TY  - GEN
TI  - TuberOus SClerosis registry to increAse disease awareness (TOSCA) Post-Authorisation Safety Study of Everolimus in Patients With Tuberous Sclerosis Complex
AU  - Kingswood, John Christopher
AU  - Belousova, Elena
AU  - Perkovic Benedik, Mirjana
AU  - Budde, Klemens
AU  - Carter, Tom
AU  - Cottin, Vincent
AU  - Macaya Ruíz, Alfons
PY  - 2021
PB  - Frontiers Media
LA  - eng
KW  - Esclerosi tuberosa
KW  - Registres mèdics
KW  - Medicaments - Administració
KW  - DISEASES::Neoplasms::Hamartoma::Tuberous Sclerosis
KW  - Other subheadings::Other subheadings::Other subheadings::/drug therapy
KW  - PUBLIC HEALTH::Epidemiology and Biostatistics::Biostatistics::Information Storage and Retrieval::Diseases Registries
KW  - ENFERMEDADES::neoplasias::hamartoma::esclerosis tuberosa
KW  - Otros calificadores::Otros calificadores::Otros calificadores::/farmacoterapia
KW  - SALUD PÚBLICA::epidemiología y bioestadística::bioestadística::almacenamiento y recuperación de la información::registros de enfermedades
AB  - TOSCA; Everolimus; Estudi de seguretat post-autorització ; TOSCA; Everolimus; Estudio de seguridad posterior a la autorización ; TOSCA; Everolimus; Post-authorization safety study ; This non-interventional post-authorisation safety study (PASS) assessed the long-term safety of everolimus in patients with tuberous sclerosis complex (TSC) who participated in the TuberOus SClerosis registry to increase disease Awareness (TOSCA) clinical study and received everolimus for the licensed indications in the European Union. The rate of adverse events (AEs), AEs that led to dose adjustments or treatment discontinuation, AEs of potential clinical interest, treatment-related AEs (TRAEs), serious AEs (SAEs), and deaths were documented. One hundred seventy-nine patients were included in the first 5 years of observation; 118 of 179 patients had an AE of any grade, with the most common AEs being stomatitis (7.8%) and headache (7.3%). AEs caused dose adjustments in 56 patients (31.3%) and treatment discontinuation in nine patients (5%). AEs appeared to be more frequent and severe in children. On Tanner staging, all patients displayed signs of age-appropriate sexual maturation. Twenty-two of 106 female (20.8%) patients had menstrual cycle disorders. The most frequent TRAEs were stomatitis (6.7%) and aphthous mouth ulcer (5.6%). SAEs were reported in 54 patients (30.2%); the most frequent SAE was pneumonia (>3% patients; grade 2, 1.1%, and grade 3, 2.8%). Three deaths were reported, all in patients who had discontinued everolimus for more than 28 days, and none were thought to be related to everolimus according to the treating physicians. The PASS sub-study reflects the safety and tolerability of everolimus in the management of TSC in real-world routine clinical practice.
UR  - http://hdl.handle.net/11351/5982
DO  - 10.3389/fneur.2021.630378
UR  - https://www.pollux-fid.de/r/base-ftscientia:oai:scientiasalut.gencat.cat:11351/5982
H1  - Pollux (Fachinformationsdienst Politikwissenschaft)
ER  -