ArticleOrphanet journal of rare diseases2018
The progression rate of spinocerebellar ataxia type 2 changes with stage of disease.
Article in Orphanet journal of rare diseases, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 1 of them a synthesis that pooled it.
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Who cites it
13 citing papers in PubMed, 1 synthesis or guideline pooled it, 31 citations in OpenAlex.
- Natural history of most common spinocerebellar ataxia: a systematic review and meta-analysis.Journal of neurology · 2021Pooled it
- Improving the Identification of the Preclinical Stages of Spinocerebellar Ataxia Type 2.Tomography (Ann Arbor, Mich.) · 2026Article
- Progression of Gait Ataxia in Spinocerebellar Ataxia Type 2: Clinical Utility of Digitally Measured Gait Metrics.Cerebellum (London, England) · 2025Article
- Localization of Potential Energy in Hydrogen Bonds of theInternational journal of molecular sciences · 2025Article
- On the Cut-Off Value of the Anteroposterior Diameter of the Midbrain Atrophy in Spinocerebellar Ataxia Type 2 Patients.Brain sciences · 2024Article
- The S-Factor, a New Measure of Disease Severity in Spinocerebellar Ataxia: Findings and Implications.Cerebellum (London, England) · 2023Article
- Temporal Dynamics of the Scale for the Assessment and Rating of Ataxia in Spinocerebellar Ataxias.Movement disorders : official journal of the Movement Disorder Society · 2023Article
- Differential Temporal Dynamics of Axial and Appendicular Ataxia in SCA3.Movement disorders : official journal of the Movement Disorder Society · 2022Article
- The progression rate of spinocerebellar ataxia type 3 varies with disease stage.Journal of translational medicine · 2022Article
- Longitudinal Assessment Using Optical Coherence Tomography in Patients with Friedreich's Ataxia.Tomography (Ann Arbor, Mich.) · 2021Article
- Quantitative assessment of postural instability in spinocerebellar ataxia type 3 patients.Annals of clinical and translational neurology · 2020Article
- Progression of Cerebellar Atrophy in Spinocerebellar Ataxia Type 2 Gene Carriers: A Longitudinal MRI Study in Preclinical and Early Disease Stages.Frontiers in neurology · 2020Article
- Information and Diagnosis Networks - tools to improve diagnosis and treatment for patients with rare genetic diseases.Genetics and molecular biology · 2019Article
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15 authors at 3 institutions in 1 country.
Funding
Abstract
backgroundSpinocerebellar ataxia type 2 (SCA2) affects several neurological structures, giving rise to multiple symptoms. However, only the natural history of ataxia is well known, as measured during the study duration. We aimed to describe the progression rate of ataxia, by the Scale for the Assessment and Rating of Ataxia (SARA), as well as the progression rate of the overall neurological picture, by the Neurological Examination Score for Spinocerebellar Ataxias (NESSCA), and not only during the study duration but also in a disease duration model. Comparisons between these models might allow us to explore whether progression is linear during the disease duration in SCA2; and to look for potential modifiers.
resultsEighty-eight evaluations were prospectively done on 49 symptomatic subjects; on average (SD), study duration and disease duration models covered 13 (2.16) months and 14 (6.66) years of individuals' life, respectively. SARA progressed 1.75 (CI 95%: 0.92-2.57) versus 0.79 (95% CI 0.45 to 1.14) points/year in the study duration and disease duration models. NESSCA progressed 1.45 (CI 95%: 0.74-2.16) versus 0.41 (95% CI 0.24 to 0.59) points/year in the same models. In order to explain these discrepancies, the progression rates of the study duration model were plotted against disease duration. Then an acceleration was detected after 10 years of disease duration: SARA scores progressed 0.35 before and 2.45 points/year after this deadline (p = 0.013). Age at onset, mutation severity, and presence of amyotrophy, parkinsonism, dystonic manifestations and cognitive decline at baseline did not influence the rate of disease progression.
conclusionsNESSCA and SARA progression rates were not constant during disease duration in SCA2: early phases of disease were associated with slower progressions. Modelling of future clinical trials on SCA2 should take this phenomenon into account, since disease duration might impact on inclusion criteria, sample size, and study duration. Our database is available online and accessible to future studies aimed to compare the present data with other cohorts.
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