NATURAL HISTORY OF PATIENTS WITH TUBEROUS SCLEROSIS COMPLEX RELATED RENAL ANGIOMYOLIPOMA
Author(s)
Cappell KA1, Song X2, Liu Z3, Eynullayeva E1, Gregory C1, Prestifilippo J3, Charles H4, Hulbert J5, Bissler JJ6
1Truven Health Analytics, Ann Arbor, MI, USA, 2Truven Health Analytics, Cambridge, MA, USA, 3Novartis, East Hanover, NJ, USA, 4NYU Radiology Associates, New York, NY, USA, 5Urologic Physicians, Edina, MN, USA, 6St. Jude Pediatric Research Hospital, Memphis, TN, USA
OBJECTIVES: This retrospective study examined the temporal relationship of diagnosis, treatment, healthcare utilization, and clinical outcomes in patients with Tuberous Sclerosis (TSC)-related renal angiomyolipoma in the US. METHODS: Patients with ≥1 diagnosis of TSC (ICD-9-CM 759.5) and angiomyolipoma (ICD-9-CM 223.0 and other codes) were extracted from the MarketScan® Commercial DatabaseRESULTS: The final sample consisted of 605 patients (mean age 26.8): 225 patients age<18 (mean age 9.8, 55% female, median follow-up 3.4 years) and 380 patients age≥18 (mean age 36.9, 73% female, median follow-up 2.2 years). In patients <18, 87% had TSC diagnosis before angiomyolipoma (angiomyolipoma was diagnosed 25.7 months after TSC on average), 2.8% had complete nephrectomy (8.5 months after index date); emergency room (ER) visit, 61% (16.6 months); inpatient admission, 48% (17.0 months); chronic kidney disease (CKD), 12% (32.3 months); embolization, 11% (36.2 months); end stage renal disease (ESRD), 1.3% (40.9 months); and 1.4% partial nephrectomy (42.0 months). In patients ≥18, 56% had TSC diagnosis before angiomyolipoma (angiomyolipoma was diagnosed 16.9 months after TSC), ER visit, 56% (9.9 months); inpatient admission, 50% (11.3 months); partial nephrectomy, 7.9% (11.9 months); complete nephrectomy, 11.4% (13.9 months); CKD, 23% (15.1 months); embolization, 19% (17.1 months); and ESRD, 5.3% (20.1 months). CONCLUSIONS: This study suggested that time from a TSC diagnosis to an angiomyolipoma diagnosis was long and sometimes was preceded with ER visits, hospitalizations and surgeries. The innovative graphing of natural history can help raise disease awareness in TSC.
Conference/Value in Health Info
2014-05, ISPOR 2014, Palais des Congres de Montreal
Value in Health, Vol. 17, No. 3 (May 2014)
Code
PND2
Topic
Epidemiology & Public Health
Topic Subcategory
Disease Classification & Coding
Disease
Rare and Orphan Diseases, Urinary/Kidney Disorders