Patient-perceived progression in multiple system atrophy: natural history of quality of life

Tiphaine Saulnier; Margherita Fabbri; Mélanie Le Goff; Catherine Helmer; Anne Pavy-Le Traon; Wassilios G. Meissner; Olivier Rascol; Cécile Proust-Lima; Alexandra Foubert-Samier

arXiv · 2309.13089

Patient-perceived progression in multiple system atrophy: natural history of quality of life

Tiphaine Saulnier·Margherita Fabbri·Mélanie Le Goff·Catherine Helmer·Anne Pavy-Le Traon·Wassilios G. Meissner·Olivier Rascol·Cécile Proust-Lima·Alexandra Foubert-Samier

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Abstract

Health-related quality of life (Hr-QoL) scales provide crucial information on neurodegenerative disease progression, help improving patient care, and constitute a meaningful endpoint for therapeutic research. However, Hr-QoL progression is usually poorly documented, as for multiple system atrophy (MSA), a rare and rapidly progressing alpha-synucleinopathy. This work aimed to describe Hr-QoL progression during the natural course of MSA, explore disparities between patients, and identify informative items using a four-step statistical strategy.We leveraged the data of the French MSA cohort comprising annual assessments with the MSA-QoL questionnaire for more than 500 patients over up to 11 years. The four-step strategy (1) determined the subdimensions of Hr-QoL in MSA; (2) modelled the subdimension trajectories over time, accounting for the risk of death; (3) mapped the sequence of item impairments with disease stages; and (4) identified the most informative items specific to each disease stage.Among the 536 patients included, 50% were women and they were aged on average 65.1 years old at entry. Among them, 63.1% died during the follow-up. Four dimensions were identified. In addition to the original motor, nonmotor, and emotional domains, an oropharyngeal component was highlighted. While the motor and oropharyngeal domains deteriorated rapidly, the nonmotor and emotional aspects were already slightly to moderately impaired at cohort entry and deteriorated slowly over the course of the disease. Impairments were associated with sex, diagnosis subtype, and delay since symptom onset. Except for the emotional domain, each dimension was driven by key identified items.Hr-QoL is a multidimensional concept that deteriorates progressively over the course of MSA and brings essential knowledge for improving patient care. As exemplified with MSA, the thorough description of Hr-QoL using the 4-step original analysis can provide new perspectives on neurodegenerative diseases' management to ultimately deliver better support focused on the patient's perspective.

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Tiphaine Saulnier, Margherita Fabbri, Mélanie Le Goff, Catherine Helmer, Anne Pavy-Le Traon, Wassilios G. Meissner, Olivier Rascol, Cécile Proust-Lima, Alexandra Foubert-Samier. 2023-09-22. Patient-perceived progression in multiple system atrophy: natural history of quality of life. https://arxiv.org/abs/2309.13089

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