patterns of disease monitoring and treatment among patients with tuberous sclerosis complex related angiomyolipomas

Accepted Manuscript Title: Patterns of Disease Monitoring and Treatment among Patients with Tuberous Sclerosis Complex-Related Angiomyolipomas Author: Elyse Swallow, Sarah King, Jinlin Song, Miranda Peeples, James E Signorovitch, Zhimei Liu, Judith Prestifilippo, Michael Frost, Michael Kohrman, Bruce Korf, Darcy Krueger, Steven Sparagana PII: DOI: Reference: S0090-4295(17)30180-2 http://dx.doi.org/doi: 10.1016/j.urology.2017.02.036 URL 20317 To appear in: Urology Received date: Accepted date: 29-11-2016 20-2-2017 Please cite this article as: Elyse Swallow, Sarah King, Jinlin Song, Miranda Peeples, James E Signorovitch, Zhimei Liu, Judith Prestifilippo, Michael Frost, Michael Kohrman, Bruce Korf, Darcy Krueger, Steven Sparagana, Patterns of Disease Monitoring and Treatment among Patients with Tuberous Sclerosis Complex-Related Angiomyolipomas, Urology (2017), http://dx.doi.org/doi: 10.1016/j.urology.2017.02.036 This is a PDF file of an unedited manuscript that has been accepted for publication As a service to our customers we are providing this early version of the manuscript The manuscript will undergo copyediting, typesetting, and review of the resulting proof before it is published in its final form Please note that during the production process errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal pertain Patterns of disease monitoring and treatment among patients with tuberous sclerosis complex-related angiomyolipomas Elyse Swallow, MPP1; Sarah King, BA1; Jinlin Song, PhD1; Miranda Peeples, BA1; James E Signorovitch, PhD1; Zhimei Liu, PhD2; Judith Prestifilippo, MD2; Michael Frost, MD3; Michael Kohrman, MD4; Bruce Korf, MD, PhD5; Darcy Krueger, MD6; Steven Sparagana, MD7 Analysis Group, Inc.; Novartis Pharmaceuticals Corporation; Minnesota Epilepsy Group; University of Chicago; University of Alabama at Birmingham; Cincinnati Children's Hospital Medical Center; Texas Scottish Rite Hospital for Children Corresponding author Elyse Swallow Analysis Group, Inc 111 Huntington Avenue #14 Boston, MA 02199 Email: elyse.swallow@analysisgroup.com Phone: 617-425-8483 Fax: 617-425-8001 Keywords Tuberous sclerosis complex; angiomyolipoma; monitor; treatment; surgery; mTOR inhibitor CONFIDENTIAL PAGE Page of 20 Source of Funding This work was funded by Novartis Author Contributions All authors participated in the design of the study and contributed to the manuscript development Statistical analyses were conducted by ES, SK, JS, MP, and JES and were interpreted in collaboration with ZL, JP, MF, MK, BK, DK, and SS All the authors vouch for the accuracy and completeness of the data reported and the adherence of the study to the protocol, and all the authors made the decision to submit the manuscript for publication Disclosures: ZL and JP are employees of Novartis and own stock/stock options MF, MK, BK, DK, and SS are independent consultants who received consultancy fees from Novartis ES, JS, MP, and JES are employees of Analysis Group Inc., which has received consultancy fees from Novartis for the analysis described in this manuscript; SK was an employee of Analysis Group Inc at the time of the study Abstract OBJECTIVE: To use the tuberous sclerosis complex (TSC) Natural History Database to describe monitoring and treatment patterns among patients with TSC-related angiomyolipomas (AMLs) MATERIALS AND METHODS: This study used the TSC Natural History Database, which contains demographics, affected areas, diagnosis, and treatments for over 1,300 TSC patients enrolled in 16 participating clinics during 2006-2013 Patient characteristics, AML monitoring tests, and AML treatments were assessed RESULTS: Among the 621 patients with TSC-related AMLs, 54% were female; 77% were Caucasian Median age at TSC diagnosis was 18 years (Table 1) A majority of patients (72%) were diagnosed with angiomyolipoma before age 18 Approximately 50% of patients were 19 years of age or older as of their most recently recorded clinic encounter More than half of the patients (54%) were female The majority were white (77%); 13% were Hispanic, 7% were African-American, 3% were Asian,