Objective: The aim was to identify patterns of the EQ-5D-3L in patients with SCA to describe the relationship between the patient’s diagnosis and clinical parameters.

Background: SCA types 1, 2, 3 and 6 belong to a heterogeneous group of autosomal dominantly inherited diseases with an estimated global prevalence of 3-4/100.000, defining it as a rare disease. The four SCA types differ in age of onset, time of progression and the presence of non-ataxia symptoms [1]. Missing evidence exists about patterns of patients’ HRQoL – measured with the non-diagnostic tool EQ-5D-3L – in association with these SCA disorders.

Method: We used baseline data from predominantly European SCA patients of two longitudinal cohort studies (n=628) to perform a latent class analysis to identify homogeneous clusters using the EQ-5D-3L dimensions (mobility, self-care, usual activity, anxiety/depression, pain/discomfort) as manifest variables. For each discovered latent class, we analysed sociodemographic (gender, age, age of onset), clinical (ataxia severity (SARA), Non-Ataxia Signs (INAS) and health-related variables (EQ-5D-3L index, EQVAS and PHQ-9) descriptively. We assigned the SCA diagnoses for each class, expecting no relationship between the SCA types and the health profile clusters. For that, we calculated the kappa coefficient for the agreement.

Results: A 4-class model provided the best fit to the data, named as follow: Class 1: nearly best subjective health, Class 2: moderate subjective health, Class 3: best subjective health, Class 4: worst subjective health. We could observe that the clinical severity of ataxia increases as self-reported health decreases (table 1). An association between the SCA types and the EQ-5D-3L latent health profile classes, determining a missing agreement concerning Cohen’s kappa <0, could not be found.

Conclusion: The subjective health profile is associated with the clinically measured ataxia severity. However, we could not detect a relationship between the EQ-5D classes and the SCA types. To support a holistic patient-centric view, it is essential to include the patients’ subjective status in treating these life-shortened disorders regardless of the SCA type. Further research is needed to analyze the mechanism influencing the HRQoL of SCA patients, e.g. the informal care situation.

References: 1. Jacobi H, Bauer P, Giunti P, Labrum R, Sweeney MG, Charles P, et al. The natural history of spinocerebellar ataxia type 1, 2, 3, and 6: a 2-year follow-up study. Neurology. 2011;77(11):1035-41.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/relationship-of-health-related-quality-of-life-hrqol-patterns-with-clinical-diagnosis-in-patients-with-spinocerebellar-ataxia-sca-a-latent-class-analysis/