TY  - JOUR
A1  - Halimeh, Susan
A1  - Bidlingmaier, Christoph
A1  - Heller, Christine
A1  - Gutsche, Sven
A1  - Holzhauer, Susanne
A1  - Kenet, Gili
A1  - Kurnik, Karin
A1  - Manner, Daniela
A1  - Iorio, Alfonso
A1  - Nowak-Göttl, Ulrike
T1  - Risk factors for high-titer inhibitor development in children with hemophilia a: Results of a cohort study
T2  - BioMed research international
N2  - 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.
Y1  - 2013
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/54314
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-543146
SN  - 2314-6141
SN  - 2314-6133
N1  - Copyright © 2013 Susan Halimeh et al. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
VL  - 2013
IS  - Art. 901975
SP  - 1
EP  - 7
PB  - Hindawi
CY  - New York [u. a.]
ER  -