scholarly journals Management of Individual Patient Expectations When Starting with Highly Effective CFTR Modulators

2021 ◽  
Vol 11 (8) ◽  
pp. 811
Author(s):  
Bente L. Aalbers ◽  
Inez Bronsveld ◽  
Regina W. Hofland ◽  
Harry G. M. Heijerman
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Lung Damage ◽  
Cell Models ◽  
Highly Effective ◽  
Near Future ◽  
Cftr Modulators ◽  
Sinonasal Symptoms ◽  
Severe Lung

Highly effective CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (ELE/TEZ/IVA will become available for an increasing number of people with cystic fibrosis (pwCF) in the near future. Before the start of this therapy, many questions may arise concerning the expected effects. We assembled the currently available data from the literature about ELE/TEZ/IVA that focused on commonly asked questions from patients. Overall, the literature so far presents a very hopeful prospect of effects, not only on lung function, but also on nutritional status, sinonasal symptoms and quality of life. The effects in patients with pwCF with severe lung damage are also favorable. Treatment is generally well tolerated. In some cases, patient-derived cell models can help in predicting the effects for individual patients.

Effect of highly effective modulator therapy on quality of life in adults with cystic fibrosis

2020 ◽  
Author(s):  
Emily DiMango ◽  
Daniel B. Spielman ◽  
Jonathan Overdevest ◽  
Claire Keating ◽  
Sarah Fracasso Francis ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Highly Effective

scholarly journals 94: Assessing symptom burden and quality of life in patients with cystic fibrosis in the era of highly effective modulator therapy

2021 ◽  
Vol 20 ◽  
pp. S47-S48
Author(s):  
A. Perez ◽  
K. Bischoff ◽  
D. Zhang ◽  
D. Dawson ◽  
M. Israni ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Symptom Burden ◽  
Highly Effective

scholarly journals Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease

2020 ◽  
Vol 29 (155) ◽  
pp. 190112 ◽  
Author(s):  
Michal Shteinberg ◽  
Jennifer L. Taylor-Cousar
Keyword(s):  
Cystic Fibrosis ◽  
Lung Disease ◽  
Case Series ◽  
Lung Damage ◽  
Cystic Fibrosis Lung Disease ◽  
Drug Compounds ◽  
The Impact ◽  
Cftr Modulators ◽  
Severe Lung Disease

Drug compounds that augment the production and activity of the cystic fibrosis (CF) transmembrane regulator (CFTR) have revolutionised CF care. Many adults and some children with CF suffer advanced and severe lung disease or await lung transplantation. While the hope is that these drug compounds will prevent lung damage when started early in life, there is an ongoing need to care for people with advanced lung disease. The focus of this review is the accumulating data from clinical trials and case series regarding the benefits of CFTR modulator therapy in people with advanced pulmonary disease. We address the impact of treatment with ivacaftor, lumacaftor/ivacaftor, tezacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor on lung function, pulmonary exacerbations, nutrition and quality of life. Adverse events of the different CFTR modulators, as well as the potential for drug–drug interactions, are discussed.

scholarly journals CFTR Modulators: Impact on Fertility, Pregnancy, and Lactation in Women with Cystic Fibrosis

2020 ◽  
Vol 9 (9) ◽  
pp. 2706 ◽  
Author(s):  
Jennifer L. Taylor-Cousar
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Genetic Disorder ◽  
Safety Data ◽  
Single Copy ◽  
The Body ◽  
Pregnancy And Lactation ◽  
The Impact ◽  
Cftr Modulators

Cystic fibrosis (CF) is a life-shortening genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. These mutations lead to abnormal ion transport in mucous membranes throughout the body, including in the respiratory and gastrointestinal and reproductive tracts. Improvements in care and therapy have led to substantial increases in the quantity and quality of life for those with CF. Consequently, women with CF are increasingly interested in having families. Although pregnancy was once discouraged for women with CF, at this point, even women with moderately severe lung disease can successfully navigate pregnancy. With the recent approval of a triple combination CFTR modulator therapy that improves lung function, nutritional status, and quality of life for people with a single copy of the most common CFTR mutation, it is expected that the number of women with CF who choose to become pregnant will continue to increase. Although animal reproduction models show no alarming signals for use during pregnancy at normal human doses, there is a paucity of human safety data in pregnancy and lactation. This review summarizes what is currently known about the impact of use of CFTR modulators on fertility, pregnancy, and lactation in women with CF.

