Cystic Fibrosis: Emergence of Highly Effective Targeted Therapeutics and Potential Clinical Implications

Mall, Marcus; Mayer-Hamblett, Nicole; Rowe, Steven M.

doi:10.1164/rccm.201910-1943so

Cited by 176 publications

(136 citation statements)

References 167 publications

Supporting

Mentioning

129

Contrasting

Unclassified

Order By: Relevance

“…Targeted therapies are effective for only a certain percentage of patients with CF and are not curative agents. 46 Therapies have improved markers of disease activity, such as forced expiratory volume and reduced pulmonary exacerbations. [14][15][16] Analogously, therapies for SCD have also improved fetal Downloaded From: https://jamanetwork.com/ on 08/21/2020 hemoglobin levels and reduced vasoocclusive crises.…”

Section: Pharmaceutical Funding and Drug Developmentmentioning

confidence: 99%

Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity

et al. 2020

View full text Add to dashboard Cite

IMPORTANCE Sickle cell disease (SCD) and cystic fibrosis (CF) are severe autosomal recessive disorders associated with intermittent disease exacerbations that require hospitalizations, progressive chronic organ injury, and substantial premature mortality. Research funding is a limited resource and may contribute to health care disparities, especially for rare diseases that disproportionally affect economically disadvantaged groups. OBJECTIVE To compare disease-specific funding between SCD and CF and the association between funding and research productivity. DESIGN, SETTING, AND PARTICIPANTS This cross-sectional study examined federal and foundation funding, publications indexed in PubMed, clinical trials registered in ClinicalTrials.gov, and new drug approvals from January 1, 2008, to December 31, 2018, in an estimated US population of approximately 90 000 individuals with SCD and approximately 30 000 individuals with CF. MAIN OUTCOMES AND MEASURES Federal and foundation funding, publications indexed in PubMed, clinical trial registrations, and new drug approvals.

show abstract

Section: Pharmaceutical Funding and Drug Developmentmentioning

confidence: 99%

Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity

et al. 2020

View full text Add to dashboard Cite

show abstract

“…Lung disease remains the leading cause of morbidity and mortality in patients with cystic fibrosis (CF) and starts in the first months of life. Therefore, early diagnosis and therapeutic intervention may be most beneficial [1][2][3][4][5][6]. To determine efficacy of new therapies that address early CF lung disease in multicentre studies, sensitive, quantitative noninvasive end-points are needed to detect early lung abnormalities and disease progression in preschool children with CF [7].…”

Section: Introductionmentioning

confidence: 99%

Multicentre feasibility of multiple-breath washout in preschool children with cystic fibrosis and other lung diseases

et al. 2020

Self Cite

View full text Add to dashboard Cite

BackgroundMultiple-breath washout (MBW)-derived lung clearance index (LCI) detects early cystic fibrosis (CF) lung disease. LCI was used as an end-point in single- and multicentre settings at highly experienced MBW centres in preschool children. However, multicentre feasibility of MBW in children aged 2–6 years, including centres naïve to this technique, has not been determined systematically.MethodsFollowing central training, 91 standardised nitrogen MBW investigations were performed in 74 awake preschool children (15 controls, 46 with CF, and 13 with other lung diseases), mean age 4.6±0.9 years at investigation, using a commercially available device across five centres in Germany (three experienced, two naïve to the performance in awake preschool children) with central data analysis. Each MBW investigation consisted of several measurements.ResultsOverall success rate of MBW investigations was 82.4% ranging from 70.6% to 94.1% across study sites. The number of measurements per investigation was significantly different between sites ranging from 3.7 to 6.2 (p<0.01), while the mean number of successful measurements per investigation was comparable with 2.1 (range, 1.9 to 2.5; p=0.46). In children with CF, the LCI was increased (median 8.2, range, 6.7–15.5) compared to controls (median 7.3, range 6.5–8.3; p<0.01), and comparable to children with other lung diseases (median 7.9, range, 6.6–13.9; p=0.95).ConclusionThis study demonstrates that multicentre MBW in awake preschool children is feasible, even in centres previously naïve, with central coordination to assure standardised training, quality control and supervision. Our results support the use of LCI as multicentre end-point in clinical trials in awake preschoolers with CF.

show abstract

“…This is the result of the development within many areas of CF care. Many factors are responsible for these major advances in treatment such as standardisation with high-quality healthcare of patients in specialised centres by multidisciplinary teams, better control of pulmonary infection with the development of new inhaled therapies, better control of Pseudomonas aeruginosa colonisation, availability of modern treatment including the cystic fibrosis transmembrane conductance regulator (CFTR) modulators, aggressive nutritional supplementation with pancreatic enzymes, early diagnosis through newborn screening, and lung transplantation [ 14 , 15 , 16 ]. In spite of this development, CF remains a progressive lethal disease.…”

Section: Introductionmentioning

confidence: 99%

Managing Cystic Fibrosis in Polish Healthcare

Rachel

Topolewicz

Śliwczyński

et al. 2020

IJERPH

View full text Add to dashboard Cite

The quality and length of life of patients with cystic fibrosis (CF) are determined by a number of factors including the quality of healthcare received by patients, as well as access to drug programs dedicated to this particular disease. The purpose of this paper is to present an overview of changes in the average life expectancy and mortality rate of the CF population in Poland between 2000 and 2018. Furthermore, we would like to evaluate access to healthcare services, including the drug program, guaranteed by public healthcare system, and funded by National Health Fund (NHF). The average life expectancy of patients with CF increased in the period in question from ca. 14.5 ± 7.6–24.5 ± 8.9 years (mean ± SD, p = 0.0001). We have observed a drop in the number of deaths in paediatric age during that period. Despite the increase in life expectancy, the use of health resources in patients with CF, especially the drug program, is dramatically low. Considering the fact that in Poland there was no active countrywide CF registry, now it is possible to estimate the frequency of use of CF healthcare services in various provinces exclusively on the basis of database maintained by the Polish NHF.

show abstract

Cystic Fibrosis: Emergence of Highly Effective Targeted Therapeutics and Potential Clinical Implications

Cited by 176 publications

References 167 publications

Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity

Comparison of US Federal and Foundation Funding of Research for Sickle Cell Disease and Cystic Fibrosis and Factors Associated With Research Productivity

Multicentre feasibility of multiple-breath washout in preschool children with cystic fibrosis and other lung diseases

Managing Cystic Fibrosis in Polish Healthcare

Contact Info

Product

Resources

About