Development of respiratory care guidelines for Duchenne muscular dystrophy in the UK: key recommendations for clinical practice

Significant inconsistencies in respiratory care provision for Duchenne muscular dystrophy (DMD) are reported across different specialist neuromuscular centres in the UK. The absence of robust clinical evidence and expert consensus is a barrier to the implementation of care recommendations in public healthcare systems as is the need to increase awareness of key aspects of care for those living with DMD. Here, we provide evidenced-based and/or consensus-based best practice for the respiratory care of children and adults living with DMD in the UK, both as part of routine care and in an emergency. Methodology Initiated by an expert working group of UK-based respiratory physicians (including British Thoracic Society (BTS) representatives), neuromuscular clinicians, physiotherapist and patient representatives, draft guidelines were created based on published evidence, current practice and expert opinion. After wider consultation with UK respiratory teams and neuromuscular services, consensus was achieved on these best practice recommendations for respiratory care in DMD. Result The resulting recommendations are presented in the form of a flow chart for assessment and monitoring, with additional guidance and a separate chart setting out key considerations for emergency management. The recommendations have been endorsed by the BTS. Conclusions These guidelines provide practical, reasoned recommendations for all those managing day-to-day and acute respiratory care in children and adults with DMD. The hope is that this will support patients and healthcare professionals in accessing high standards of care across the UK

Highlights in asthma 2005

It is usual at the end of a year for top tens to feature large in our collective consciousness. These inevitably include the occasional controversial selection and without fail will overlook a number of gems, whether in the field of literature, art, or science. The approaches to such compilations include personal selections, convening expert committees through to letting the market decide. However, it is well recognised that experts can be wrong and markets distorted. A novel approach to identifying the key publications in the field of medicine, Faculty of 1000 Medicine (http://www.f1000medicine.com), uses faculty evaluations to assign ratings to published papers within topics and categories, bringing a flavour of the dynamism of post hoc peer review to biological sciences that exists in other fields of scholarship. Archives of Disease in Childhood has also developed a market led system of peer evaluation through its web based top ten most read articles feature

Current barriers to treatment for wet age-related macular degeneration (wAMD): findings from the wAMD patient and caregiver survey

Monica Varano,1 Nicole Eter,2 Steve Winyard,3 Kim U Wittrup-Jensen,4 Rafael Navarro,5 Julie Heraghty6On behalf of the wAMD Patient and Caregiver Survey Committee members1Department of Ophthalmology, Fondazione GB Bietti-IRCCS, Rome, Italy; 2Department of Ophthalmology, University of Münster, Münster, Germany; 3Department of Policy and Campaigns, Royal National Institute of Blind People, London, UK; 4Bayer Pharma AG, Berlin, Germany; 5Instituto de Microcirugía Ocular, Barcelona, Spain; 6Macular Disease Foundation Australia, Sydney, NSW, AustraliaPurpose: A cross-sectional survey to evaluate the current management of wet age-related macular degeneration (wAMD) and to identify barriers to treatment from a patient and caregiver perspective.Methods: An ophthalmologist-devised questionnaire was given to a global cohort of patients who were receiving (or had previously received) antivascular endothelial growth factor injections and to caregivers (paid and unpaid) to evaluate the impact of wAMD on their lives.Results: Responders included 910 patients and 890 caregivers; wAMD was diagnosed in both eyes in 45% of patients, and 64% had been receiving injections for >1 year. Many caregivers were a child/grandchild (47%) or partner (23%) of the patient; only 7% were professional caregivers. Most (73%) patients visited a health care professional within 1 month of experiencing vision changes and 54% began treatment immediately. Most patients and caregivers reported a number of obstacles in managing wAMD, including the treatment itself (35% and 39%, respectively). Sixteen percent of patients also missed a clinic visit.Conclusion: Most patients seek medical assistance promptly for a change in vision; however, about a quarter of them do not. This highlights a lack of awareness surrounding eye health and the impact of a delayed diagnosis. Most patients and caregivers identified a number of obstacles in managing wAMD.Keywords: antivascular endothelial growth factor agents, patient-reported outcomes, wAMD patient and caregiver survey, wet age-related macular degeneratio

The emotional and physical impact of wet age-related macular degeneration: findings from the wAMD Patient and Caregiver Survey

Monica Varano,1 Nicole Eter,2 Steve Winyard,3 Kim U Wittrup-Jensen,4 Rafael Navarro,5 Julie Heraghty6 On behalf of the wAMD Patient and Caregiver Survey Committee members 1Department of Ophthalmology, Fondazione GB Bietti-IRCCS, Rome, Italy; 2Department of Ophthalmology, University of Münster, Münster, Germany; 3Department of Policy and Campaigns, Royal National Institute of Blind People, London, UK; 4Bayer Pharma AG, Berlin, Germany; 5Instituto de Microcirugia Ocular, Barcelona, Spain; 6Macular Disease Foundation Australia, Sydney, NSW, Australia Objectives: This was a cross-sectional survey to evaluate the physical and emotional impact of wet age-related macular degeneration (wAMD) on a global cohort of patients who were receiving (or had previously received) antivascular endothelial growth factor injections, and caregivers (paid and unpaid).Methods: The survey was performed in nine countries using an ophthalmologist-devised questionnaire.Results: A total of 910 patients and 890 caregivers completed the questionnaire. Most patients had been diagnosed and receiving antivascular endothelial growth factor injections for more than 1 year (74.7% and 63.8%, respectively), and many patients (82.1%) received support from a caregiver (usually a child/grandchild [47.3%] or partner [23.3%]). wAMD had a negative impact on most patients (71.6%); many rated fear (44.9%), sadness (39.9%), frustration (37.3%), and depression (34.0%) as common. It was linked to physical consequences, such as difficulty in reading (61.1%). Many effects were significantly greater in patients with a longer duration of disease or with wAMD in both eyes. Some caregivers (unpaid) also reported that caregiving had a negative impact on them (31.1%); many reported emotions such as sadness (34.9%) and depression (24.4%), but many also felt useful (48.4%). Overall, 27.2% of caregivers (unpaid) rated caregiving as inconvenient; this was linked to days of employment/personal obligations missed.Conclusion: wAMD has a significant negative impact on the lives of patients, including vision-related depression, poor mobility, and limitations in day-to-day activities. The impact on nonprofessional caregivers may be underestimated in terms of emotional impact (such as depression) and loss of productivity. Keywords: wet age-related macular degeneration, anti-vascular endothelial growth factor agents, wAMD Patient and Caregiver Survey, patient-reported outcome