What was this study about?
People with Spinocerebellar Ataxia Type 3 (SCA3) experience problems with balance, walking, coordination, and speech that gradually worsen over time. Doctors usually measure these symptoms during clinic visits using a test called the Scale for the Assessment and Rating of Ataxia (SARA). However, a single clinic visit only shows how someone is doing at one moment and may miss the day-to-day ups and downs in their symptoms.
In 2021, Ataxia UK co-funded a research project with the National Ataxia Foundation (NAF) led by Dr Marcus Grobe-Einsler at the German Center for Neurodegenerative Diseases. They wanted to find out whether people with SCA3 could reliably measure their symptoms at home using SARAhome, a smartphone/tablet-based system where patients record short videos of themselves performing simple movement tasks. The results of this work have recently been published in the scientific journal Annals of Clinical and Translational Neurology.
What did the researchers do?
· They recruited 65 people with genetically confirmed SCA3 from seven medical centres across Europe (the UK, Germany, the Netherlands and Portugal). The London Ataxia Centre was one of the study sites.
· Participants were taught how to use the app and then recorded themselves twice a day for 14 days at home.
· Before each recording, participants also reported whether they felt their symptoms were better, worse, or unchanged.
· A smaller group of 11 participants repeated the process about one year later so the researchers could see how well the system detected progression of their SCA3 symptoms over time.
What did they find?
The study found that using SARAhome was practical and well accepted.
· Participants completed about 80% of the requested recordings, which is considered good for this type of home-monitoring study.
· Nearly 80% of all recordings were of sufficient quality to calculate a complete SARAhome score.
The researchers also found that:
· Ataxia symptoms naturally fluctuate from day to day.
· The difference between a person’s best and worst scores over two weeks was typically about 3 points.
· These changes were generally consistent with how participants said they felt. For example, people often reported feeling worse when they were tired or exhausted and better when they felt well rested.
Another important finding was that:
· Four days of recordings were enough to provide a reliable picture of a person’s typical level of ataxia.
· Recording for longer than four days gave only modest additional benefit while participant adherence gradually declined.
Did SARAhome detect progression of SCA3?
SARAhome was able to detect progression of SCA3 symptoms. In the small group followed for about a year, the average SARAhome score over four days detected a clearer change and was more sensitive to disease progression than the clinic-based SARA assessment. This suggests that averaging several home measurements may give a more accurate picture than relying on a single clinic visit.
Why is this important?
This study suggests that home video assessments could improve both patient care and clinical trials by:
· reducing the need for frequent travel to specialist clinics,
· capturing day-to-day changes in symptoms,
· giving researchers a more reliable measure of SCA severity,
· making it easier to detect whether new treatments are working.
This could be especially valuable as new therapies for SCA3 are being developed.
What are the limitations of the research?
The study also had some limitations:
· Only 11 participants were followed for the long-term comparison.
· People with more advanced SCA3, or significant cognitive problems, were underrepresented.
· Participants needed access to reliable internet and enough space at home to complete the walking tests, so the approach may not work for everyone.
Conclusions
This study provides strong evidence that SARAhome is a practical and reliable way for people with SCA3 to assess their symptoms at home. Recording short videos over four days appears to give a better picture of SCA3 severity than a single clinic visit and may help future clinical trials measure treatment effects more accurately. Larger long-term studies are still needed to confirm these findings, before this approach can be used more widely.
