Quantitative Systems Pharmacology Modeling of Platelet Responses to Recombinant ADAMTS13 in Patients With Congenital Thrombotic Thrombocytopenic Purpura. [PDF]
ABSTRACT Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra‐rare, life‐threatening thrombotic microangiopathy caused by a severe inherited deficiency of ADAMTS13, a von Willebrand factor (VWF) cleaving enzyme. Inadequate clinical endpoint data often make it challenging to statistically power clinical trials in ultra‐rare diseases ...
McBride C +8 more
europepmc +2 more sources
ABSTRACT Introduction Von Willebrand disease (VWD) is a bleeding disorder characterized by a deficiency or dysfunction of Von Willebrand factor (VWF) and/or Factor VIII (FVIII), critical coagulation proteins. Individuals with VWD often use combination oral contraceptives (COCs) to manage heavy menstrual bleeding.
Eve Justason +4 more
wiley +1 more source
ABSTRACT Introduction Heavy menstrual bleeding (HMB) is a common and burdensome symptom in women with von Willebrand disease (VWD). Although VWD and HMB have been studied, the prevalence and impact of HMB across different age groups remains underexplored. Aim To investigate the prevalence and impact of HMB in women with VWD across different age groups.
Anna Olsson +3 more
wiley +1 more source
ADAMTS‐13 activity in plasma is rapidly measured by a new ELISA method that uses recombinant VWF‐A2 domain as substrate [PDF]
Jody L. Whitelock +5 more
openalex +1 more source
ABSTRACT Introduction Although clinical experience of a triple‐secured, plasma‐derived, von Willebrand factor (pdVWF), almost devoid of Factor VIII (FVIII) in adults with von Willebrand disease (VWD), is widely reported, its use in children is less documented.
Jenny Goudemand +10 more
wiley +1 more source
C1qTNF–related protein-1 (CTRP-1): a vascular wall protein that inhibits collagen-induced platelet aggregation by blocking VWF binding to collagen [PDF]
Gerald Lasser +8 more
openalex +1 more source
The Mirage of Factor Equivalence: Examining the Complexities of Non‐Factor Therapies in Haemophilia
ABSTRACT Background This article provides a critical analysis of the ‘factor equivalence’ concept as applied to non‐factor therapies (NFTs) for haemophilia, highlighting its limitations and clinical implications. Although factor equivalence estimates serve as a comparative tool for evaluating pro‐coagulant effects across different therapies, they may ...
Yesim Dargaud +2 more
wiley +1 more source
The role of platelet membrane proteins in the interaction of platelet-vWF-collagen.
Morio Aihara +7 more
openalex +2 more sources
A critical role for 14-3-3ζ protein in regulating the VWF binding function of platelet glycoprotein Ib-IX and its therapeutic implications [PDF]
Kesheng Dai +3 more
openalex +1 more source

