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| Frontiers in neurology. 2021 Mar 23. doi: 10.3389/fneur.2021.630378. pmc: PMC8021912 |
| TuberOus SClerosis registry to increAse disease awareness (TOSCA) Post-Authorisation Safety Study of Everolimus in Patients With Tuberous Sclerosis Complex. |
| Kingswood JC1, Belousova E2, Benedik MP3, Budde K4, Carter T5, Cottin V6, Curatolo P7, Dahlin M8, D'Amato L9, d'Augères GB10, de Vries PJ11, Ferreira JC12, Feucht M13, Fladrowski C14, Hertzberg C15, Jozwiak S16, Lawson JA17, Macaya A18, Marques R19, Nabbout R20, O'Callaghan F21, Qin J22, Sander V23, Sauter M24, Shah S25, Takahashi Y26, Touraine R27, Youroukos S28, Zonnenberg B29, Jansen AC30 |
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Abstract 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. |
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Copyright © 2021 Kingswood, Belousova, Benedik, Budde, Carter, Cottin, Curatolo, Dahlin, D'Amato, d'Augères, de Vries, Ferreira, Feucht, Fladrowski, Hertzberg, Jozwiak, Lawson, Macaya, Marques, Nabbout, O'Callaghan, Qin, Sander, Sauter, Shah, Takahashi, Touraine, Youroukos, Zonnenberg, Jansen and TOSCA Consortium and TOSCA Investigators. |
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KEYWORDS: TOSCA, everolimus, mammalian target of rapamycin, post-authorization safety study, tuberous sclerosis complex |
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Publikations ID: 33833726 Quelle: öffnen |
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