Jeanette Boyd - Academia.edu (original) (raw)

Papers by Jeanette Boyd

Research paper thumbnail of International consensus statement on core outcomes for clinical trials in children and adolescents with bronchiectasis – Child-BEAR-net ERS CRC

Paediatric respiratory infection and immun.

Research paper thumbnail of Quality standards for managing children and adolescents with bronchiectasis: an international consensus

Breathe

The global burden of bronchiectasis in children and adolescents is being recognised increasingly.... more The global burden of bronchiectasis in children and adolescents is being recognised increasingly. However, marked inequity exists between, and within, settings and countries for resources and standards of care afforded to children and adolescents with bronchiectasis compared with those with other chronic lung diseases. The European Respiratory Society (ERS) clinical practice guideline for the management of bronchiectasis in children and adolescents was published recently. Here we present an international consensus of quality standards of care for children and adolescents with bronchiectasis based upon this guideline.The panel used a standardised approach that included a Delphi process with 201 respondents from the parents and patients’ survey, and 299 physicians (across 54 countries) who care for children and adolescents with bronchiectasis.The seven quality standards of care statements developed by the panel address the current absence of quality standards for clinical care related...

Research paper thumbnail of Electronic cigarettes: a task force report from the European Respiratory Society

European Respiratory Journal, 2019

There is a marked increase in the development and use of electronic nicotine delivery systems or ... more There is a marked increase in the development and use of electronic nicotine delivery systems or electronic cigarettes (ECIGs). This statement covers electronic cigarettes (ECIGs), defined as “electrical devices that generate an aerosol from a liquid” and thus excludes devices that contain tobacco. Database searches identified published articles that were used to summarise the current knowledge on the epidemiology of ECIG use; their ingredients and accompanied health effects; second-hand exposure; use of ECIGs for smoking cessation; behavioural aspects of ECIGs and social impact;in vitroand animal studies; and user perspectives.ECIG aerosol contains potentially toxic chemicals. As compared to conventional cigarettes, these are fewer and generally in lower concentrations. Second-hand exposures to ECIG chemicals may represent a potential risk, especially to vulnerable populations. There is not enough scientific evidence to support ECIGs as an aid to smoking cessation due to a lack of ...

Research paper thumbnail of The European Multicentre Bronchiectasis Audit and Research Collaboration (EMBARC): experiences from a successful ERS Clinical Research Collaboration

Breathe, 2017

In contrast to airway diseases like chronic obstructive pulmonary disease or asthma, and rare dis... more In contrast to airway diseases like chronic obstructive pulmonary disease or asthma, and rare diseases such as cystic fibrosis, there has been little research and few clinical trials in bronchiectasis. Guidelines are primarily based on expert opinion and treatment is challenging because of the heterogeneous nature of the disease.In an effort to address decades of underinvestment in bronchiectasis research, education and clinical care, the European Multicentre Bronchiectasis Audit and Research Collaboration (EMBARC) was established in 2012 as a collaborative pan-European network to bring together bronchiectasis researchers. The European Respiratory Society officially funded EMBARC in 2013 as a Clinical Research Collaboration, providing support and infrastructure to allow the project to grow.EMBARC has now established an international bronchiectasis registry that is active in more than 30 countries both within and outside Europe. Beyond the registry, the network participates in design...

Research paper thumbnail of Principles of patient partnership: integrating patient perspectives into ERS Clinical Research Collaborations

Breathe

Patient and public involvement in research is increasingly considered a cornerstone of good resea... more Patient and public involvement in research is increasingly considered a cornerstone of good research practice, and the research community recognises people with lived experience as valuable stakeholders within the research process. European Respiratory Society (ERS) strongly encourages patient input into its research programme and scientific activities, working in partnership with the European Lung Foundation (ELF) to facilitate this. Based on the ERS and ELF experience and best practice in the field of patient and public involvement, we developed a set of principles to which future ERS and ELF collaborations should adhere. These principles provide guidance on how to address key challenges when planning and conducting patient and public involvement in order to develop successful partnerships with patients and drive forward patient-centred research.

