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@ARTICLE{Wilkens:282298,
author = {Wilkens, Ida and Bebermeier, Sarah and Heine, Johanne and
Ruf, Viktoria Constanze and Compta, Yaroslau and Molina
Porcel, Laura and Troakes, Claire and Vamanu, Albert and
Downes, Sophia and Irwin, David John and Cohen, Jesse and
Lee, Edward B and Nilsson, Christer F and Englund, Elisabet
M and Nemati, Mojtaba and Katzdobler, Sabrina and Levin,
Johannes and Bernhardt, Alexander Maximilian and Pantelyat,
Alexander and Seemiller, Joseph and Berger, Stephen and Van
Swieten, John C and Dopper, Elise G P and Rozemuller,
Annemieke J M and Kovacs, Gabor G and Bendahan, Nathaniel
and Lang, Anthony E and Herms, Jochen and Höglinger,
Günter U and Hopfner, Franziska},
title = {{M}ultiple {S}ystem {A}trophy {W}ithout {D}ysautonomia:
{A}n {A}utopsy-{C}onfirmed {S}tudy.},
journal = {Neurology},
volume = {105},
number = {11},
issn = {0028-3878},
address = {Philadelphia, Pa.},
publisher = {Wolters Kluwer},
reportid = {DZNE-2025-01268},
pages = {e214316},
year = {2025},
abstract = {Multiple system atrophy (MSA) is a neurodegenerative
disorder characterized by 3 core symptom complexes:
parkinsonism, cerebellar syndrome, and dysautonomia. Recent
Movement Disorder Society (MDS) criteria allow for the
clinical diagnosis of MSA based solely on motor symptoms,
without requiring dysautonomia. This study aimed to evaluate
the frequency and disease trajectory of MSA patients without
dysautonomia compared with those with autonomic
involvement.A multicenter cohort of autopsy-confirmed
patients with MSA was analyzed for demographic
characteristics, symptom onset, and progression of
parkinsonism, cerebellar syndrome, and dysautonomia.
Clinical data were collected through standardized chart
reviews across participating centers and categorized using
the MDS-MSA criteria. Patients were grouped according to
their initial symptom complex and tracked for the evolution
of additional symptoms. Analyses included time to
development of further symptom complexes, age at symptom
onset, disease duration, and phenotype at the last recorded
visit. Patients with motor symptoms only were matched to
patients with similar demographics but with dysautonomia.
Statistical methods included ANOVA, t tests, Welch t tests,
and χ2 tests, with significance set at p < 0.05.Among 140
patients (mean age at onset 62.3 ± 8.9 years; $44\%$
female), 81 $(58\%)$ initially presented without
dysautonomia (57 with parkinsonism only, 17 with cerebellar
syndrome only, 7 with both). At final follow-up, 12 patients
$(9\%)$ had not developed dysautonomia. These patients
showed significantly longer disease duration (mean 8.1 ±
2.1 years) than matched patients with dysautonomia (mean 6.3
± 2.6 years; p = 0.035). Overall, $51\%$ of patients
developed all 3 symptom complexes. Patients with cerebellar
onset progressed more rapidly to multisystem involvement
than those with parkinsonian onset (mean interval to second
symptom: 2.0 vs 3.4 years; p < 0.05).The MDS-MSA criteria
expand the diagnostic scope by identifying a motor-only
subgroup with a distinct and potentially slower disease
course. These findings underscore the importance of
including motor-only patients in natural history and
interventional studies. Limitations include retrospective
data collection and potential variability in symptom
documentation.},
keywords = {Humans / Multiple System Atrophy: pathology / Multiple
System Atrophy: diagnosis / Multiple System Atrophy:
physiopathology / Female / Male / Middle Aged / Aged /
Autopsy / Primary Dysautonomias / Disease Progression /
Cohort Studies / Parkinsonian Disorders},
cin = {Clinical Research (Munich) / AG Levin / AG Herms},
ddc = {610},
cid = {I:(DE-2719)1111015 / I:(DE-2719)1111016 /
I:(DE-2719)1110001},
pnm = {353 - Clinical and Health Care Research (POF4-353) / 352 -
Disease Mechanisms (POF4-352)},
pid = {G:(DE-HGF)POF4-353 / G:(DE-HGF)POF4-352},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:41232058},
pmc = {pmc:PMC12615010},
doi = {10.1212/WNL.0000000000214316},
url = {https://pub.dzne.de/record/282298},
}