US approval for bleeding disorder drug
(Max Delbrück Center for Molecular Medicine in the Helmholtz Association) 'VONVENDI,' the new drug from Baxalta Incorporated, has just been approved by the United States Food and Drug Administration. It is the first, and so far the only, recombinant protein for the treatment of von Willebrand disease, and offers those affected a new therapeutic option for the first time in more than 10 years.
Hemophilia and von Willebrand disease (VWD) are the most common congenital coagulation factor deficiencies. Patients with these disorders who experience bleeding complications are often initially managed in the emergency department (ED).
Acquired von Willebrand syndrome is implicated in the pathology of gastrointestinal bleeding (GIB) after implantation of continuous-flow left ventricular assist systems (CF-LVAS). Adults with blood type (BT) O have lower circulating levels of von Willebrand factor. In this analysis, we sought to explore the association between BT O and GIB following CF-LVAS implantation.
In this study, a typical axial flow LVAD was redesigned using aerodynamics-based strategies to improve its hemodynamics and hence clinical outcomes for recipients.
Abstract Von Willebrand factor (VWF) and coagulation factor VIII (FVIII) circulate as a complex in plasma and have a major role in the hemostatic system. VWF has a dual role in hemostasis. It promotes platelet adhesion by anchoring the platelets to the subendothelial matrix of damaged vessels and it protects FVIII from proteolytic degradation. Moreover, VWF is an acute phase protein that has multiple roles in vascular inflammation and is massively secreted from Weibel-Palade bodies upon endothelial cell activation. Activated FVIII on the other hand, together with coagulation factor IX forms the tenase complex, an ...
We report a case of AVWS associated with plasma cell myeloma. The patient was a 57-year-old male with recurrent bleeding symptoms for a few months. Physical examination was normal. Laboratory studies revealed isolated prolongation of the activated partial thromboplastin time. His factor VIII activity, von Willebrand factor (VWF) Ag, and VWF activity were low. Bone marrow aspirate showed diffuse infiltration of atypical plasma cells and erythroid line hyperplasia. PMID: 32146801 [PubMed - in process]
We report a case in whom response of both the acquired von Willebrand syndrome and smoldering multiple myeloma persist 14 months after daratumumab treatment discontinuation. AbstractAcquired von Willebrand syndrome is a rare bleeding disorder often secondary to an underlying lymphoproliferative disorder. We report a case in whom response of both the acquired von Willebrand syndrome and smoldering multiple myeloma persist 14 months after daratumumab treatment discontinuation.
Complex regional pain syndrome (CRPS) is a regional neuropathic pain syndrome. Excruciating pain often interferes with patients’ ability to function normally. Spinal cord stimulators (SCS) have been effective in treating pain along with signs of sympathetic overactivity in patients with CRPS. Implantation of SCS is a high-risk interventional procedure with the potential for serious bleeding and neurological consequences in the spine. Meticulous evaluation and careful optimization are needed in patients with bleeding disorders.
AbstractAim : Type 3 von Willebrand disease (VWD) is the most severe form of VWD, characterized by a near-total absence of von Willebrand factor (vWF) leading to a huge deficiency in plasmatic factor VIII (FVIII). VWD may be confused with hemophilia A, sometimes leading to misdiagnosis. The purpose of this work was to finalize the biological diagnosis of patients with FVIII activity deficiency in Abidjan, in order to guide the best type of management.Methods: We conducted a cross-sectional descriptive study from June 2018 to April 2019. Forty-nine patients, all of whom had lower FVIII levels or had been referred for b...
It’s common for girls and their parents to wonder if the bleeding with their periods is too often or too much. Especially in the first few years of having a period, any bleeding can feel like too much. Usually, it’s not — but sometimes it is, and it’s important for parents to know what to watch for, and when to call the doctor. In the first couple of years after periods begin, it’s really normal for periods to be irregular — and for some of them to be heavy. At the beginning, periods aren’t associated with ovulation, and the hormones and hormonal patterns that help regulate periods...
von Willebrand disease (VWD) is the most common inherited bleeding disorder and may alternatively arise as an acquired condition (AVWS). These represent deficiency and/or defects in von Willebrand factor (VWF). Closure times (CTs) obtained from the platelet function analyser (PFA) are highly sensitive to both VWD/AVWS. The current study has evaluated the utility of the PFA-100/-200 to exclude or detect laboratory identified VWD.