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000284358 1001_ $$aSilva, Patrick$$b0
000284358 245__ $$aThe Medication Patterns of Spinocerebellar Ataxia Type 3 Mutation Carriers Enrolled in the ESMI Cohort.
000284358 260__ $$aBerlin [u.a.]$$bSpringer$$c2026
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000284358 520__ $$aSpinocerebellar ataxia type 3 (SCA3) is one of the most common dominantly inherited ataxias worldwide. Despite research advances, no approved disease-modifying treatment exists, and management focuses on symptom alleviation and functional capacity maximization. Symptomatic treatment guidelines are scarce, leaving decisions to physicians' discretion. The lack of studies on SCA3 symptom management hinders therapy standardization. The aim of this study was to investigate medication-usage patterns among SCA3 mutation carriers and controls included in the multicentric European Spinocerebellar Ataxia Type-3/Machado-Joseph Disease Initiative (ESMI) cohort.We conducted a retrospective cross-sectional analysis of the medication taken by ESMI participants enrolled in the study between 2016 and 2023. Medication being used at the most recent follow-up visit available was categorized according to the Anatomical Therapeutic Chemical system. Comparisons between groups were performed using nonparametric tests for continuous variables and Fisher's exact test for categorical variables. In addition, a retrospective longitudinal analysis was conducted to study the impact of medication subclasses on disease progression, using linear mixed-effects models adjusted for relevant covariates.A total of 474 participants were included, comprising 344 SCA3 mutation carriers and 130 controls. Compared with controls, SCA3 subjects took more vitamins, mineral supplements, muscle relaxants, and medications targeting the nervous system. Psychoanaleptics and vitamins were introduced early in the disease course, whereas most other subclasses were initiated in mid-to-late stages, coinciding with the onset of neurological symptoms. Substantial disparities in medication usage were observed across the study centers. None of the medication subclasses commonly used by patients with SCA3 showed a significant impact on disease progression.This is the first study to explore medication usage patterns in SCA3 mutation carriers. Our study provides a comprehensive overview of the medications administered in SCA3 and underscores the importance of collaborative efforts toward achieving standardized clinical practices in the management of this disease.
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000284358 650_7 $$0EC 3.4.19.12$$2NLM Chemicals$$aAtaxin-3
000284358 650_2 $$2MeSH$$aHumans
000284358 650_2 $$2MeSH$$aMachado-Joseph Disease: drug therapy
000284358 650_2 $$2MeSH$$aMachado-Joseph Disease: genetics
000284358 650_2 $$2MeSH$$aMale
000284358 650_2 $$2MeSH$$aFemale
000284358 650_2 $$2MeSH$$aCross-Sectional Studies
000284358 650_2 $$2MeSH$$aRetrospective Studies
000284358 650_2 $$2MeSH$$aMiddle Aged
000284358 650_2 $$2MeSH$$aMutation
000284358 650_2 $$2MeSH$$aAdult
000284358 650_2 $$2MeSH$$aCohort Studies
000284358 650_2 $$2MeSH$$aDisease Progression
000284358 650_2 $$2MeSH$$aHeterozygote
000284358 650_2 $$2MeSH$$aLongitudinal Studies
000284358 650_2 $$2MeSH$$aAged
000284358 650_2 $$2MeSH$$aAtaxin-3: genetics
000284358 650_2 $$2MeSH$$aEurope
000284358 7001_ $$aCosta, Marina A$$b1
000284358 7001_ $$aGaspar, Laetitia$$b2
000284358 7001_ $$aDurães, João$$b3
000284358 7001_ $$aCunha, Inês$$b4
000284358 7001_ $$aRibeiro, Joana A$$b5
000284358 7001_ $$aJanuário, Cristina$$b6
000284358 7001_ $$aOliveiros, Bárbara$$b7
000284358 7001_ $$aHübener-Schmid, Jeannette$$b8
000284358 7001_ $$0P:(DE-2719)2811327$$aFaber, Jennifer$$b9$$udzne
000284358 7001_ $$aRaposo, Mafalda$$b10
000284358 7001_ $$aLima, Manuela$$b11
000284358 7001_ $$aGarcia-Moreno, Hector$$b12
000284358 7001_ $$aGiunti, Paola$$b13
000284358 7001_ $$aBeichert, Lukas$$b14
000284358 7001_ $$0P:(DE-2719)2810795$$aSchöls, Ludger$$b15$$udzne
000284358 7001_ $$avan de Warrenburg, Bart P$$b16
000284358 7001_ $$ade Vries, Jeroen$$b17
000284358 7001_ $$aThieme, Andreas$$b18
000284358 7001_ $$aReetz, Kathrin$$b19
000284358 7001_ $$aJacobi, Heike$$b20
000284358 7001_ $$aInfante, Jon$$b21
000284358 7001_ $$0P:(DE-2719)2810314$$aKlockgether, Thomas$$b22$$udzne
000284358 7001_ $$aGroup, ESMI Study$$b23$$eCollaboration Author
000284358 7001_ $$ade Almeida, Luís Pereira$$b24
000284358 7001_ $$00000-0003-4076-7516$$aSantana, Magda M$$b25
000284358 7001_ $$aFerreira, Ana$$b26$$eContributor
000284358 7001_ $$aRosa, Ana$$b27$$eContributor
000284358 7001_ $$aGonzalez, Carlos$$b28$$eContributor
000284358 7001_ $$aGonzalez-Robles, Cristina$$b29$$eContributor
000284358 7001_ $$aTimmann, Dagmar$$b30$$eContributor
000284358 7001_ $$aErdlenbruch, Friedrich$$b31$$eContributor
000284358 7001_ $$aLemos, João$$b32$$eContributor
000284358 7001_ $$aVasconcelos, João$$b33$$eContributor
000284358 7001_ $$aTeves, Luís$$b34$$eContributor
000284358 7001_ $$aPires, Paula$$b35$$eContributor
000284358 7001_ $$aLopes, Pedro$$b36$$eContributor
000284358 7001_ $$aCoelho, Pedro$$b37$$eContributor
000284358 7001_ $$aKay, Teresa$$b38$$eContributor
000284358 773__ $$0PERI:(DE-600)2043806-0$$a10.1007/s40263-025-01237-w$$gVol. 40, no. 2, p. 233 - 246$$n2$$p233 - 246$$tCNS drugs$$v40$$x1172-7047$$y2026
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