Research paper thumbnail of Task Force report: European Respiratory Society guidelines for the management of children and adolescents with bronchiectasis

There is increasing awareness of bronchiectasis in children and adolescents, a chronic pulmonary ... more There is increasing awareness of bronchiectasis in children and adolescents, a chronic pulmonary disorder associated with poor quality-of-life for the child/adolescent and their parents, recurrent exacerbations and costs to the family and health systems. Optimal treatment improves clinical outcomes. Several national guidelines exist, but there are no international guidelines.The European Respiratory Society (ERS) Task Force for the management of paediatric bronchiectasis sought to identify evidence-based management (investigation and treatment) strategies. It used the ERS standardised process that included a systematic review of the literature and application of the GRADE approach to define the quality of the evidence and level of recommendations.A multidisciplinary team of specialists in paediatric and adult respiratory medicine, infectious disease, physiotherapy, primary care, nursing, radiology, immunology, methodology, patient advocacy and parents of children/adolescents with br...

Research paper thumbnail of Research priorities in alpha-1 antitrypsin deficiency (AATD) for healthcare professionals

European Respiratory Journal, 2020

Research paper thumbnail of Children's Bronchiectasis Education Advocacy and Research Network (Child-BEAR-Net): an ERS Clinical Research Collaboration on improving outcomes of children and adolescents with bronchiectasis

European Respiratory Journal, 2021

Child-BEAR-Net (Children's Bronchiectasis Education, Advocacy and Research Network) is a new ... more Child-BEAR-Net (Children's Bronchiectasis Education, Advocacy and Research Network) is a new European Respiratory Society (ERS)-funded Clinical Research Collaboration (CRC). Child-BEAR-Net is an international collaboration whose overall aim is to enhance the lives of children and young people (henceforth referred to as children) with bronchiectasis, including their parents, through education, advocacy and research. A newly funded ERS Clinical Research Collaboration network (Child-BEAR-Net) will create vital infrastructure to progress education, advocacy and research collaboration in paediatric bronchiectasis https://bit.ly/3ku2Xbx

Research paper thumbnail of ERS statement on standardisation of cardiopulmonary exercise testing in chronic lung diseases

European Respiratory Review, 2019

The objective of this document was to standardise published cardiopulmonary exercise testing (CPE... more The objective of this document was to standardise published cardiopulmonary exercise testing (CPET) protocols for improved interpretation in clinical settings and multicentre research projects. This document: 1) summarises the protocols and procedures used in published studies focusing on incremental CPET in chronic lung conditions; 2) presents standard incremental protocols for CPET on a stationary cycle ergometer and a treadmill; and 3) provides patients' perspectives on CPET obtained through an online survey supported by the European Lung Foundation. We systematically reviewed published studies obtained from EMBASE, Medline, Scopus, Web of Science and the Cochrane Library from inception to January 2017. Of 7914 identified studies, 595 studies with 26 523 subjects were included. The literature supports a test protocol with a resting phase lasting at least 3 min, a 3-min unloaded phase, and an 8- to 12-min incremental phase with work rate increased linearly at least every minut...

Research paper thumbnail of New understanding in the treatment of cough (NEUROCOUGH) ERS Clinical Research Collaboration: improving care and treatment for patients with cough

European Respiratory Journal, 2019

Research paper thumbnail of Challenges facing parents/caregivers of children with interstitial lung disease (chILD): international survey

