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dc.contributorVall d'Hebron Barcelona Hospital Campus
dc.contributor.authorMarques, Ruben
dc.contributor.authorBelousova, Elena
dc.contributor.authorPerkovic Benedik, Mirjana
dc.contributor.authorCarter, Tom
dc.contributor.authorCottin, Vincent
dc.contributor.authorCuratolo, Paolo
dc.contributor.authorMacaya Ruíz, Alfons
dc.date.accessioned2020-09-16T07:57:11Z
dc.date.available2020-09-16T07:57:11Z
dc.date.issued2019-10-25
dc.identifier.citationMarques R, Belousova E, Benedik MP, Carter T, Cottin V, Curatolo P, et al. Treatment patterns and use of resources in patients with tuberous sclerosis complex: insights from the TOSCA registry. Front Neurol. 2019 Oct 25;10:1144.
dc.identifier.issn1664-2295
dc.identifier.urihttp://hdl.handle.net/11351/5263
dc.descriptionTSC; Resource use; TOSCA
dc.description.abstractTuberous Sclerosis Complex (TSC) is a rare autosomal-dominant disorder caused by mutations in the TSC1 or TSC2 genes. Patients with TSC may suffer from a wide range of clinical manifestations; however, the burden of TSC and its impact on healthcare resources needed for its management remain unknown. Besides, the use of resources might vary across countries depending on the country-specific clinical practice. The aim of this paper is to describe the use of TSC-related resources and treatment paterns within the TOSCA registry. A total of 2,214 patients with TSC from 31 countries were enrolled and had a follow-up of up to 5 years. A search was conducted to identify the variables containing both medical and non-medical resource use information within TOSCA. This search was performed both at the level of the core project as well as at the level of the research projects on epilepsy, subependymal giant cell astrocytoma (SEGA), lymphangioleiomyomatosis (LAM), and renal angiomyolipoma (rAML) taking into account the timepoints of the study, age groups, and countries. Data from the quality of life (QoL) research project were analyzed by type of visit and age at enrollment. Treatments varied greatly depending on the clinical manifestation, timepoint in the study, and age groups. GAB Aergics were the most prescribed drugs for epilepsy, and mTOR inhibitors are dramatically replacing surgery in patients with SEGA, despite corrent recommendations proposing both treatment options.mTOR inhibitors are also becoming common treatments in rAML and LAMpatients. Forty-two out of the 143 patients (29.4%) who participated in the QoL research project reported inpatient stays over the last year. Data from non-medical resource use showed the critical impact of TSC on job status and capacity. Disability allowances were more common in children than adults (51.1% vs 38.2%). Psychological counseling, social services and social worker services were needed by <15% of the patients, regardless of age. The long-term nature, together with the variability in its clinical manifestations, makes TSC a complex and resourcedemanding disease. The present study shows a comprehensive picture of the resource use implications of TSC.
dc.language.isoeng
dc.publisherFrontiers Media
dc.relation.ispartofseriesFrontiers in Neurology;10
dc.rightsAttribution 4.0 International
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/
dc.sourceScientia
dc.subjectEsclerosi tuberosa
dc.subjectServeis sanitaris - Administració
dc.subjectBases de dades - Disseny
dc.subject.meshTuberous Sclerosis
dc.subject.meshHealth Resources
dc.subject.meshRegistries
dc.titleTreatment patterns and use of resources in patients with tuberous sclerosis complex: insights from the TOSCA registry
dc.typeinfo:eu-repo/semantics/article
dc.identifier.doi10.3389/fneur.2019.01144
dc.subject.decsesclerosis tuberosa
dc.subject.decsrecursos en salud
dc.subject.decsregistros
dc.relation.publishversionhttps://www.frontiersin.org/articles/10.3389/fneur.2019.01144/full
dc.type.versioninfo:eu-repo/semantics/publishedVersion
dc.audienceProfessionals
dc.contributor.authoraffiliation[Marques R] Novartis Farma SpA, Origgio, Italy. Institute of Biomedicine (IBIOMED), University of Leon, León, Spain. [Belousova E] Research and Clinical Institute of Pediatrics, Pirogov Russian National Research Medical University, Moscow, Russia. [Benedik MP] SPS Pediatrična Klinika, Ljubljana, Slovenia. [Carter T] TSA Tuberous Sclerosis Association, Nottingham, United Kingdom. [Cottin V] Hôpital Louis Pradel, Claude Bernard University Lyon 1, Lyon, France. [Curatolo P] Tor Vergata University Hospital, Rome, Italy. [Macaya A] Servei de Pediatria General i Especialitats, Vall d'Hebron Hospital Universitari, Barcelona, Spain
dc.identifier.pmid31708865
dc.identifier.wos000494442700001
dc.rights.accessrightsinfo:eu-repo/semantics/openAccess


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