![Phenotypic characteristics and genetic data of the index patient. (A) The phenotypic and genetic data of the index patient and his parents. No, no VWF gene large deletion was detected. (B) VWF:Ag, VWF:GPIb, and FVIII:C pharmacokinetics in the type 3 VWD index patient following administration of Voncento VWF/FVIII concentrate. Blood samples were obtained at multiple time points, before and at 15 and 30 minutes, and 1, 2, 4, 6, 8, 12, and 24 hours after infusion of Voncento (50 IU VWF:RCo/kg). The amount of VWF:Ag, VWF:GPIb, and FVIII:C was determined from collected plasma samples. The patient did not receive any VWF product 1 day before the PK study period. Data were analyzed by using GraphPad Prism version 8.0.1. The half-life (T1/2) of VWF:Ag, VWF:GPIb, and FVIII:C was calculated based on 1-phase exponential decay half-life of VWF:Ag, VWF:GPIb, and FVIII:C was determined as 5.2 hours (95% confidence intervals [CI], 3.6-7.7), 3.9 hours (95% CI, 3.2-4.7, and 14.9 hours (95% CI, 10.3-23.6), respectively. FVIII:C showed longer T1/2 compared with VWF markers apparently from the effect of increasing endogenous FVIII levels combined with decreasing levels of exogenous FVIII. VWF:RCo, von Willebrand factor ristocetin cofactor assay.](https://ash.silverchair-cdn.com/ash/content_public/journal/bloodadvances/6/3/10.1182_bloodadvances.2021005895/4/advancesadv2021005895f1.png?Expires=1742851819&Signature=D6O1fHbXPoHH7LGMlaZbHfJpuS4WiDRh1onBvTQPz3wbJn5gBauamnMceaNtPfFkKnq-xOW83LB6yu4qw4ZdkIL243O5UJzNLi0zxesx93Ptz2-BgSwc3UvmOfpFxjKohxtQMZjNTXTgzB7Qs5UzSYV7YY4nvHvS1Ar3tOVIy23W4dOpiZ0~6bu1tBamzaetpegdKG544dZewv9FrxFstsCCQNtW0~1qkKELl0Gh4-UP3R7bvl9MjkIgFlEJ4cngVMqYMwMqjOXns5kGSO32NnkTGL-9r-WI3mpI2mWhZl4qKFGEOCslRDFiGv1azCZX7JXELrrD0Hw57ES9BEVSAw__&Key-Pair-Id=APKAIE5G5CRDK6RD3PGA)
Phenotypic characteristics and genetic data of the index patient. (A) The phenotypic and genetic data of the index patient and his parents. No, no VWF gene large deletion was detected. (B) VWF:Ag, VWF:GPIb, and FVIII:C pharmacokinetics in the type 3 VWD index patient following administration of Voncento VWF/FVIII concentrate. Blood samples were obtained at multiple time points, before and at 15 and 30 minutes, and 1, 2, 4, 6, 8, 12, and 24 hours after infusion of Voncento (50 IU VWF:RCo/kg). The amount of VWF:Ag, VWF:GPIb, and FVIII:C was determined from collected plasma samples. The patient did not receive any VWF product 1 day before the PK study period. Data were analyzed by using GraphPad Prism version 8.0.1. The half-life (T1/2) of VWF:Ag, VWF:GPIb, and FVIII:C was calculated based on 1-phase exponential decay half-life of VWF:Ag, VWF:GPIb, and FVIII:C was determined as 5.2 hours (95% confidence intervals [CI], 3.6-7.7), 3.9 hours (95% CI, 3.2-4.7, and 14.9 hours (95% CI, 10.3-23.6), respectively. FVIII:C showed longer T1/2 compared with VWF markers apparently from the effect of increasing endogenous FVIII levels combined with decreasing levels of exogenous FVIII. VWF:RCo, von Willebrand factor ristocetin cofactor assay.