Paediatric respiratory epidemiology, 2018

Introduction: The ERS Clinical Research Collaboration (CRC) for chILD aims to develop critical in... more Introduction: The ERS Clinical Research Collaboration (CRC) for chILD aims to develop critical interest in these rare diseases among early career clinicians, researchers, scientists and industry. Patient involvement is integral and through partnering with the chILD Lung Foundation, Starke Lunge Foundation and European Lung Foundation, patient/parent contacts globally are being strengthened. Methods: To understand challenges that parents face and to inform activities, a mixed methods survey was devised and made available online in 9 languages from August - November 2017. Results: 156 respondents from 13 countries responded relating to the following chILD conditions: neuroendocrine cell hyperplasia (NEHI) (39%), undiagnosed/unknown (20%), bronchiolitis obliterans (15%), surfactant dysfunction mutations (SPC, ABCA3) (13%), pulmonary interstitial glycogenosis (PIG) (6%) and other (7%). Described by one responder as ‘a very lonely disease’, the most challenging aspects were: not knowing how the disease will progress (82%), anxiety/depression (74%), time management (67%), daily activities (64%) and feeding issues (60%). On rating aspects of treatment and care, 100% wanted better clinicians working interaction; 94% to know how to get a second opinion and 80% the option for genetic counselling. More availability of online information was requested by 95%. Future research direction included causes, genetics, risk factors, diagnostics and new treatments and reduction of impact of the condition on patients over the long-term. Conclusion: These findings will inform CRC activities and research going forward. Online information, clinical networks and industry links are in active development

Research paper thumbnail of ERS statement on harmonised standards for lung cancer registration and lung cancer services in Europe

European Respiratory Journal, 2018

The European Respiratory Society (ERS) task force for harmonised standards for lung cancer regist... more The European Respiratory Society (ERS) task force for harmonised standards for lung cancer registration and lung cancer services in Europe recognised the need to create a single dataset for use in pan-European data collection and a manual of standards for European lung cancer services.The multidisciplinary task force considered evidence from two different sources, reviewing existing national and international datasets alongside the results of a survey of clinical data collection on lung cancer in 35 European countries. A similar process was followed for the manual of lung cancer services, with the task force using existing guidelines and national or international recommendations for lung cancer services to develop a manual of standards for services in Europe.The task force developed essential and minimum datasets for lung cancer registration to enable all countries to collect the same essential data and some to collect data with greater detail. The task force also developed a manual...

Research paper thumbnail of Research priorities in α1-antitrypsin deficiency: results of a patients' and healthcare providers' international survey from the EARCO Clinical Research Collaboration

ERJ Open Research, 2020

α1-antitrypsin deficiency (AATD) is a rare and under-recognised genetic condition. Owing to its l... more α1-antitrypsin deficiency (AATD) is a rare and under-recognised genetic condition. Owing to its low prevalence, international initiatives are key for conducting high-quality research in the field.From July 2018 to December 2019, the European Alpha-1 Research Collaboration (EARCO) developed and conducted two surveys, one for healthcare providers and one for patients and caregivers, aiming to identify research priorities and barriers in access to treatment for AATD.A survey on 164 research questions was electronically sent to 230 AATD experts in Europe, and 94 completed surveys from 24 countries were received. The top research areas identified by healthcare providers were causes of variable progression and poor outcomes, improvement in diagnosis, initiation and optimal dosing of augmentation therapy and effectiveness of self-management interventions. During the same period, 438 surveys were completed by patients and caregivers from 26 countries. The top research areas identified were ...

Research paper thumbnail of International consensus statement on quality standards for managing children/adolescents with bronchiectasis from the ERS CRC Child-BEAR-Net

European Respiratory Journal

Research paper thumbnail of European Respiratory Society statement for defining respiratory exacerbations in children and adolescents with bronchiectasis for clinical trials

European Respiratory Journal

Bronchiectasis is being diagnosed increasingly in children and adolescents. Recurrent respiratory... more Bronchiectasis is being diagnosed increasingly in children and adolescents. Recurrent respiratory exacerbations are common in children and adolescents with this chronic pulmonary disorder. Respiratory exacerbations are associated with an impaired quality of life, poorer long-term clinical outcomes, and substantial costs to the family and health systems. The 2021 European Respiratory Society (ERS) clinical practice guideline for the management of children and adolescents with bronchiectasis provided a definition of acute respiratory exacerbations for clinical use but to date there is no comparable universal definition for clinical research. Given the importance of exacerbations in the field, this ERS Task Force sought to obtain robust definitions of respiratory exacerbations for clinical research. The panel was a multidisciplinary team of specialists in paediatric and adult respiratory medicine, infectious disease, physiotherapy, primary care, nursing, radiology, methodology, patient...

