Population pharmacokinetic modelling: a useful tool to predict response to DDAVP in mild Hemophilia A - PCURVE

Academisch Medisch Centrum0 个研究点目标入组 20 人开始时间: 待定最近更新: 2024年4月15日

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•* Mild hemophilia A: defined as a FVIII:C plasma concentration of 0.06-0.40 IU/ml;
•* Administration of DDAVP for a test (intraveneous administration with 0.3 ug/ kg) between 2000 and 2015 with documented FVIII and von Willebrand plasma levels prior to and FVIII levels after DDAVP administration;
•* Administration of DDAVP in past was performed in AMC;
•* Known F8 gene mutation, preferably limited to two large mutation groups;
•* >19 and <70 years of age.

•* Severe or moderate hemophilia A patients (factor VIII *0.05 IU/ml);
•* Von Willebrand Factor plasma levels are unknown (von Willebrand disease (VWD) cannot be excluded);
•* Type 2 Normandy (2N) VWD is identified, or patient is highly likely to have type 2N VWD since there are female patients with bleeding symptoms in the pedigree;
•* Other bleeding disorders;
•* Presence of antibodies for FVIII (an inhibitor) at the time of DDAVP administration;
•* F8 genotype that never showed a >2 fold increase in VWF levels upon DDAVP administration;
•* Contraindication DDAVP use: contusio cerebri, cardiovascular disease (history, multiple cardiovascular risk factors and age > 70 years), electrolyte deficiencies, hematuria, von Willebrand disease type 2b, TTP, reduced kidney function, kidney hemorrhage.

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