An International Survey of Patients with Ataxia: Trends in Patient-Reported Symptoms

Ataxia disorders have complex symptomology and few treatment options. Limited information is available on symptoms and quality of life with ataxia from the perspective of patients and caregivers. We aimed to assess whether trends in symptoms and quality of life varied by ataxia type and disease stage. We conducted an anonymous, international survey of 680 National Ataxia Foundation Members, including 587 people with ataxia and 93 caregivers. Data was analyzed for the whole group, by ataxia type, and by Functional Disability Stage (FDS). Respondents self-reported the first symptoms experienced, symptoms currently experienced, and the one symptom that currently has the greatest impact on their life. Impaired balance (51%, n = 343) and coordination (24%, n = 161) were the most reported first symptoms noticed by respondents across ataxia types. Impaired balance (92%, n = 627), impaired coordination (80%, n = 546), and fatigue (70%, n = 479) were the most prevalent symptoms across ataxia types and FDS. Impaired balance (65%, n = 443) was the most reported symptom as having the greatest impact on respondents' day-to-day life. While there were commonalities between FDS and ataxia types, some subtype differences emerged amongst self-reported first symptom, symptom prevalence, and symptoms impacting daily living. This study highlights trends in symptomology across ataxia types and disease stages from the perspective of people with ataxia and caregivers. While similarities exist across ataxia types, variation in which symptoms most impact quality of life across disease stages underscores the importance of selecting patient-relevant clinical trial outcome measures and endpoints. These findings can further guide clinicians in prioritizing symptom management and treatment planning, while supporting more meaningful, patient-centered discussions about disease impact.