Research paper thumbnail of The patient perspective of alpha-1 antitrypsin deficiency: disease burden and unmet needs

Breathe, 2021

Three people with alpha-1 antitrypsin deficiency (AATD) share their experiences of living with th... more Three people with alpha-1 antitrypsin deficiency (AATD) share their experiences of living with this rare, genetic condition. They reflect on living with AATD and its impact on their lives including inequality of access to treatment across Europe. https://bit.ly/3rrYS9s Receiving the AATD diagnosis, in my case when I was 40 years old, was a hard blow (figure 1). My doctor had a strong suspicion (that is why he carried out the test) but gave me no information about the disease-everything was just terrible.

Research paper thumbnail of What’s important for people with NTM? An EMBARC-ELF patient survey

European Respiratory Journal, 2020

Research paper thumbnail of The journey to bronchiectasis diagnosis and care: an EMBARC/ELF patient survey

Respiratory infections and bronchiectasis, 2021

Research paper thumbnail of Management of children and adolescents with bronchiectasis: summary of the ERS clinical practice guideline

Breathe, 2021

Bronchiectasis, characterised by chronic wet/productive cough with recurrent respiratory exacerba... more Bronchiectasis, characterised by chronic wet/productive cough with recurrent respiratory exacerbations and abnormal bronchial dilatation on computed tomography scans, remains an increasingly recognised but often neglected chronic pulmonary disorder in children and adolescents. An early diagnosis combined with optimal management offers the prospect, at least in some patients, of curing a condition previously considered irreversible. However, unlike in adults, until now no international paediatric guidelines existed. The recently published European Respiratory Society clinical practice guidelines for the management of children and adolescents with bronchiectasis attempts to address this clinical information gap. The guidelines were formulated by panel members comprised of experts from several relevant health fields, the European Lung Foundation and parents of children with bronchiectasis. Systematic reviews and the GRADE (Grading of Recommendations, Assessment, Development and Evaluat...

Research paper thumbnail of Sarcoidosis treatment and outcomes: what is most important to patients?

Sarcoidosis and other granulomatous ILD/DPLD, 2018

Purpose of Study: A sarcoidosis treatment guidleine is being developed by a European Respiratory ... more Purpose of Study: A sarcoidosis treatment guidleine is being developed by a European Respiratory Society (ERS) working group. The sarcoidosis support groups were asked to provide input regarding what outcomes were most important to the patient. Methods: Sarcoidosis support groups in collaboration with the European Lung Foundation developed an outcome survey that was translated into six languages. Individual outcomes were given overall titles and defintiions for each outcome were provided. Patients were asked to rank each outcome on a 5 point scale from 1 (extremely important) to 5 (not important at all). Results: To date, 778 individuals have completed the survey in their native langauge: 270 German, 158 English, 147 Italians. 112 Dutch, 73 Spanish, and 8 French using an online survey. The eight outcomes and scores were: quality of life (1.41); Functionality (1.43); Survival (1.69); Imaging (1.84); Adverse events (1.86); Blood tests (1.98); Pulmonary function tests (2.06). All outcomes ranged from Extremely important (1) to Very important (2). Of the outcomes, quality of life and functionality were the most important, while blood tests and pulmonary function tests were less important outcomes. Imaging was ranked as a more important outcome than blood or pulmonary function tests. Conclusion: Sarcoidosis patients ranked treatments which improved quality of life and functionality as most important outcomes. Patients ranked adverse events as a less important outcome than quality of life, survival, and functionality.

