i
A Study of Prospective Surveillance for Inhibitors among Persons with Haemophilia in the United States
-
Nov 22 2013
-
-
Source: Haemophilia. 20(2):230-237.
[PDF-595.26 KB]
Details:
-
Alternative Title:Haemophilia
-
Personal Author:
-
Corporate Authors:
-
Description:Introduction
Inhibitors are a rare but serious complication of treatment of patients with haemophilia. Phase III clinical trials enroll too few patients to adequately assess new product inhibitor risk.
Aim
This project explores the feasibility of using a public health surveillance system to conduct national surveillance for inhibitors.
Methods
Staff at 17 U.S. haemophilia treatment centers (HTC) enrolled patients with haemophilia A and B into this prospective study. HTC staff provided detailed historic data on product use and inhibitors at baseline, and post-enrollment patients provided monthly detailed infusion logs. A central laboratory performed inhibitor tests on blood specimens that were collected at baseline, annually, prior to any planned product switch or when clinically indicated. The central laboratory also performed genotyping of all enrolled patients.
Results
From January 2006 through June 2012, 1163 patients were enrolled and followed for 3,329 person years. A total of 3,048 inhibitor tests were performed and 23 new factor VIII inhibitors were identified, 61%% of which were not clinically apparent. Infusion logs were submitted for 113,205 exposure days. Genotyping revealed 431 distinct mutations causing haemophilia, 151 of which had not previously been reported elsewhere in the world.
Conclusion
This study provided critical information about the practical issues that must be addressed to successfully implement national inhibitor surveillance. Centralized testing with routine monitoring and confirmation of locally identified inhibitors will provide valid and representative data with which to evaluate inhibitor incidence and prevalence, monitor trends in occurrence rates, and identify potential inhibitor outbreaks associated with products.
-
Subjects:
-
Source:
-
Pubmed ID:24261612
-
Pubmed Central ID:PMC4519821
-
Document Type:
-
Funding:
-
Place as Subject:
-
Collection(s):
-
Main Document Checksum:
-
Download URL:
-
File Type:
