Document Detail


Inhibitor development in previously untreated patients with hemophilia A: a prospective long-term follow-up comparing plasma-derived and recombinant products.
MedLine Citation:
PMID:  12098090     Owner:  NLM     Status:  MEDLINE    
Abstract/OtherAbstract:
In order to assess inhibitor development in previously untreated patients (PUPs) with severe (factor VIII [FVIII]<1%) and moderate (FVIII 1 to 5%) hemophilia A, a prospective study was initiated in 1976. During the 23-year study period, 72 hemophiliacs were frequently exposed prophylactically or on demand to plasma-derived (pd) (n = 51) or recombinant FVIII (rFVIII) (n = 21) concentrates (median 270 exposure days [ED]). Inhibitor testing was performed before the first exposure and at regular intervals thereafter. Of the 72 hemophilia A patients, 22 (32%) developed an inhibitor after 15 ED in median (range 4 to 195); 17 (77%) were high responders (>5 Bethesda Units [BU]), and the remaining 5 patients (23%) were low responders (>0.6 to 5 BU). The severely affected patients (n = 46) showed a significantly higher frequency of inhibitor formation (43%) than did the moderate ones (8%). Comparing the severely affected patients receiving pd products exclusively (n = 35) with those treated with recombinant concentrate (n = 11), 37% of the pd group developed a high-titer inhibitor (>5 BU, median 290 ED in noninhibitor patients) and 36% of the recombinant group (median 49 ED in the noninhibitor patients). However, the exposure status of the recombinant noninhibitor patients is rather low and therefore remains a high risk of developing further inhibitors in the future. The mutation type profile revealed no difference between the pd- and the recombinant-treated patients.
Authors:
Wolfhart Kreuz; Carmen Escuriola Ettingshausen; Alex Zyschka; Johannes Oldenburg; Inmaculada Martinez Saguer; Silke Ehrenforth; Thomas Klingebiel
Publication Detail:
Type:  Journal Article    
Journal Detail:
Title:  Seminars in thrombosis and hemostasis     Volume:  28     ISSN:  0094-6176     ISO Abbreviation:  Semin. Thromb. Hemost.     Publication Date:  2002 Jun 
Date Detail:
Created Date:  2002-07-04     Completed Date:  2003-02-25     Revised Date:  2006-03-07    
Medline Journal Info:
Nlm Unique ID:  0431155     Medline TA:  Semin Thromb Hemost     Country:  United States    
Other Details:
Languages:  eng     Pagination:  285-90     Citation Subset:  IM    
Affiliation:
The Centre of Pediatrics III, Department of Hematology and Oncology, Johann-Wolfgang-Goethe-University Hospital, Frankfurt am Main and the Institute of Human Genetics (J. Oldenburg) University, Würzburg, Germany. carmen.escuriola@kgu.de
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MeSH Terms
Descriptor/Qualifier:
Autoantibodies / blood*
Cohort Studies
Factor VIII / administration & dosage,  immunology*
Follow-Up Studies
Hemophilia A / drug therapy*,  genetics,  immunology
Hemophilia B / drug therapy,  genetics,  immunology
Humans
Mutation
Prospective Studies
Recombinant Proteins / immunology*
Risk
Time Factors
Chemical
Reg. No./Substance:
0/Autoantibodies; 0/F8 protein, human; 0/Recombinant Proteins; 9001-27-8/Factor VIII

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine


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