Research paper thumbnail of International consensus statement on core outcomes for clinical trials in children and adolescents with bronchiectasis – Child-BEAR-net ERS CRC

Paediatric respiratory infection and immun.

Research paper thumbnail of Quality standards for managing children and adolescents with bronchiectasis: an international consensus

Breathe

The global burden of bronchiectasis in children and adolescents is being recognised increasingly.... more The global burden of bronchiectasis in children and adolescents is being recognised increasingly. However, marked inequity exists between, and within, settings and countries for resources and standards of care afforded to children and adolescents with bronchiectasis compared with those with other chronic lung diseases. The European Respiratory Society (ERS) clinical practice guideline for the management of bronchiectasis in children and adolescents was published recently. Here we present an international consensus of quality standards of care for children and adolescents with bronchiectasis based upon this guideline.The panel used a standardised approach that included a Delphi process with 201 respondents from the parents and patients’ survey, and 299 physicians (across 54 countries) who care for children and adolescents with bronchiectasis.The seven quality standards of care statements developed by the panel address the current absence of quality standards for clinical care related...

Research paper thumbnail of Electronic cigarettes: a task force report from the European Respiratory Society

European Respiratory Journal, 2019

There is a marked increase in the development and use of electronic nicotine delivery systems or ... more There is a marked increase in the development and use of electronic nicotine delivery systems or electronic cigarettes (ECIGs). This statement covers electronic cigarettes (ECIGs), defined as “electrical devices that generate an aerosol from a liquid” and thus excludes devices that contain tobacco. Database searches identified published articles that were used to summarise the current knowledge on the epidemiology of ECIG use; their ingredients and accompanied health effects; second-hand exposure; use of ECIGs for smoking cessation; behavioural aspects of ECIGs and social impact;in vitroand animal studies; and user perspectives.ECIG aerosol contains potentially toxic chemicals. As compared to conventional cigarettes, these are fewer and generally in lower concentrations. Second-hand exposures to ECIG chemicals may represent a potential risk, especially to vulnerable populations. There is not enough scientific evidence to support ECIGs as an aid to smoking cessation due to a lack of ...

Research paper thumbnail of The European Multicentre Bronchiectasis Audit and Research Collaboration (EMBARC): experiences from a successful ERS Clinical Research Collaboration

Breathe, 2017

In contrast to airway diseases like chronic obstructive pulmonary disease or asthma, and rare dis... more In contrast to airway diseases like chronic obstructive pulmonary disease or asthma, and rare diseases such as cystic fibrosis, there has been little research and few clinical trials in bronchiectasis. Guidelines are primarily based on expert opinion and treatment is challenging because of the heterogeneous nature of the disease.In an effort to address decades of underinvestment in bronchiectasis research, education and clinical care, the European Multicentre Bronchiectasis Audit and Research Collaboration (EMBARC) was established in 2012 as a collaborative pan-European network to bring together bronchiectasis researchers. The European Respiratory Society officially funded EMBARC in 2013 as a Clinical Research Collaboration, providing support and infrastructure to allow the project to grow.EMBARC has now established an international bronchiectasis registry that is active in more than 30 countries both within and outside Europe. Beyond the registry, the network participates in design...

Research paper thumbnail of Principles of patient partnership: integrating patient perspectives into ERS Clinical Research Collaborations

Breathe

Patient and public involvement in research is increasingly considered a cornerstone of good resea... more Patient and public involvement in research is increasingly considered a cornerstone of good research practice, and the research community recognises people with lived experience as valuable stakeholders within the research process. European Respiratory Society (ERS) strongly encourages patient input into its research programme and scientific activities, working in partnership with the European Lung Foundation (ELF) to facilitate this. Based on the ERS and ELF experience and best practice in the field of patient and public involvement, we developed a set of principles to which future ERS and ELF collaborations should adhere. These principles provide guidance on how to address key challenges when planning and conducting patient and public involvement in order to develop successful partnerships with patients and drive forward patient-centred research.

