teams to learn to navigate this new disease landscape.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Evaluating the Effect of Kaftrio on Perspectives of Health and Wellbeing in Individuals with Cystic Fibrosis

Aspinall

Mackintosh

Hill

et al. 2022

IJERPH

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“…Ivacaftor (Kalydeko), lumacaftor/ivacaftor (Orkambi), tezacaftor/ivacaftor (Symkevi) and elexacaftor/tezacaftor/ivacaftor (Kaftrio) are all cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies that will potentiate or correct the CFTR pathway caused by defective variants within the CFTR gene [ 16 , 17 ]. Ivacaftor (Kalydeko) and lumacaftor/ivacaftor (Orkambi) are available as granules and tablets and the remaining CFTR modulators are all available as tablets only [ 7 ].…”

Section: Non-interchangeable Oral Formulationsmentioning

confidence: 99%

It's not all about inhaled treatment: challenges with oral therapy in paediatric respiratory medicine

Sareen

Ramphul

Bhatt

2021

Breathe

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Advances in therapies and management of conditions encountered by paediatric respiratory specialists have led to improved outcomes and improved survival rates dramatically in chronic diseases such as cystic fibrosis. However, this has also meant an increase in treatment burden. A variety of inhaled treatments are crucial in managing paediatric respiratory diseases, but these patients also have to take many oral medications. It is widely recognised that developing oral formulations appropriate for the paediatric population can affect how well a product is received by patients and their families. Consideration should be given to palatability and the number of medicines to be administered as these can all contribute to treatment adherence.Polypharmacy specifically in the context of management of patients with cystic fibrosis is not a new concept, but the recently introduced cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies and their potential for interactions and adverse reactions create novel challenges. There are some strategies that families and healthcare professionals can implement to reduce treatment burden. This review will also provide some insight into the life of a teenager with cystic fibrosis and the relative complexities of her treatment and the impacts on daily life.Educational aimsTo describe the difficulties faced by children with long-term respiratory conditions having to take oral medication.To discuss oral drug interactions that may exist within paediatric respiratory medicine and to consider issues with polypharmacy.To highlight strategies that may be used to reduce the burden of care for children on oral medication.

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“…There are a number of modulators dependent on an individual's genotype mutation: (i) ivacaftor for individuals with sequence variants associated with class III mutation; (ii) combination therapies in individuals homozygous for ΔF508 such as lumacaftor/ivacaftor or tezacaftor/ivacaftor; and (iii) a triple combination therapy (tezacaftor/ivacaftor and elexacaftor), which has been shown to be a highly effective modulator treatment for individuals with at least one ΔF508 mutation and more robust compared to tezacaftor/ivacaftor alone. These modulators have shown improvements in quality of life, reduced pulmonary exacerbations and variable improvement FEV1 in those with CF (Elborn, 2020). These drugs have revolutionised the treatment of CF, which is likely to evolve over the next decade as modulator treatment is more widely implemented.…”

Section: Pathophysiology Of Cystic Fibrosismentioning

confidence: 99%

Exploring Pulmonary Rehabilitation Strategies for those with Respiratory Conditions

McCreery¹

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Effective rehabilitation strategies are paramount to improve physiological and psychological health in pulmonary disease. The aim of this thesis was to investigate traditional and alternative pulmonary rehabilitation strategies in those with chronic respiratory disease. Chapter Four found that traditional pulmonary rehabilitation (PR) was physiologically and psychologically effective, regardless of respiratory disease, with socioeconomic status being a key determinant of adherence. Chapter Five investigated the feasibility and acceptability of IMT. Children aged 10.8 ± 0.8 years with Cystic Fibrosis (CF) enjoyed the IMT intervention, perceiving improvements in their physical ability and psychosocial health. The care team highlighted that future interventions needed to be longer and to monitor engagement and adherence. Chapter Six assessed the effectiveness of an alternative rehabilitation strategy, using a four-week inspiratory muscle training (IMT) intervention, on lung function and heart rate variability in children with CF aged 10.8 ± 1.1 years. There were significant and clinically meaningful increases in respiratory muscle strength, a clinically meaningful decrease in sympathetic modulation, and decreases in respiratory symptoms. Subsequently, utilising the formative, physiological and psychological findings derived from Chapters Five and Six, an eight-week IMT intervention with live biofeedback, performed at 80% maximal inspiratory pressure, three times a week was implemented, with an eight-week optional IMT top-up. Overall, Chapter Seven found that eight weeks of IMT elicited significant increases in respiratory muscle strength, aerobic capacity and in CF-specific questionnaire domains in children (11.0 ± 2.2 years) with CF, which were maintained following the eight-week top-up period. Chapter Eight demonstrated significant improvements in inspiratory muscle strength and endurance after eight weeks, with sustained improvements in physiological health after 16-weeks in adults with bronchiectasis (64.5 ± 10.3 years). CF and bronchiectasis participants demonstrated high levels of adherence and reported competency and autonomy. Overall, IMT may be an effective and feasible alternative to pulmonary rehabilitation.

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Modulator treatment for people with cystic fibrosis: moving in the right direction

Cited by 8 publications

References 4 publications

Evaluating the Effect of Kaftrio on Perspectives of Health and Wellbeing in Individuals with Cystic Fibrosis

Evaluating the Effect of Kaftrio on Perspectives of Health and Wellbeing in Individuals with Cystic Fibrosis

It's not all about inhaled treatment: challenges with oral therapy in paediatric respiratory medicine

Exploring Pulmonary Rehabilitation Strategies for those with Respiratory Conditions

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