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- W4377013410 abstract "Abstract Background Adrenoleukodystrophy (ALD) is a multifaceted, x-linked, neurodegenerative disorder that affect patients through a variety of physical, emotional, social, and other disease-specific factors that collectively contribute to disease burden. Adrenomyeloneuropathy (AMN) is one of the most frequent clinical manifestations and a subset of ALD. To facilitate clinical care and research, it is important to identify which symptoms are most common and relevant to individuals with ALD and AMN. Methods We conducted semi-structured qualitative interviews and an international cross-sectional study to determine the most prevalent and important symptoms of ALD and AMN. Our studies included adult participants with a diagnosis of ALD and AMN who were recruited from national and international patient registries. Responses were categorized by age, sex, functional status, and other demographic and clinical features. Results Seventeen individuals with ALD and AMN participated in qualitative interviews, providing 1,709 direct quotes regarding their symptomatic burden. One hundred and nine individuals participated in the cross sectional survey study, which inquired about 182 unique symptoms representing 24 distinct symptomatic themes. The symptomatic themes with the highest prevalence in the ALD and AMN sample cohort were problems with balance (90.9%), limitations with mobility or walking (87.3%), fatigue (86.4%), and leg weakness (86.4%). The symptomatic themes with the highest impact scores (on a 0–4 scale with 4 being the most severe) were trouble getting around (2.35), leg weakness (2.25), and problems with balance (2.21). A higher prevalence of symptomatic themes was associated with functional ability, employment disruption, and speech status. Conclusions There are many patient-relevant symptoms and themes that contribute to disease burden in individuals with ALD and AMN. These symptoms, identified by those diagnosed with ALD and AMN, present key targets for further research and therapeutic development." @default.
- W4377013410 created "2023-05-19" @default.
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- W4377013410 date "2023-05-18" @default.
- W4377013410 modified "2023-09-29" @default.
- W4377013410 title "Patient-Reported Impact of Symptoms in Adrenoleukodystrophy (PRISM-ALD)" @default.
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- W4377013410 doi "https://doi.org/10.21203/rs.3.rs-2856459/v1" @default.
- W4377013410 hasPublicationYear "2023" @default.
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