Research paper thumbnail of Task Force report: European Respiratory Society guidelines for the management of children and adolescents with bronchiectasis

There is increasing awareness of bronchiectasis in children and adolescents, a chronic pulmonary ... more There is increasing awareness of bronchiectasis in children and adolescents, a chronic pulmonary disorder associated with poor quality-of-life for the child/adolescent and their parents, recurrent exacerbations and costs to the family and health systems. Optimal treatment improves clinical outcomes. Several national guidelines exist, but there are no international guidelines.The European Respiratory Society (ERS) Task Force for the management of paediatric bronchiectasis sought to identify evidence-based management (investigation and treatment) strategies. It used the ERS standardised process that included a systematic review of the literature and application of the GRADE approach to define the quality of the evidence and level of recommendations.A multidisciplinary team of specialists in paediatric and adult respiratory medicine, infectious disease, physiotherapy, primary care, nursing, radiology, immunology, methodology, patient advocacy and parents of children/adolescents with br...

Research paper thumbnail of Research priorities in alpha-1 antitrypsin deficiency (AATD) for healthcare professionals

European Respiratory Journal, 2020

Research paper thumbnail of Children's Bronchiectasis Education Advocacy and Research Network (Child-BEAR-Net): an ERS Clinical Research Collaboration on improving outcomes of children and adolescents with bronchiectasis

European Respiratory Journal, 2021

Child-BEAR-Net (Children's Bronchiectasis Education, Advocacy and Research Network) is a new ... more Child-BEAR-Net (Children's Bronchiectasis Education, Advocacy and Research Network) is a new European Respiratory Society (ERS)-funded Clinical Research Collaboration (CRC). Child-BEAR-Net is an international collaboration whose overall aim is to enhance the lives of children and young people (henceforth referred to as children) with bronchiectasis, including their parents, through education, advocacy and research. A newly funded ERS Clinical Research Collaboration network (Child-BEAR-Net) will create vital infrastructure to progress education, advocacy and research collaboration in paediatric bronchiectasis https://bit.ly/3ku2Xbx

Research paper thumbnail of ERS statement on standardisation of cardiopulmonary exercise testing in chronic lung diseases

European Respiratory Review, 2019

The objective of this document was to standardise published cardiopulmonary exercise testing (CPE... more The objective of this document was to standardise published cardiopulmonary exercise testing (CPET) protocols for improved interpretation in clinical settings and multicentre research projects. This document: 1) summarises the protocols and procedures used in published studies focusing on incremental CPET in chronic lung conditions; 2) presents standard incremental protocols for CPET on a stationary cycle ergometer and a treadmill; and 3) provides patients' perspectives on CPET obtained through an online survey supported by the European Lung Foundation. We systematically reviewed published studies obtained from EMBASE, Medline, Scopus, Web of Science and the Cochrane Library from inception to January 2017. Of 7914 identified studies, 595 studies with 26 523 subjects were included. The literature supports a test protocol with a resting phase lasting at least 3 min, a 3-min unloaded phase, and an 8- to 12-min incremental phase with work rate increased linearly at least every minut...

Research paper thumbnail of New understanding in the treatment of cough (NEUROCOUGH) ERS Clinical Research Collaboration: improving care and treatment for patients with cough

European Respiratory Journal, 2019

Research paper thumbnail of Challenges facing parents/caregivers of children with interstitial lung disease (chILD): international survey

