Randomised controlled trials in cystic fibrosis: what, when and how?

Sly, Peter D.; Ware, Robert S.; Klerk, Nicholas de; Stick, Stephen M.

doi:10.1183/09031936.00166810

Cited by 5 publications

(6 citation statements)

References 14 publications

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“…Similarly, the European Cystic Fibrosis Society Clinical Trial Network (ECFS‐CTN) was set up in 2008 in Europe, and continues to expand, with 30 sites across 11 countries planned to be participating by 2012, giving access to 13,500 patients 74. These networks work closely together to standardize research procedures and outcome parameters, which will improve consistency in study design including outcome parameters, and which will assist in comparing results across studies 75…”

Section: Treatment Complexity In Cf: Opportunities For the Futurementioning

confidence: 99%

Managing treatment complexity in cystic fibrosis: Challenges and Opportunities

Sawicki

Tiddens

2012

Pediatric Pulmonology

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Cystic fibrosis (CF) is a complex, chronic, multisystem disease for which there is currently no cure. Nonetheless, advances in management have led to dramatic improvements in patient survival. With this development, new issues have arisen for CF patients and their care providers, including an increased symptom burden and increased frequency of co-morbidities as patients reach older ages, leading to the need for a highly complicated and time-consuming regimen of treatments. Such high symptom and treatment burden often leads to non-adherence and low levels of competence with administration of therapy, both of which may have detrimental impacts on CF outcomes. Optimal management is also hindered by other patient-related factors, including inadequacies in disease education which may lead to issues with self-management. This is particularly important during the transition from parent-directed therapy to independent self-management that occurs during adolescence and early adulthood. Clinicians are also faced with a considerable challenge when selecting interventions for individual patients; although the paradigm of aggressive care necessitates a wide range of therapies, there is a limited evidence base with which to compare available therapeutic regimens. Novel pharmacological agents are being developed to target the underlying cause of CF, while non-pharmacological interventions aim to improve competence and maximize adherence and health outcomes. Comparative effectiveness research is needed to simplify management and facilitate the implementation of appropriate treatment strategies.

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Section: Treatment Complexity In Cf: Opportunities For the Futurementioning

confidence: 99%

Managing treatment complexity in cystic fibrosis: Challenges and Opportunities

Sawicki

Tiddens

2012

Pediatric Pulmonology

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“… 46 , 50 These findings suggest that chest CT could be used to detect the presence and extent of structural lung disease, particularly as the majority of infants with lung disease may be asymptomatic. 46 …”

Section: Introductionmentioning

confidence: 96%

“…Chest CT scans have been shown to be more sensitive at detecting disease severity than spirometry. 25 , 36 , 45 , 46 In particular, CT can be used to detect bronchiectasis and trapped air, which reflects abnormal ventilation and perfusion in infants. 47 Studies have shown that there are weak associations between the presence and extent of structural lung damage and functional parameters.…”

Section: Introductionmentioning

confidence: 99%

“… 47 Studies have shown that there are weak associations between the presence and extent of structural lung damage and functional parameters. 45 , 47 , 48 It has also been shown that infection, inflammation and abnormal chest CT findings are already present in a significant proportion of infants with CF at 3 months of age 46 , 49 and that these early structural changes are progressive. 46 , 50 These findings suggest that chest CT could be used to detect the presence and extent of structural lung disease, particularly as the majority of infants with lung disease may be asymptomatic.…”

Section: Introductionmentioning

confidence: 99%

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Novel outcome measures for clinical trials in cystic fibrosis

Tiddens

Puderbach²,

Venegas

et al. 2014

Pediatric Pulmonology

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Cystic fibrosis (CF) is a common inherited condition caused by mutations in the gene encoding the CF transmembrane regulator protein. With increased understanding of the molecular mechanisms underlying CF and the development of new therapies there comes the need to develop new outcome measures to assess the disease, its progression and response to treatment. As there are limitations to the current endpoints accepted for regulatory purposes, a workshop to discuss novel endpoints for clinical trials in CF was held in Anaheim, California in November 2011. The pros and cons of novel outcome measures with potential utility for evaluation of novel treatments in CF were critically evaluated. The highlights of the 2011 workshop and subsequent advances in technologies and techniques that could be used to inform the development of clinical trial endpoints are summarized in this review. Pediatr Pulmonol. © 2014 The Authors. Pediatric Pulmonology published by Wiley Periodicals, Inc.

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“…These areas were chosen because both tuberculosis (TB) and cystic fibrosis are high priority in the infectious disease arena [8][9][10].…”

Section: @Erspublicationsmentioning

confidence: 99%

Future directions for the ERS: Presidential plans

et al. 2013

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@ERSpublicationsThe ERS, through the Presidential plan, contributes to better prevention and management of respiratory diseases in Europe http://ow.ly/n2JWAThe mission of the European Respiratory Society (ERS) is to promote respiratory health and alleviate suffering from respiratory disease and it fulfils this goal by promoting science/research, education and advocacy. Among the Society's activities are a successful annual Congress (attended by approximately 20,000 delegates), research fellowships, grants and seminars and a comprehensive educational programme through the ERS School [1][2][3][4][5]. The educational programme encompasses postgraduate courses during the Congress, courses and seminars on various topics throughout the year across Europe, a website with a wealth of educational modules and interactive materials. Importantly, the ERS School has initiated a training programme called Harmonisation in Education in Respiratory Medicine for European Specialists (HERMES), which sets training standards across Europe, runs examination and certification processes for the European Diplomas in Respiratory Medicine and Paediatric Respiratory Medicine, and allows mobility of European specialists. Furthermore, the ERS has a strong advocacy role through its Advocacy Committee and Brussels office, and through the European Lung Foundation (ELF) -the public voice of the ERS, which links with patients and patient organisations, provides information and listens to and empowers the patients' voices.In a dynamic European and global setting, where health priorities in general (and respiratory medicine in particular) are changing rapidly, the ERS is planning to do even more in order to attract the attention of the media and policymakers and to bring more research funding into respiratory medicine. The ERS is proud to be the first major respiratory medicine society to have agreed and launched a public health Presidential plan, which is now being implemented.A series of ERS Summits has been organised since 2011, when the European Respiratory Roadmap, which set out directions for respiratory medicine and research over the next decade, was approved in Leuven (Belgium) and was then discussed with officials from European Union in Brussels [6]. In 2012, the Tallinn (Estonia) Summit tackled the burning issue of respiratory inequalities and highlighted the striking differences in respiratory outcomes across Europe and even within countries and the way in which this related to poverty and availability of resources. This was followed in 2013 by the Summit on ''Changing the natural history of respiratory diseases'' organised in Dublin (Ireland).

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Randomised controlled trials in cystic fibrosis: what, when and how?

Cited by 5 publications

References 14 publications

Managing treatment complexity in cystic fibrosis: Challenges and Opportunities

Managing treatment complexity in cystic fibrosis: Challenges and Opportunities

Novel outcome measures for clinical trials in cystic fibrosis

Future directions for the ERS: Presidential plans

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