Burden of renal angiomyolipomas associated with tuberous sclerosis complex: results of a patient and caregiver survey.

Background: Tuberous sclerosis complex (TSC) is a rare genetic disorder characterized by benign tumors in multiple organs, including non-cancerous kidney lesions known as renal angiomyolipomas. This study's objective is to describe the age-stratified morbidity, treatment patterns, and health-related quality of life of TSC patients with renal angiomyolipomas in the United States. A cross-sectional, anonymous web-based survey was conducted with a convenience sample of TSC patients and caregivers identified through a patient advocacy organization.

Management of a Rare Disease Population: A Model for Identifying a Patient Population With Tuberous Sclerosis Complex.

Tuberous sclerosis complex (TSC) is a rare genetic disorder affecting the brain and other vital organs with varying symptoms and severity among patients. This study developed and validated a risk model to identify patients with TSC using large databases of medical and pharmacy claims.

Natural history of patients with tuberous sclerosis complex related renal angiomyolipoma.

Objective: To examine temporal relationships between tuberous sclerosis complex (TSC) and renal angiomyolipoma diagnosis and outcomes, treatment, and healthcare utilization.

Methods: Administrative data from the MarketScan Commercial Database was used to select TSC-related renal angiomyolipoma patients during 1 January 2000-31 March 2013. Patients were followed until the earliest of inpatient death or end of enrollment or study.

Patterns of Disease Monitoring and Treatment Among Patients With Tuberous Sclerosis Complex-related Angiomyolipomas.

Objective: To use the tuberous sclerosis complex (TSC) Natural History Database to describe monitoring and treatment patterns among patients with TSC-related angiomyolipomas (AMLs).

Methods: This study used the TSC Natural History Database, which contains demographics, affected areas, diagnosis, and treatments for more than 1300 patients with TSC enrolled in 16 participating clinics during 2006-2013. Patient characteristics, AML monitoring tests, and AML treatments were assessed.

Healthcare utilization and costs in patients with tuberous sclerosiscomplex-related renal angiomyolipoma.

Objective: To quantify healthcare utilization and costs in patients with tuberous sclerosis complex (TSC) and renal angiomyolipoma (AML) in a matched cohort of patients without TSC or AML.

Methods: Administrative data from the MarketScan Research Databases were used to select patients with TSC and renal AML during January 1, 2000-March 31, 2013 from the Commercial database and January 1, 2000-June 30, 2012 from the Medicaid database. Patients were required to have at least 30 days of follow-up from initiation into the study, and were followed until inpatient death, end of insurance coverage, or the end of study.

Long-term Clinical Morbidity in Patients With Renal Angiomyolipoma Associated With Tuberous Sclerosis Complex.

Objective: To estimate the incidence rates of kidney-related clinical outcomes among patients with tuberous sclerosis complex (TSC)-related angiomyolipoma (AML) compared to an age-matched control cohort in the United States.

Materials And Methods: This was a retrospective, observational study. Administrative data from the MarketScan Research Databases were used to select patients with TSC and renal AML.

Rates of interventional procedures in patients with tuberous sclerosis complex-related renal angiomyolipoma.

Objective: To describe rates of renal artery embolization, partial nephrectomy, and complete nephrectomy in patients with tuberous sclerosis complex (TSC) and renal angiomyolipoma.

Methods: Data from the MarketScan® Research Databases were used to select patients with TSC and renal angiomyolipoma during January 1, 2000-March 31,2013 (Commercial database) and January 1, 2000-June 30, 2012 (Medicaid database). Patients had at least 30 days of follow-up and were followed until the earliest of inpatient death, end of enrollment, or end of study.

Caring for Children With Tuberous Sclerosis Complex: What Is the Physical and Mental Health Impact on Caregivers?

Disease burden associated with tuberous sclerosis complex, a genetic disorder characterized by benign tumor growth including lesions in multiple organs, puts tremendous demands on families. This analysis examines the physical and mental health burden of tuberous sclerosis complex caregivers in the United States. An institutional review board-approved web-based survey of tuberous sclerosis complex caregivers collected information; descriptive analyses were conducted on age-based subgroups.

Tuberous sclerosis complex: a survey of health care resource use and health burden.

Purpose: Tuberous sclerosis complex (TSC) is a multiorgan, autosomal-dominant genetic disorder with incomplete penetrance.

Methods: This analysis of a web-based survey focuses on the clinical presentation, management, and associated burden of patients with TSC in the United States.

Results: A total of 676 TSC patients or caregivers responded.

The burden of subependymal giant cell astrocytomas associated with tuberous sclerosis complex: results of a patient and caregiver survey.

Tuberous sclerosis complex is a genetic disorder characterized by benign tumor growth including lesions in the ventricular system of the brain known as subependymal giant cell astrocytomas. This analysis focuses on the clinical presentation, management, and associated burden of subependymal giant cell astrocytomas in patients with tuberous sclerosis complex in the United States. An institutional review board-approved web-based survey of tuberous sclerosis complex patients and caregivers collected information, and descriptive analyses were conducted on age-based subgroups.