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Catherine Brun-Strang, Genevieve Bonnelye, Majorie Leclerc, Ronald BUGGAGE; Stargardt Disease: burden from a patient association perspective.. Invest. Ophthalmol. Vis. Sci. 2015;56(7 ):2142.
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Stargardt disease (SD) is a rare, inherited macular dystrophy with no treatment available. It most commonly occurs in children and young adults and leads to a progressive central visual loss which can result in blindness. Few data from a patient perspective are presently available. The objective of this study is to better understand the burden of SD on affected patients.
We conducted 18 in-depth 60-minutes interviews (IDIs) among representatives from patient associations (RPAs) focusing on SD disease and having SD patients, patients’ family or relatives as members in 18 countries. IDIs moderation was based on a semi-directive guide. All IDIs were audio-recorded and the data collected were analyzed descriptively following a common analysis grid.
Preliminary data, based on 10 IDIs conducted in France, Germany, Italy, Spain, Argentina, Brazil, Chile, Colombia and Mexico showed that most patient associations are not specifically dedicated to SD, but cover more broadly conditions causing visual impairment. Their main goal is to help patients cope with their ocular conditions. RPAs state that SD can affect all daily life activities. Hence, associations offer support to help patients remain autonomous including referral to rehabilitations centers. RPAs estimate that 30% to 100% of their members with SD are employed or at school. The degenerative nature of SD and the lack of treatments also impact on patients and their families. Therefore associations provide psychological support, counseling on medical results and medical orientation to prevent patients from isolating themselves. They also inform patients about ongoing research and on treatments in development. While the cost of SD was perceived to be a burden to patients, the magnitude of the economic burden was difficult to estimate by RPAs.
From the patient association perspective, the main areas of impact of SD on patients are adapting to daily-life and maintaining autonomy. The support offered by patient associations, beyond that addressed routinely in clinic practice, can help to relieve the burden of SD. As important allies in the global management of visually impaired persons participation in patient associations should be recommended for all patients with inherited retinal diseases such as SD.
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