Paediatric respiratory epidemiology, 2018

Introduction: The ERS Clinical Research Collaboration (CRC) for chILD aims to develop critical in... more Introduction: The ERS Clinical Research Collaboration (CRC) for chILD aims to develop critical interest in these rare diseases among early career clinicians, researchers, scientists and industry. Patient involvement is integral and through partnering with the chILD Lung Foundation, Starke Lunge Foundation and European Lung Foundation, patient/parent contacts globally are being strengthened. Methods: To understand challenges that parents face and to inform activities, a mixed methods survey was devised and made available online in 9 languages from August - November 2017. Results: 156 respondents from 13 countries responded relating to the following chILD conditions: neuroendocrine cell hyperplasia (NEHI) (39%), undiagnosed/unknown (20%), bronchiolitis obliterans (15%), surfactant dysfunction mutations (SPC, ABCA3) (13%), pulmonary interstitial glycogenosis (PIG) (6%) and other (7%). Described by one responder as ‘a very lonely disease’, the most challenging aspects were: not knowing how the disease will progress (82%), anxiety/depression (74%), time management (67%), daily activities (64%) and feeding issues (60%). On rating aspects of treatment and care, 100% wanted better clinicians working interaction; 94% to know how to get a second opinion and 80% the option for genetic counselling. More availability of online information was requested by 95%. Future research direction included causes, genetics, risk factors, diagnostics and new treatments and reduction of impact of the condition on patients over the long-term. Conclusion: These findings will inform CRC activities and research going forward. Online information, clinical networks and industry links are in active development

Research paper thumbnail of ERS statement on harmonised standards for lung cancer registration and lung cancer services in Europe

European Respiratory Journal, 2018

The European Respiratory Society (ERS) task force for harmonised standards for lung cancer regist... more The European Respiratory Society (ERS) task force for harmonised standards for lung cancer registration and lung cancer services in Europe recognised the need to create a single dataset for use in pan-European data collection and a manual of standards for European lung cancer services.The multidisciplinary task force considered evidence from two different sources, reviewing existing national and international datasets alongside the results of a survey of clinical data collection on lung cancer in 35 European countries. A similar process was followed for the manual of lung cancer services, with the task force using existing guidelines and national or international recommendations for lung cancer services to develop a manual of standards for services in Europe.The task force developed essential and minimum datasets for lung cancer registration to enable all countries to collect the same essential data and some to collect data with greater detail. The task force also developed a manual...

Research paper thumbnail of Research priorities in α1-antitrypsin deficiency: results of a patients' and healthcare providers' international survey from the EARCO Clinical Research Collaboration

ERJ Open Research, 2020

α1-antitrypsin deficiency (AATD) is a rare and under-recognised genetic condition. Owing to its l... more α1-antitrypsin deficiency (AATD) is a rare and under-recognised genetic condition. Owing to its low prevalence, international initiatives are key for conducting high-quality research in the field.From July 2018 to December 2019, the European Alpha-1 Research Collaboration (EARCO) developed and conducted two surveys, one for healthcare providers and one for patients and caregivers, aiming to identify research priorities and barriers in access to treatment for AATD.A survey on 164 research questions was electronically sent to 230 AATD experts in Europe, and 94 completed surveys from 24 countries were received. The top research areas identified by healthcare providers were causes of variable progression and poor outcomes, improvement in diagnosis, initiation and optimal dosing of augmentation therapy and effectiveness of self-management interventions. During the same period, 438 surveys were completed by patients and caregivers from 26 countries. The top research areas identified were ...

Research paper thumbnail of International consensus statement on quality standards for managing children/adolescents with bronchiectasis from the ERS CRC Child-BEAR-Net

European Respiratory Journal

Research paper thumbnail of European Respiratory Society statement for defining respiratory exacerbations in children and adolescents with bronchiectasis for clinical trials

European Respiratory Journal

Bronchiectasis is being diagnosed increasingly in children and adolescents. Recurrent respiratory... more Bronchiectasis is being diagnosed increasingly in children and adolescents. Recurrent respiratory exacerbations are common in children and adolescents with this chronic pulmonary disorder. Respiratory exacerbations are associated with an impaired quality of life, poorer long-term clinical outcomes, and substantial costs to the family and health systems. The 2021 European Respiratory Society (ERS) clinical practice guideline for the management of children and adolescents with bronchiectasis provided a definition of acute respiratory exacerbations for clinical use but to date there is no comparable universal definition for clinical research. Given the importance of exacerbations in the field, this ERS Task Force sought to obtain robust definitions of respiratory exacerbations for clinical research. The panel was a multidisciplinary team of specialists in paediatric and adult respiratory medicine, infectious disease, physiotherapy, primary care, nursing, radiology, methodology, patient...

