LONG-TERM COSTS AND CONSEQUENCES OF PATIENTS WITH FAMILIAL CHYLOMICRONEMIA SYNDROME – A SIMULATION MODEL APPROACH
Author(s)
Lin F, Thomas S, Calado F, Clegg J
Novartis, East Hanover, NJ, USA
Presentation Documents
BACKGROUND: Familial chylomicronemia syndrome (FCS) is a rare genetic disorder characterized by deficiency of lipoprotein lipase, causing accumulation of chylomicrons. An estimated 0.1-0.2 per 100,000 people has FCS worldwide. FCS patients present massively elevated triglyceride levels (typically >2,000 mg/dL), resulting in increased risk of recurring acute pancreatitis. Standard triglyceride lowering medications are ineffective for FCS patients, who rely on restrictive low fat diet to control their triglyceride. There is limited literature about long-term progression, the burden of illness or consequences of acute pancreatitis for FCS. OBJECTIVES: To estimate long term disease progression, costs and consequences of FCS. METHODS: An individual Monte Carlo simulation model was built to track disease progression of a cohort of FCS patients with a mean age of 37.8 years, 60% male, and a mean triglyceride level of 2,741 mg/dL. The model projected the number of acute pancreatitis events, mortality and medical costs. Benefits of a hypothetical triglyceride reduction intervention were assessed. RESULTS: With standard diet control, the average life expectancy of the studied cohort was estimated to be 16.45 years. These patients were expected to experience 10.16 episodes of acute pancreatitis during their lifetime, resulting in 80.7 inpatient days. The discounted lifetime cost of acute pancreatitis was projected to be $154,126 per patient. The cumulative mortality due to acute pancreatitis was estimated to be 54.3%. Should an intervention reduce triglyceride levels by 50% in FCS patients, the life expectancy would be increased by 3.16 years and 7.72 fewer episodes of acute pancreatitis would occur, preventing 61.21 inpatient days and saving $118,594 in medical cost. CONCLUSIONS: FCS patients are at high risk of life-threatening and costly acute pancreatitis. Reduction in triglyceride levels has a significant impact of morbidity and mortality associated with acute pancreatitis. An effective triglyceride lowering intervention could mitigate the consequences of FCS significantly.
Conference/Value in Health Info
2014-11, ISPOR Europe 2014, Amsterdam, The Netherlands
Value in Health, Vol. 17, No. 7 (November 2014)
Code
PND54
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Rare and Orphan Diseases