Talbird SE, La EM. Treatment patterns, health care resource use, and financial impact of CADASIL in the United States. Poster presented at the 2019 ISPOR 24th Annual International Meeting; May 21, 2019. New Orleans, LA.

OBJECTIVES: Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL) is a rare, genetic disorder that affects small cerebral artery blood flow leading to neurodegeneration and eventually vascular dementia. This study used patient-reported data from a publicly available registry to evaluate treatment patterns, health care resource use (HCRU), and financial impact associated with CADASIL in the United States (US).

METHODS: Data from the CADASIL Family Registry were analyzed for US participants with a CADASIL diagnosis using the most recent completed survey between August 2015 and June 8, 2018. Outcomes of interest included select measures of CADASIL-related treatment patterns and HCRU (e.g., provider types, treatments, frequency of medical visits), as well as financial impact of CADASIL (e.g., annual medication/treatment costs, overall financial impact). All analyses were conducted in SAS version 9.4 (Cary, NC).

RESULTS: Out of 191 total US registry participants, 62 (32.5%) provided information on HCRU and financial impact. Among these respondents, the majority reported seeing a neurologist for CADASIL (82.3%), although a variety of other provider types were also reported. Frequency of medical provider visits ranged from 8.1% reporting no annual visits to 14.5% reporting ≥1 visit monthly. Eighteeen patients (29.0%) reported ≥1 emergency department visit and 9 patients (14.5%) reported ≥1 hospitalization in the last year. Most patients reported using some type of symptomatic treatment for CADASIL (88.7%), with nearly one-third of patients also using alternative therapies (32.3%). Patients most frequently reported annual out-of-pocket treatment and medication costs between $1,000-$10,000 (29.0%) and that CADASIL was associated with a major financial impact (38.7%).

CONCLUSIONS: Although no disease-modifying treatments for CADASIL exist, for many patients CADASIL is associated with considerable HCRU and financial impact. Additional research is needed to better understand HCRU over the course of the disorder and the value of potential disease-modifying treatments for CADASIL.

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