title: Natural history of patients with tuberous sclerosis complex related renal angiomyolipoma.
abstract: To examine temporal relationships between tuberous sclerosis complex (TSC) and renal angiomyolipoma diagnosis and outcomes, treatment, and healthcare utilization.
  
  
  
  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. Occurrence of kidney-related outcomes, kidney-related procedures, and all-cause healthcare utilization and time to occurrence were reported. Kaplan-Meier curves were used to display the unadjusted distribution of time to outcome.
  
  
  
  A total of 605 patients were selected (<18 years N = 225; ≥18 years N = 380). Mean time from TSC to renal angiomyolipoma diagnosis was 25.7 months in younger and 16.9 months in older patients. Patients ≥18 years had higher rates of chronic kidney disease (CKD), hematuria, kidney failure, embolization (EMB), and partial and complete nephrectomy compared to patients <18 years (all p < .05). Mean time from TSC-related renal angiomyolipoma diagnosis to CKD, hematuria, kidney failure, EMB, first emergency room and inpatient visits was shorter in older compared to younger patients (all p < .05). Probability of developing CKD was approximately 0.8 and 0.95 within 3 years in younger and older patients, respectively.
  
  
  
  Patients with TSC-related renal angiomyolipoma had high rates of kidney-related outcomes and procedures. These events sometimes preceded the angiomyolipoma diagnosis. A key study limitation was that due to the small sample size, results may have been biased by outliers. Research is needed to determine whether earlier angiomyolipoma diagnosis can impact occurrence of events and reduce healthcare utilization.
authors.affiliation: a Truven Health Analytics Inc., an IBM Company , Cambridge , MA , USA.
authors.forename: Xue
authors.initials: X
authors.lastname: Song
authors.affiliation: b Novartis Pharmaceuticals Corporation , East Hanover , USA.
authors.forename: Zhimei
authors.initials: Z
authors.lastname: Liu
authors.affiliation: a Truven Health Analytics Inc., an IBM Company , Cambridge , MA , USA.
authors.forename: Katherine
authors.initials: K
authors.lastname: Cappell
authors.affiliation: a Truven Health Analytics Inc., an IBM Company , Cambridge , MA , USA.
authors.forename: Christopher
authors.initials: C
authors.lastname: Gregory
authors.affiliation: b Novartis Pharmaceuticals Corporation , East Hanover , USA.
authors.forename: Qayyim
authors.initials: Q
authors.lastname: Said
authors.affiliation: b Novartis Pharmaceuticals Corporation , East Hanover , USA.
authors.forename: Judith
authors.initials: J
authors.lastname: Prestifilippo
authors.affiliation: c South Florida Vascular Associates , Coconut Creek , FL , USA.
authors.forename: Hearns
authors.initials: H
authors.lastname: Charles
authors.affiliation: d Urologic Physicians , Edina , MN , USA.
authors.forename: John
authors.initials: J
authors.lastname: Hulbert
authors.affiliation: e LeBonheur Children's Hospital and St. Jude Children's Research Hospital , Memphis , TN , USA.
authors.forename: John
authors.initials: J
authors.lastname: Bissler
mesh_terms: D000293:Adolescent; D000328:Adult; D018207:Angiomyolipoma / Q000209:etiology*; D002648:Child; D004621:Embolization, Therapeutic / Q000379:methods; D005260:Female; D006801:Humans; D007668:Kidney / Q000502:physiology; D007680:Kidney Neoplasms / Q000209:etiology* / Q000628:therapy; D008297:Male; D008875:Middle Aged; D009392:Nephrectomy / Q000379:methods*; D051436:Renal Insufficiency, Chronic / Q000453:epidemiology; D012189:Retrospective Studies; D014402:Tuberous Sclerosis / Q000150:complications*; D055815:Young Adult
keywords: Angiomyolipoma; natural history of disease; observational study; tuberous sclerosis