Hemophilia A Clinical Trial
Official title:
Prevalence and Epitope Specificity of Non-neutralizing Antibodies in Haemophilia A Patients Without Inhibitors, Immunogenicity of B Domain: A Prospective Study
Antibodies (Abs) directed against factorVIII (FVIII) remain the main iatrogenic complication
in haemophilia A (HA) patients. Anti-FVIII Abs inhibiting pro-coagulant properties of the
molecule are named inhibitors whereas Abs directed towards non-functional epitopes are named
non-neutralizing antibodies (NNA). These NNA are poorly studied and their prevalence is
ill-defined.
In a recent retrospective study the investigators evaluated, in a cohort of 210 patients
without inhibitor, the NNA prevalence and the NNA epitope specificity against the heavy
chain (HC)or the light chain(LC). For the first time, the investigators used two x-MAP based
assays: the first to determine the specificity of anti-FVIII Abs against the HC or the LC,
the second to display Abs directed towards the B domain. NNA were found in 38 out of 210
patients (18).
Among this NNA positive population, 74% and 13% of patients had anti-FVIII Abs against both
chains. The proportion of NNA directed towards the B domain was 18%.
Considering an approximate inhibitor prevalence of 30% and a NNA prevalence of 19% in severe
HA patients, approximately 50% of severe HA patients develop an immune response against
infused FVIII. Due to their unclear relevance, the NNA detection does not yet belong to the
routine clinical practice.
However, in 2006, Dimichele advancedf a hypothesis concerning the influence of NNA on the
variations in the kinectics of FVIII observed in certain patients.
The mechanism explaining the role of these NNA in the FVIII in the FVIII kinectics has not
still been demonstrated.
The investigators propose to perform a multicentre prospective study with the aim to
confirm, in severe, moderate and mild HA treated patietns, the NNA prevalence observed in
our retrospective study, to study the evolution over time of the epitopemapping of these NNA
and to explore the correlation between these NNA and clinical/biological parameters.
n/a
Observational Model: Cohort, Time Perspective: Prospective
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