Natural history of patients with tuberous sclerosis complex related renal angiomyolipoma. (3rd July 2017)

• Record Type: Journal Article
• Authors: Song, Xue; Liu, Zhimei; Cappell, Katherine; Gregory, Christopher; Said, Qayyim; Prestifilippo, Judith; Charles, Hearns; Hulbert, John; Bissler, John
• Is Part Of: Current medical research and opinion. Volume 33:Number 7(2017)
• Page Start: 1277
• Page End: 1282
• Publication Date: 2017-07-03
• Subjects: Angiomyolipoma -- natural history of disease -- observational study -- tuberous sclerosis; Clinical medicine -- Periodicals; Therapeutics -- Periodicals; 615.5
• DOI: 10.1080/03007995.2017.1313726
• Languages: English
• ISSNs: 0300-7995

Abstract

Abstract: 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. 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. Results: 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. Conclusions: 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.