Optimising the care and quality of life of people with cystic fibrosis: the influence of cystic fibrosis transmembrane conductance regulator modulators

2021 ◽  
pp. 1-6
Author(s):  
Nicola Shaw ◽  
Sarah Collins ◽  
Thomas Smith ◽  
Anna McCulloch ◽  
Ian Ketchell ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Healthcare Professionals ◽  
The Body ◽  
Transmembrane Conductance Regulator ◽  
Transmembrane Conductance ◽  
Hospital Beds ◽  
Cystic Fibrosis Transmembrane ◽  
Cftr Modulators

Cystic fibrosis is a life-limiting, inherited, multi-organ disease which affects many systems of the body. Until recently, treatments were only able to ameliorate symptoms, but the introduction of precision medications which modulate the underlying defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene has changed this. Notably improvements in nutrition and lung function, reduced use of antibiotics and reduced occupation rates for hospital beds have been seen. This article summarises the discussion of a group of healthcare professionals from different specialties and an expert patient, representing their personal views and experience of treating patients who are using CFTR modulators. The discussion was sponsored by an unrestricted grant from Chiesi Limited (Manchester, UK).

Quality of Life for Children with Persistent Sinonasal Symptoms

2003 ◽  
Vol 128 (1) ◽  
pp. 17-26 ◽  
Author(s):  
David J. Kay ◽  
Richard M. Rosenfeld
Keyword(s):  
Quality Of Life ◽  
Clinical Care ◽  
Longitudinal Change ◽  
Good Test ◽  
Routine Clinical Care ◽  
Before And After ◽  
The Mean ◽  
The Individual ◽  
Sinonasal Symptoms

OBJECTIVE: The goal was to validate the SN-5 survey as a measure of longitudinal change in health-related quality of life (HRQoL) for children with persistent sinonasal symptoms. DESIGN AND SETTING: We conducted a before and after study of 85 children aged 2 to 12 years in a metropolitan pediatric otolaryngology practice. Caregivers completed the SN-5 survey at entry and at least 4 weeks later. The survey included 5 symptom-cluster items covering the domains of sinus infection, nasal obstruction, allergy symptoms, emotional distress, and activity limitations. RESULTS: Good test-retest reliability ( R = 0.70) was obtained for the overall SN-5 score and the individual survey items ( R ≥ 0.58). The mean baseline SN-5 score was 3.8 (SD, 1.0) of a maximum of 7.0, with higher scores indicating poorer HRQoL. All SN-5 items had adequate correlation ( R ≥ 0.36) with external constructs. The mean change in SN-5 score after routine clinical care was 0.88 (SD, 1.19) with an effect size of 0.74 indicating good responsiveness to longitudinal change. The change scores correlated appropriately with changes in related external constructs ( R ≥ 0.42). CONCLUSIONS: The SN-5 is a valid, reliable, and responsive measure of HRQoL for children with persistent sinonasal symptoms, suitable for use in outcomes studies and routine clinical care.

Psychosocial well-being and quality of life in siblings of children with congenital heart disease: A systematic review

2021 ◽  
pp. 136749352110129
Author(s):  
Alice S Schamong ◽  
Hannah Liebermann-Jordanidis ◽  
Konrad Brockmeier ◽  
Elisabeth Sticker ◽  
Elke Kalbe
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Congenital Heart Disease ◽  
Heart Disease ◽  
Associated Factors ◽  
Congenital Heart ◽  
Negative Impact ◽  
Well Being

Congenital heart disease (CHD) is a major global health problem. Until recently, the siblings of this group did not receive much attention. This review, conducted from November 2019 to October 2020, aims to summarize knowledge about psychosocial well-being and quality of life (QoL), associated factors, and interventions for siblings of children with CHD. Systematic searches were conducted in PubMed, PsycINFO, PsycARTICLES, Web of Science via EBSCOhost, and CENTRAL. Twelve articles were included. Results showed that psychosocial well-being was impaired in 14% to 40% of siblings. Negative impact of illness was highest for CHD siblings compared to siblings of children with cancer, cystic fibrosis, or diabetes. QoL was impaired in up to one-third. Siblings of children with CHD and cancer rated their QoL lower than those of siblings of children with cystic fibrosis or type-1 diabetes. Associated factors were sibling age, gender, socioeconomic status, miscarriage, previous sibling death, visibility of illness, and severity of condition. Only one of two interventions focused on siblings of CHD children. Although data are scarce and inhomogeneous, it indicates that siblings of CHD children suffer from lower psychosocial well-being and QoL than siblings of children with other chronic conditions. Interventions to improve their situation should be developed.

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