Renal Manifestations of Tuberous Sclerosis Complex: Key Findings From the Final Analysis of the TOSCA Study Focussing Mainly on Renal Angiomyolipomas.

TOSCA mTOR registry renal angiomyolipoma tuberous sclerosis complex

Journal

Frontiers in neurology
ISSN: 1664-2295
Titre abrégé: Front Neurol
Pays: Switzerland
ID NLM: 101546899

Informations de publication

Date de publication:
2020
Historique:
received: 27 04 2020
accepted: 24 07 2020
entrez: 12 10 2020
pubmed: 13 10 2020
medline: 13 10 2020
Statut: epublish

Résumé

Renal angiomyolipomas are one of the most common renal manifestations in patients with tuberous sclerosis complex (TSC), with potentially life-threatening complications and a poor prognosis. Despite the considerable progress in understanding TSC-associated renal angiomyolipomas, there are no large scale real-world data. The aim of our present study was to describe in detail the prevalence and outcome of renal angiomyolipomas in patients with TSC, enrolled into the TuberOus SClerosis registry to increase disease Awareness (TOSCA) from 170 sites across 31 countries worldwide. We also sought to evaluate the relationship of TSC-associated renal angiomyolipomas with age, gender and genotype. The potential risk factors for renal angiomyolipoma-related bleeding and chronic kidney disease (CKD) were studied in patients who participated in the TOSCA renal angiomyolipoma substudy. Of the 2,211 eligible patients, 1,062 (48%) reported a history of renal angiomyolipomas. The median age of TSC diagnosis for the all subjects (

Identifiants

pubmed: 33041968
doi: 10.3389/fneur.2020.00972
pmc: PMC7526256
doi:

Types de publication

Journal Article

Langues

eng

Pagination

972

Informations de copyright

Copyright © 2020 Kingswood, Belousova, Benedik, Carter, Cottin, Curatolo, Dahlin, D'Amato, Beaure d'Augères, de Vries, Ferreira, Feucht, Fladrowski, Hertzberg, Jozwiak, Lawson, Macaya, Marques, Nabbout, O'Callaghan, Qin, Sander, Shah, Takahashi, Touraine, Youroukos, Zonnenberg, Jansen and Sauter.

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Auteurs

J Chris Kingswood (JC)

Cardiology Clinical Academic Group, Molecular and Clinical Sciences Research Centre, St Georges University of London, London, United Kingdom.

Elena Belousova (E)

Research and Clinical Institute of Pediatrics, Pirogov Russian National Research Medical University, Moscow, Russia.

Mirjana P Benedik (MP)

Child Neurology Department, SPS Pediatrična Klinika, Ljubljana, Slovenia.

Tom Carter (T)

TSA Tuberous Sclerosis Association, Nottingham, United Kingdom.

Vincent Cottin (V)

Hôpital Louis Pradel, Claude Bernard University Lyon 1, Lyon, France.

Paolo Curatolo (P)

Child Neurology and Psychiatry Unit, Systems Medicine Department, Tor Vergata University Hospital, Rome, Italy.

Maria Dahlin (M)

Neuropediatric Department, Astrid Lindgren Children's Hospital, Stockholm, Sweden.

Lisa D'Amato (L)

Novartis Farma S.p.A., Origgio, Italy.

Guillaume Beaure d'Augères (G)

Association Sclérose Tubéreuse de Bourneville, Gradignan, France.

Petrus J de Vries (PJ)

Division of Child and Adolescent Psychiatry, University of Cape Town, Cape Town, South Africa.

José C Ferreira (JC)

Neurology Department, Centro Hospitalar Lisboa Ocidental, Lisbon, Portugal.

Martha Feucht (M)

Medical University of Vienna, Universitätsklinik für Kinder-und Jugendheilkunde, Affiliated Partner of the ERN EpiCARE, Vienna, Austria.

Carla Fladrowski (C)

Associazione Sclerosi Tuberosa ONLUS, Milan, Italy.
European Tuberous Sclerosis Complex Association, Dattein, Germany.

Christoph Hertzberg (C)

Zentrum für Neuropädiatrie und Sozialpädiatrie, Vivantes-Klinikum Neukölln, Berlin, Germany.

Sergiusz Jozwiak (S)

Department of Child Neurology, Medical University of Warsaw, Warsaw, Poland.
Department of Neurology and Epileptology, The Children's Memorial Health Institute, Warsaw, Poland.

John A Lawson (JA)

The Tuberous Sclerosis Multidisciplinary Management Clinic, Sydney Children's Hospital, Randwick, NSW, Australia.

Alfons Macaya (A)

Pediatric Neurology Section, Hospital Universitari Vall d'Hebron, Barcelona, Spain.

Ruben Marques (R)

Novartis Farma S.p.A., Origgio, Italy.
Institute of Biomedicine (IBIOMED), University of Leon, León, Spain.

Rima Nabbout (R)

Department of Pediatric Neurology, Necker Enfants Malades Hospital, Paris Descartes University, Paris, France.

Finbar O'Callaghan (F)

Institute of Child Health, University College London, London, United Kingdom.

Jiong Qin (J)

Department of Pediatrics, Peking University People's Hospital (PKUPH), Beijing, China.

Valentin Sander (V)

Department of Neurology and Rehabilitation, Tallinn Children Hospital, Tallinn, Estonia.

Seema Shah (S)

Novartis Healthcare Pvt. Ltd., Hyderabad, India.

Yukitoshi Takahashi (Y)

National Epilepsy Center, Shizuoka Institute of Epilepsy and Neurological Disorders, Shizuoka, Japan.

Renaud Touraine (R)

Department of Genetics, CHU-Hôpital Nord, Saint-Étienne, France.

Sotiris Youroukos (S)

First Department of Paediatrics, Athens University, St. Sophia Children's Hospital, Athens, Greece.

Bernard Zonnenberg (B)

Department of Internal Medicine, University Medical Center, Utrecht, Netherlands.

Anna C Jansen (AC)

Pediatric Neurology Unit, Department of Pediatrics, UZ Brussel VUB, Brussels, Belgium.

Matthias Sauter (M)

Klinikverbund Allgäu gGmbH, Kempten, Germany.

Classifications MeSH