TY - T1的开发和内部验证临床风险评分预测事件的肾和肺肿瘤结节性硬化症患者复杂(好- 9.008)JF -神经学乔-神经学做- 10.1212 / WNL。首页0000000000201921六世- 100 - 17补充2 SP - 1421 AU -弗雷德里克Loubert AU -安德鲁家盟凯瑟琳Larochelle AU - Philippe主要盟马克Keezer Y1 - 2023/04/25 UR - //www.ez-admanager.com/content/100/17_Supplement_2/1421.abstra首页ct N2 -目的:内部开发和验证临床风险评分预测事件肾血管肌脂肪瘤(AML)和肺lymphangioleiomyomatosis (LAM)结节性硬化症(TSC)复杂的人。背景:TSC影响大约1/6000活产。儿童发病率和死亡率的主要来源是影响大脑和心脏的表现。青少年和成年人,另一方面,发展中风险最大的AML和林。设计/方法:我们将利用数据从2420年的参与者TSC联盟自然历史数据库进行这些分析。这是最大的TSC国际研究数据库。我们将使用Cox回归模型预测AML和林发展使用13的大脑,心脏和皮肤的TSC的表现潜力预测。我们将使用这些回归模型的结果发展临床风险评分。这些将在内部验证使用引导重采样、测量和校准的歧视。结果:我们获得所有必需的数据访问和分析正在进行中,到2022年底完工。 So far, descriptive analyses have shown that 426 out of 2,420 individuals in our data developed AML, while 77 developed LAM. Preliminary analyses have demonstrated excellent calibration for AML, and discrimination that is either good or strong for AML and LAM (c-statistics from 0.72 to 0.81).Conclusions: It is currently unknown whether the clinical TSC features during the childhood of an individual can accurately estimate the risk of developing AML or LAM during adolescence and adulthood. Our work will be an important step towards identifying individuals who would benefit from prophylactic treatments (e.g. with mTOR inhibitors) and lead to more tailored schedules for screening imaging. We expect that our work will allow for more personalized medicine in people with TSC.Disclosure: Mr. Loubert has nothing to disclose. Dr. House has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AstraZeneca. Dr. House has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Baxter. Dr. House has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Canadian Medical Protective Association. Dr. Larochelle has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen, Novartis, Sanofi-Genzyme, Alexion, EMD-Serono, FIND therapeutics, Actelion. The institution of Dr. Larochelle has received research support from Université de Montréal-CHUM. Dr. Major has nothing to disclose. The institution of Dr. Keezer has received research support from UCB . The institution of Dr. Keezer has received research support from Eisai. The institution of Dr. Keezer has received research support from TD Bank. The institution of Dr. Keezer has received research support from Savoy Foundation. The institution of Dr. Keezer has received research support from TSC Alliance. The institution of Dr. Keezer has received research support from Quebec Bio-imaging Network. The institution of Dr. Keezer has received research support from Canadian Institutes of Health Research. The institution of Dr. Keezer has received research support from Fonds de Recherche Québec Santé. The institution of Dr. Keezer has received research support from Canadian Institutes of Health Research. ER -