Document Detail

The principles of PK-tailored prophylaxis.
MedLine Citation:
PMID:  24169861     Owner:  NLM     Status:  In-Data-Review    
While prophylaxis with factor VIII (FVIII) is considered the first choice therapy for patients with severe haemophilia A the optimal prophylaxis regimen is still under scientific debate. A recent study demonstrated efficacy and safety of a PK-tailored prophylaxis regimen with rFVIII (ADVATE) aimed to maintain FVIII trough levels of ≥1% (19). The annual bleed rate (ABR) could be significantly reduced compared to the previous on-demand treatment period (p < 0.0001) and bodily pain, a health-related quality of life dimension of the SF-36v1 questionnaire also significantly improved (p = 0.0007). Thus PK-tailored prophylaxis with ADVATE might offer a valid alternative to standard prophylaxis. Open issues to be considered for implementation of PK-tailored prophylaxis are: What FVIII trough level is needed to prevent any bleed? Do patients with target joints need higher FVIII trough levels to stay bleed-free? Are there user-friendly tools available to calculate individualized PK-driven prophylaxis doses and frequency without the need for a full 9-sample PK curve? Current knowledge on these aspects as well as some considerations about the future of PK-tailored prophylaxis is discussed.
A J Reininger; H E Chehadeh
Publication Detail:
Type:  Journal Article    
Journal Detail:
Title:  Hämostaseologie     Volume:  33 Suppl 1     ISSN:  0720-9355     ISO Abbreviation:  Hamostaseologie     Publication Date:  2013 Oct 
Date Detail:
Created Date:  2013-10-30     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  8204531     Medline TA:  Hamostaseologie     Country:  Germany    
Other Details:
Languages:  eng     Pagination:  S32-5     Citation Subset:  IM    
Prof. Dr. med. Armin J. Reininger, Baxter Innovations GmbH, Industriestr. 67, 1220 Vienna, Austria, Tel. +43/1/201 00-247 34 94 Fax +43/1/201 00-530, E-mail:
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