TREATMENT PATTERNS IN TUBEROUS SCLEROSIS COMPLEX (TSC) PATIENTS WITH RENAL ANGIOMYOLIPOMA AND SUBEPENDYMAL GIANT CELL ASTROCYTOMA (SEGA)

Author(s)

Song X1, Said Q2, Tran OV3, Prestifilippo J2, Bissler J4, Krueger DA5
1Truven Health Analytics, Shrewsbury, MA, USA, 2Novartis Pharmaceutical Corporation, East Hanover, NJ, USA, 3Truven Health Analytics, Cambridge, MA, USA, 4The University of Tennessee Health Science Center, Memphis, TN, USA, 5Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA

OBJECTIVES:  TSC is a rare multi-organ disease, often with neurologic and renal complications. This study examined commercial and Medicaid insurance treatment patterns (everolimus, sirolimus, and surgeries) in TSC patients with renal angiomyolipoma or SEGA in the US. METHODS:  Patients with ≥1 diagnosis of TSC and with renal angiomyolipoma or SEGA were extracted from the MarketScan® Commercial (1/1/2009-8/31/2016) and Medicaid (1/1/2009-6/30/2015) databases. Patients were followed from index date (earliest TSC, renal angiomyolipoma or SEGA diagnosis) until inpatient death or end of data. The proportion of patients treated, types of initial treatment and subsequent treatments were examined during the variable follow-up. RESULTS:  The final sample included 1,497 TSC patients (n=896 renal angiomyolipoma only, n=411 SEGA only, and n=190 with both). Compared with commercial patients (n=984), Medicaid patients (n=513) had the same mean age (22 years), a higher proportion of males (50.3% vs. 42.9%), and a longer follow-up period (48 vs. 38 months). Treatment rates were similar between commercial and Medicaid (58.0% vs. 61.7%, p=0.182), but it took significantly more days for Medicaid patients to initiate treatment (377 vs. 516 days, p<0.001). In the commercial cohort, among the 46.4% patients starting treatment with surgery, 33.7% had repeat surgeries and 16.2% switched to drug treatment; among the 11.6% patients starting treatment with a drug, only 4.4% had subsequent surgeries. In the Medicaid cohort, among 48.0% who started treatment with surgery, 57.8% had repeat surgeries and 10.2% switched to drug treatment; among the 14.4% patients starting treatment with a drug, 16.2% had subsequent surgeries. Analysis was also conducted separately for patients with renal angiomyolipoma only, SEGA only, and both renal angiomyolipoma and SEGA. CONCLUSIONS: This analysis finds that only 58.0-61.7% of TSC patients with renal angiomyolipoma or SEGA received treatments and Medicaid patients had a longer time to initiate treatment than Commercial patients.

Conference/Value in Health Info

2017-05, ISPOR 2017, Boston, MA, USA

Value in Health, Vol. 20, No. 5 (May 2017)

Code

PND66

Topic

Health Service Delivery & Process of Care

Topic Subcategory

Treatment Patterns and Guidelines

Disease

Rare and Orphan Diseases

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