The principles of PK-tailored prophylaxis

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 t...

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Bibliographic Details
Published inHämostaseologie Vol. 33 Suppl 1; p. S32
Main Authors Reininger, A J, Chehadeh, H E
Format Journal Article
LanguageEnglish
Published Germany 01.01.2013
Subjects
Biomarkers - blood
Dose-Response Relationship, Drug
Drug Monitoring - methods
Evidence-Based Medicine
Factor VIII - administration & dosage
Factor VIII - metabolism
Hemophilia A - blood
Hemophilia A - complications
Hemophilia A - prevention & control
Hemorrhage - blood
Hemorrhage - etiology
Hemorrhage - prevention & control
Humans
Precision Medicine - methods
Recombinant Proteins - administration & dosage
Recombinant Proteins - metabolism
Treatment Outcome
FVIII trough level
ABR
Haemophilia A
annual bleed rate
individualized treatment
target joint
PK tailored prophylaxis
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Summary: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.
ISSN:0720-9355
DOI:10.1055/s-0037-1619800