Research paper thumbnail of The patient perspective of alpha-1 antitrypsin deficiency: disease burden and unmet needs

Breathe, 2021

Three people with alpha-1 antitrypsin deficiency (AATD) share their experiences of living with th... more Three people with alpha-1 antitrypsin deficiency (AATD) share their experiences of living with this rare, genetic condition. They reflect on living with AATD and its impact on their lives including inequality of access to treatment across Europe. https://bit.ly/3rrYS9s Receiving the AATD diagnosis, in my case when I was 40 years old, was a hard blow (figure 1). My doctor had a strong suspicion (that is why he carried out the test) but gave me no information about the disease-everything was just terrible.

Research paper thumbnail of What’s important for people with NTM? An EMBARC-ELF patient survey

European Respiratory Journal, 2020

Research paper thumbnail of The journey to bronchiectasis diagnosis and care: an EMBARC/ELF patient survey

Respiratory infections and bronchiectasis, 2021

Research paper thumbnail of Management of children and adolescents with bronchiectasis: summary of the ERS clinical practice guideline

Breathe, 2021

Bronchiectasis, characterised by chronic wet/productive cough with recurrent respiratory exacerba... more Bronchiectasis, characterised by chronic wet/productive cough with recurrent respiratory exacerbations and abnormal bronchial dilatation on computed tomography scans, remains an increasingly recognised but often neglected chronic pulmonary disorder in children and adolescents. An early diagnosis combined with optimal management offers the prospect, at least in some patients, of curing a condition previously considered irreversible. However, unlike in adults, until now no international paediatric guidelines existed. The recently published European Respiratory Society clinical practice guidelines for the management of children and adolescents with bronchiectasis attempts to address this clinical information gap. The guidelines were formulated by panel members comprised of experts from several relevant health fields, the European Lung Foundation and parents of children with bronchiectasis. Systematic reviews and the GRADE (Grading of Recommendations, Assessment, Development and Evaluat...

Research paper thumbnail of Sarcoidosis treatment and outcomes: what is most important to patients?

Sarcoidosis and other granulomatous ILD/DPLD, 2018

Purpose of Study: A sarcoidosis treatment guidleine is being developed by a European Respiratory ... more Purpose of Study: A sarcoidosis treatment guidleine is being developed by a European Respiratory Society (ERS) working group. The sarcoidosis support groups were asked to provide input regarding what outcomes were most important to the patient. Methods: Sarcoidosis support groups in collaboration with the European Lung Foundation developed an outcome survey that was translated into six languages. Individual outcomes were given overall titles and defintiions for each outcome were provided. Patients were asked to rank each outcome on a 5 point scale from 1 (extremely important) to 5 (not important at all). Results: To date, 778 individuals have completed the survey in their native langauge: 270 German, 158 English, 147 Italians. 112 Dutch, 73 Spanish, and 8 French using an online survey. The eight outcomes and scores were: quality of life (1.41); Functionality (1.43); Survival (1.69); Imaging (1.84); Adverse events (1.86); Blood tests (1.98); Pulmonary function tests (2.06). All outcomes ranged from Extremely important (1) to Very important (2). Of the outcomes, quality of life and functionality were the most important, while blood tests and pulmonary function tests were less important outcomes. Imaging was ranked as a more important outcome than blood or pulmonary function tests. Conclusion: Sarcoidosis patients ranked treatments which improved quality of life and functionality as most important outcomes. Patients ranked adverse events as a less important outcome than quality of life, survival, and functionality.