Longitudinal study of a cohort of MSA-C patients in South Italy: survival and clinical features

Lieto, Maria; Roca, Alessandro; Bruzzese, Dario; Antenora, Antonella; Alfieri, Girolamo; Saccà, Francesco; Bellofatto, Marta; Bilo, Leonilda; Barbato, Stefano; Michele, Giuseppe De; Filla, Alessandro

doi:10.1007/s10072-019-03948-7

Cited by 14 publications

(7 citation statements)

References 19 publications

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“…68 UMSARS-II correlated with ICARS in patients with MSA (r = 0.93) 65 or SCA3 (r = 0.96). 43 Higher UMSARS scores were significant predictors of loss of independent walking and wheelchair use in patients with MSA-C. 69 Responsiveness statistics are shown in Supporting Information Table E-1 in Appendix S1, indicating that the scale can adequately record changes in MSA.…”

Section: Unified Multiple System Atrophy Rating Scalementioning

confidence: 99%

Assessment of Ataxia Rating Scales and Cerebellar Functional Tests: Critique and Recommendations

Pérez-Lloret

Warrenburg

Rossi³

et al. 2020

Movement Disorders

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A BS TRACT: Background: We assessed the clinimetric properties of ataxia rating scales and functional tests, and made recommendations regarding their use. Methods: A systematic literature search was conducted to identify the instruments used to rate ataxia symptoms. The identified rating scales and functional ability tests were reviewed and ranked by the panel as "recommended," "suggested," or "listed" for the assessment of patients with discrete cerebellar disorders, using previously established criteria. Results: We reviewed 14 instruments (9 rating scales and 5 functional tests).

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Section: Unified Multiple System Atrophy Rating Scalementioning

confidence: 99%

Assessment of Ataxia Rating Scales and Cerebellar Functional Tests: Critique and Recommendations

Pérez-Lloret

Warrenburg

Rossi³

et al. 2020

Movement Disorders

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“…Multiple System Atrophy is a rare atypical parkinsonian disorder in which affected individuals deteriorate quickly [26][27][28]. MSA is pathologically characterized both by the accumulation of ␣-syn within the oligodendrocytes and by an elevation in brain iron levels.…”

Section: Discussionmentioning

confidence: 99%

The Compound ATH434 Prevents Alpha-Synuclein Toxicity in a Murine Model of Multiple System Atrophy

Finkelstein

Shukla

Cherny

et al. 2022

JPD

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Background: An elevation in iron levels, together with an accumulation of α-synuclein within the oligodendrocytes, are features of the rare atypical parkinsonian disorder, Multiple System Atrophy (MSA). We have previously tested the novel compound ATH434 (formally called PBT434) in preclinical models of Parkinson’s disease and shown that it is brain-penetrant, reduces iron accumulation and iron mediated redox activity, provides neuroprotection, inhibits alpha synuclein aggregation and lowers the tissue levels of alpha synuclein. The compound was also well-tolerated in a first-in-human oral dosing study in healthy and older volunteers with a favorable, dose-dependent pharmacokinetic profile. Objective: To evaluate the efficacy of ATH434 in a mouse MSA model. Methods: The PLP-α-syn transgenic mouse overexpresses α-synuclein, demonstrates oligodendroglial pathology, and manifests motor and non-motor aspects of MSA. Animals were provided ATH434 (3, 10, or 30 mg/kg/day spiked into their food) or control food for 4 months starting at 12 months of age and were culled at 16 months. Western blot was used to assess oligomeric and urea soluble α-synuclein levels in brain homogenates, whilst stereology was used to quantitate the number of nigral neurons and glial cell inclusions (GCIs) present in the substantia nigra pars compacta. Results: ATH434 reduced oligomeric and urea soluble α-synuclein aggregation, reduced the number of GCIs, and preserved SNpc neurons. In vitro experiments suggest that ATH434 prevents the formation of toxic oligomeric species of synuclein. Conclusion: ATH434 is a promising small molecule drug candidate that has potential to move forward to trial for treating MSA.

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“…In this study, the swallowing difficulties were only diagnosed by questioning the patients, but it showed that survival time was shorter if dysphagia was present (RR = 2.56) without giving details on the degree of severity [ 24 ]. But another retrospective study [ 30 ] (also cited in the 2021 literature review [ 11 ]) only including patients with MSA-C ( n = 66), stated that dysphagia (prevalence of 78%) was not an independent predictive factor for mortality, but that no other clinical sign was either.…”

Section: Discussionmentioning

confidence: 99%

Pharyngolaryngeal semiology and prognostic factors in multiple system atrophy

Fassi

Gallois

Crestani

et al. 2022

Eur Arch Otorhinolaryngol

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Introduction Multiple system atrophy (MSA) is a rare degenerative neurological disorder in adults. It induces parkinsonian and/or cerebellar syndrome associated with dysautonomia. Pharyngolaryngeal symptoms are common. Our aim is to describe the Pharyngolaryngeal semiology on one hand, and to ascertain whether the presence of these symptoms represents a prognostic factor for MSA on the other. Methods Thus, we carried out a retrospective, single-centre study, on a cohort receiving care at the centre of reference for MSA. The patients were referred for otorhinolaryngology assessment. The data was collected over the year 2020 with the help of computer software from the university hospital centre (UHC). Firstly, we described the Pharyngolaryngeal semiology specific to MSA by questioning patients, and by the results of nasofibroscopic examinations and swallowing tests. We then used multivariate analysis of variance to describe the prognostic factors of MSA progression (in UMSARS I and II points per month of progression) and survival (number of years between the first symptoms and death). Results This study included a hundred and one patients and made it possible to define a Pharyngolaryngeal semiology profile of MSA, which is: a reduction in laryngeal mobility (primarily vocal cord abduction defects), abnormal movements (particularly at rest or when initiating a movement) and a defect in the protection mechanisms of the upper airways. The swallowing difficulties are moderate and the main mechanisms are delayed pharyngeal swallow and/or an oro-pharyngeal transport defect. In the multivariate analyses, the contributing factors are laryngeal anomalies, modification of solid food to fluid food and nutritional complication. Conclusion ENT specialists should pay close attention to problems in the Pharyngolaryngeal dynamic and then consider a neurological cause. They can also itemize the clinical factors that could have a negative effect on the prognosis of the patient with MSA. Indeed, early detection makes it possible to provide care for respiratory and nutritional complications.

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Longitudinal study of a cohort of MSA-C patients in South Italy: survival and clinical features

Cited by 14 publications

References 19 publications

Assessment of Ataxia Rating Scales and Cerebellar Functional Tests: Critique and Recommendations

Assessment of Ataxia Rating Scales and Cerebellar Functional Tests: Critique and Recommendations

The Compound ATH434 Prevents Alpha-Synuclein Toxicity in a Murine Model of Multiple System Atrophy

Pharyngolaryngeal semiology and prognostic factors in multiple system atrophy

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