Journal article
A Study of Prospective Surveillance for Inhibitors among Persons with Haemophilia in the United States
Haemophilia : the official journal of the World Federation of Hemophilia, Vol.20(2), pp.230-237
03/2014
DOI: 10.1111/hae.12302
PMCID: PMC4519821
PMID: 24261612
Abstract
nhibitors are a rare but serious complication of treatment of patients with haemophilia. Phase III clinical trials enrol too few patients to adequately assess new product inhibitor risk. This project explores the feasibility of using a public health surveillance system to conduct national surveillance for inhibitors. Staff at 17 U.S. haemophilia treatment centres (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 postenrolment 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. From January 2006 through June 2012, 1163 patients were enrolled and followed up for 3329 person-years. A total of 3048 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. 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.
Details
- Title: Subtitle
- A Study of Prospective Surveillance for Inhibitors among Persons with Haemophilia in the United States
- Creators
- J. Michael Soucie - Division of Blood Disorders, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GAConnie H Miller - Division of Blood Disorders, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GAFiona M Kelly - Division of Blood Disorders, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GAAmanda B Payne - Division of Blood Disorders, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GAMelissa Creary - Division of Blood Disorders, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GAPaula L Bockenstedt - University of Michigan Haemophilia and Coagulation Disorders, Ann Arbor, MIChristine L Kempton - Emory University, Atlanta, GAMarilyn J Manco-Johnson - Mountain States Regional Haemophilia and Thrombosis Center, University of Colorado and The Children’s Hospital, Aurora, COAnne T Neff - Vanderbilt University Medical Center, Nashville, TNHaemophilia Inhibitor Research Study Investigators
- Contributors
- Steven R Lentz (Contributor) - University of Iowa, Internal Medicine
- Resource Type
- Journal article
- Publication Details
- Haemophilia : the official journal of the World Federation of Hemophilia, Vol.20(2), pp.230-237
- DOI
- 10.1111/hae.12302
- PMID
- 24261612
- PMCID
- PMC4519821
- NLM abbreviation
- Haemophilia
- ISSN
- 1351-8216
- eISSN
- 1365-2516
- Language
- English
- Date published
- 03/2014
- Academic Unit
- Hematology, Oncology, and Blood & Marrow Transplantation; Internal Medicine
- Record Identifier
- 9984094351102771
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