Development and Validation of a Patient-Reported Questionnaire of Ataxia Progression - Ataxia UK

Development and Validation of a Patient-Reported Questionnaire of Ataxia Progression

Post Published: June 23, 2021

In the clinic, assessment of cerebellar ataxias has been largely confined to clinical rating scales (e.g. Scale for Assessment and Rating of Ataxia -SARA-), gait laboratories, imaging of the brain, and collection of biosamples. However, all these measures do not take into consideration the patients’ experience of the condition or its change over time. Patient reports of their daily activities and symptoms are critical for optimizing care and medicine regulatory agencies are very interested in the inclusion of patient-reported measures in clinical trials.

Dr. Jeremy Schmahmann and his colleagues from Harvard Medical School in the United States have developed a new patient-reported questionnaire for ataxias in collaboration with Biohaven Pharmaceuticals. The National Ataxia Foundation helped these researchers to get in touch with hundreds of people with different ataxias in the US (e.g. SCA 1, 2, 3, 5, 6, 7, and 8, dentatorubral pallidoluysian atrophy, Friedreich’s ataxia, ataxia oculomotor apraxia, episodic ataxia) to participate in the design of this questionnaire and learn from patients about the evolution of their condition.

After gathering all the feedback from patients, carers, and experts in the field Dr. Schmahmann and colleagues developed and validated the Patient-Reported Outcome Measure of Ataxia (PROM-Ataxia). The PROM-Ataxia is a 70-item questionnaire emerging from patient experience. PROM-Ataxia includes questions about gait, lower and upper extremity control, manual dexterity, visual/ocular motor control; patient experience of dysphagia, bowel and bladder function, sleep, fatigue, vertigo, neuropathy, ability to manage household chores and employment responsibilities, driving, libido and self-care; neuropsychiatric domains of mood, anxiety, motivation, social interactions and cognitive tasks such as multitasking, learning, comprehension, and verbal skills.

This new patient-reported questionnaire has the potential to include the patients’ perspective in clinical care and quantify the efficacy of medicines in clinical trials.

To view the research article please visit:


Article published on June 21st, 2021

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