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Risk Factors for High-Titer Inhibitor Development in Children with Hemophilia A: Results of a Cohort Study
- Source :
- BioMed Research International, BioMed Research International, Vol 2013 (2013)
- Publication Year :
- 2013
- Publisher :
- Hindawi Limited, 2013.
-
Abstract
- Among the discussed risk factors for high-titre inhibitor (HRI) development in patients with hemophilia A (HA) are high dose FVIII replacement therapy and use of recombinant FVIII concentrates (rFVIII). The aim of this study was to evaluate the aforementioned risk factors for HRI development in children with hemophilia A ≤2%. About 288 ascertained PUPs (Israel and Germany) were followed after initial HA diagnosis over 200 exposure days. Inhibitor-free survival, hazard ratios (HR), and 95% confidence intervals (CIs) were calculated. Adjustment was performed for factor VIII concentrates, median single dose over the first three months of treatment, first FVIII administration before the age of three months, presence of risk HA gene mutations, “intensive treatment moments” and “year of birth” (proxy for different treatment periods). HRI occurred in 71/288 children (24.7%). In multivariate analysis adjusted for “year of birth”, underlying risk gene mutations (HR/CI: 2.37/1.40–3.99), FVIII dose, measured per one IU increase per kgbw (HR/CI: 1.05/1.04–1.07), and first FVIII administration before the age of three months showed a significant impact on HR development. The risk of HRI development was similar for recombinant or plasmatic FVIII products. Children at risk should be treated with carefully calculated lower dose regimens, adapted to individual bleeding situations.
- Subjects :
- Male
Pediatrics
medicine.medical_specialty
Multivariate analysis
Article Subject
Adolescent
lcsh:Medicine
Risk gene
Hemophilia A
General Biochemistry, Genetics and Molecular Biology
Cohort Studies
Risk Factors
hemic and lymphatic diseases
medicine
Humans
In patient
ddc:610
High titer
Child
Factor VIII
General Immunology and Microbiology
business.industry
Intensive treatment
lcsh:R
Hazard ratio
Infant, Newborn
Infant
General Medicine
Confidence interval
Treatment Outcome
Child, Preschool
Multivariate Analysis
Mutation
Clinical Study
Female
business
Cohort study
Subjects
Details
- ISSN :
- 23146141 and 23146133
- Volume :
- 2013
- Database :
- OpenAIRE
- Journal :
- BioMed Research International
- Accession number :
- edsair.doi.dedup.....9c7e5c799d56baa58a08823eb384daad