Abstract Details

Title Fabry disease and Stroke: Effectiveness of Enzyme Replacement Therapy (ERT) in Stroke Prevention, a Review with Meta-Analysis

Topic Cerebrovascular Disease and Interventional Neurology

Presentation(s) P4 - Poster Session 4 (5:30 PM-6:30 PM)

Poster/Presentation
Number 3-017

Objective We did a meta-analysis of the available data to review the benefit of Enzyme Replacement Therapy (ERT) for stroke prevention.

Background Fabry disease, is the most prevalent lysosomal storage disorder and notorious for its early multi-organ complications, especially ischemic stroke. Since 2001 ERT has become the mainstay treatment for Fabry patients.

Design/Methods A literature search was performed from NCBI/PubMed database without restriction of years. A systematic review of clinical cohort studies and trials was performed with pooled analysis of proportions. Inclusion criteria were cohort studies, random controlled trials (RCTs), Fabry disease on ERT or natural history and ischemic stroke outcomes. The pooled proportion and the confidence interval (CI) for stroke recurrent ratio are shown for ERT treatment group and native treatment group.

Results A total of 7 cohort studies and 2 RCTs involving a total of 7513 participants (1471 on ERT vs 6042 native treatment) proved eligible. The pooled proportions analysis shows stroke recurrent ratio in ERT treatment group is 8.2% [95% CI 0.038, 0.126]; in native-treatment group: 16% [95% CI 0.102, 0.217]. Effective differences favored ERT treatment group over native treatment group (p=0.03).

Conclusions Our meta-analysis bases on the currently available data showed that ERT for Fabry disease has beneficial effect on stroke prevention, Female carriers and atypically affected males should be started on ERT early as soon as diagnosis is made. Further studies are warranted to support the role of ERT in stroke prevention.

Authors/Disclosures
Sen Sheng, MD PRESENTER	Dr. Sheng has nothing to disclose.
	No disclosure on file
Yohei Harada, MD (UCB Biopharma)	Dr. Harada has nothing to disclose.
Hisham G. Elkhider, MD	Dr. Elkhider has nothing to disclose.
Nidhi Kapoor, MD, MBBS	Dr. Kapoor has nothing to disclose.
Onteddu Reddy	No disclosure on file
Krishna Nalleballe, MD, FAAN	Dr. Nalleballe has nothing to disclose.

��ɫ��

��ɫ��