TuberOus SClerosis registry to increAse disease awareness (TOSCA) Post-Authorisation Safety Study of Everolimus in Patients With Tuberous Sclerosis Complex.
TuberOus SClerosis registry to increAse disease awareness (TOSCA) Post-Authorisation Safety Study of Everolimus in Patients With Tuberous Sclerosis Complex.
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DOI：
10.3389/fneur.2021.630378
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发表时间：
2021
影响因子：
3.4
通讯作者：
TOSCA Consortium and TOSCA Investigators
中科院分区：
文献类型：
--
作者：
Kingswood JC;Belousova E;Benedik MP;Budde K;Carter T;Cottin V;Curatolo P;Dahlin M;D'Amato L;d'Augères GB;de Vries PJ;Ferreira JC;Feucht M;Fladrowski C;Hertzberg C;Jozwiak S;Lawson JA;Macaya A;Marques R;Nabbout R;O'Callaghan F;Qin J;Sander V;Sauter M;Shah S;Takahashi Y;Touraine R;Youroukos S;Zonnenberg B;Jansen AC;TOSCA Consortium and TOSCA Investigators
本项非干预性许可后安全性研究（PASS）评估了依维莫司在结节性硬化症（TSC）患者中的长期安全性，这些患者参加了Toscus Sclerosis Registry to Increase Disease Awareness（TOSCA）临床研究，并接受依维莫司治疗欧盟许可的适应症。记录不良事件（AE）、导致剂量调整或治疗中止的AE、潜在临床关注的AE、治疗相关AE（TRAE）、严重AE（SAE）和死亡的发生率。在前5年的观察中纳入了179例患者; 179例患者中有118例发生了任何级别的AE，最常见的AE是口腔炎（7.8%）和头痛（7.3%）。AE导致56例患者（31.3%）剂量调整和9例患者（5%）治疗中止。儿童中的AE似乎更频繁和严重。在坦纳分期中，所有患者均表现出与年龄相适应的性成熟迹象。106例女性患者中有22例（20.8%）有月经周期紊乱。最常见的TRAE是口腔炎（6.7%）和口疮性口腔溃疡（5.6%）。54例患者（30.2%）报告了SAE;最常见的SAE是肺炎（>3%的患者; 2级，1.1%和3级，2.8%）。报告了3例死亡，均发生在停用依维莫司超过28天的患者中，治疗医生认为均与依维莫司无关。PASS子研究反映了依维莫司在现实世界常规临床实践中管理TSC的安全性和耐受性。
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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影响因子：
158.5
作者：
Krueger, Darcy A.;Care, Marguerite M.;Franz, David Neal
通讯作者：
Franz, David Neal
影响因子：
168.9
作者：
Bissler, John J.;Kingswood, J. Christopher;Budde, Klemens
通讯作者：
Budde, Klemens
影响因子：
36.4
作者：
Curatolo, Paolo;Franz, David N.;French, Jacqueline A.
通讯作者：
French, Jacqueline A.
影响因子：
3.1
作者：
Kotulska, Katarzyna;Chmielewski, Dariusz;Jozwiak, Sergiusz
通讯作者：
Jozwiak, Sergiusz
影响因子：
168.9
作者：
Franz, David Neal;Belousova, Elena;Jozwiak, Sergiusz
通讯作者：
Jozwiak, Sergiusz